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Clinical Trial Results:
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multi-Center Study to Investigate the Safety and Efficacy of MultiStem (PF-05285401) in Subjects With Moderate to Severe Ulcerative Colitis

Due to the EudraCT – Results system being out of service between 31 July 2015 and 12 January 2016, these results have been published in compliance with revised timelines.

Summary
EudraCT number	2010-022766-27
Trial protocol	SE BE HU SK IT
Global end of trial date	19 Nov 2014

Results information
Results version number	v1(current)
This version publication date	27 Jul 2016
First version publication date	27 Jul 2016
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

B3041001

ISRCTN number

US NCT number

WHO universal trial number (UTN)

Sponsor organisation name

Pfizer, Inc.

Sponsor organisation address

235 E 42nd Street, New York, United States, 10017

Public contact

Pfizer, Inc., Pfizer ClinicalTrials.gov Call Center, 001 8007181021, ClinicalTrials.gov_Inquiries@pfizer.com

Scientific contact

Pfizer ClinicalTrials.gov Call Center, Pfizer, Inc., 001 8007181021, ClinicalTrials.gov_Inquiries@pfizer.com

Is trial part of an agreed paediatric investigation plan (PIP)

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Analysis stage

Final

Date of interim/final analysis

08 Jul 2015

Is this the analysis of the primary completion data?

Yes

Primary completion date

19 Nov 2014

Global end of trial reached?

Yes

Global end of trial date

19 Nov 2014

Was the trial ended prematurely?

Main objective of the trial

The main objective of the trial was to evaluate the safety and tolerability of intravenous doses of MultiStem in moderate-to-severe ulcerative colitis.

Protection of trial subjects

The study used an independent External Data Monitoring Committee (EDMC), who was responsible for ongoing monitoring of the safety of subjects according to the Charter. The recommendations made by the EDMC to alter the conduct of the study was forwarded to Pfizer for final decision.

Background therapy

Evidence for comparator

Actual start date of recruitment

28 Feb 2011

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

Canada: 4

Country: Number of subjects enrolled

Germany: 12

Country: Number of subjects enrolled

Hungary: 10

Country: Number of subjects enrolled

Italy: 2

Country: Number of subjects enrolled

Slovakia: 5

Country: Number of subjects enrolled

Sweden: 9

Country: Number of subjects enrolled

United States: 63

Worldwide total number of subjects

105

EEA total number of subjects

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

Screening details

There were 3 cohorts planned. Cohorts 1 and 2 each included 9 subjects. The size of Cohort 3 was reviewed as the study progressed, based purely upon emerging variability estimates of the primary endpoints.

Period 1 title

Overall Study

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Investigator, Subject

Are arms mutually exclusive

Yes

Cohort 1: Placebo, MultiStem 300

Arm description

Subjects received a single dose (Day 1) or 3 doses (Day 1, Week 1, Week 2) of placebo infusion, followed by a single dose of MultiStem 300 Million Cells infusion at Week 8.

Arm type

Experimental

Investigational medicinal product name

MultiStem

Investigational medicinal product code

Other name

Pharmaceutical forms

Infusion

Routes of administration

Intravenous use

Dosage and administration details

300

Cohort 2: Placebo, MultiStem 750

Arm description

Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

Arm type

Experimental

Investigational medicinal product name

MultiStem

Investigational medicinal product code

Other name

Pharmaceutical forms

Infusion

Routes of administration

Intravenous use

Dosage and administration details

750

Cohort 1: MultiStem 300, Placebo

Arm description

Subjects received a single dose of MultiStem 300 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Arm type

Experimental

Investigational medicinal product name

MultiStem

Investigational medicinal product code

Other name

Pharmaceutical forms

Infusion

Routes of administration

Intravenous use

Dosage and administration details

300

Cohort 1: MultiStem 300 (x3), Placebo

Arm description

Subjects received up to 3 doses of MultiStem 300 Million Cells infusion (Day 1, Week, Week 2), followed by a single dose of placebo infusion at Week 8.

Arm type

Experimental

Investigational medicinal product name

MultiStem

Investigational medicinal product code

Other name

Pharmaceutical forms

Infusion

Routes of administration

Intravenous use

Dosage and administration details

300

Cohort 2: MultiStem 750, Placebo

Arm description

Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Arm type

Experimental

Investigational medicinal product name

MultiStem

Investigational medicinal product code

Other name

Pharmaceutical forms

Infusion

Routes of administration

Intravenous use

Dosage and administration details

750

Cohort 3: MultiStem 750, MultiStem 750

Arm description

Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

Arm type

Experimental

Investigational medicinal product name

MultiStem

Investigational medicinal product code

Other name

Pharmaceutical forms

Infusion

Routes of administration

Intravenous use

Dosage and administration details

750

Cohort 3: MultiStem 750, Placebo

Arm description

Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Arm type

Experimental

Investigational medicinal product name

MultiStem

Investigational medicinal product code

Other name

Pharmaceutical forms

Infusion

Routes of administration

Intravenous use

Dosage and administration details

750

Cohort 3: Placebo, MultiStem 750

Arm description

Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

Arm type

Experimental

Investigational medicinal product name

MultiStem

Investigational medicinal product code

Other name

Pharmaceutical forms

Infusion

Routes of administration

Intravenous use

Dosage and administration details

750

Cohort 3: Placebo, Placebo

Arm description

Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Arm type

Placebo

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Pharmaceutical forms

Infusion

Routes of administration

Intravenous use

Dosage and administration details

placebo

Number of subjects in period 1	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Started	2	3	2	4	6	23	25	19	21
Completed	0	2	2	1	2	13	17	15	13
Not completed	2	1	0	3	4	10	8	4	8
Consent withdrawn by subject	2	1	-	2	-	4	2	3	3
Adverse event, non-fatal	-	-	-	1	-	2	-	-	1
Insufficient Clinical Response	-	-	-	-	1	3	6	-	3
Unspecified	-	-	-	-	2	-	-	-	-
Lost to follow-up	-	-	-	-	1	1	-	1	1

Period 2 title

Pooled Cohort 3

Is this the baseline period?

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator

Are arms mutually exclusive

Pooled MultiStem

Arm description

Arm type

Experimental

Investigational medicinal product name

MultiStem

Investigational medicinal product code

Other name

Pharmaceutical forms

Infusion

Routes of administration

Intravenous use

Dosage and administration details

350 M or 750 M

Pooled Placebo

Arm description

Arm type

Placebo

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Pharmaceutical forms

Infusion

Routes of administration

Intravenous use

Dosage and administration details

placebo

Number of subjects in period 2	Pooled MultiStem	Pooled Placebo
Started	48	40
Completed	30	28
Not completed	18	12
Consent withdrawn by subject	6	6
Adverse event, non-fatal	2	1
Insufficient Clinical Response	9	3
Lost to follow-up	1	2

Baseline characteristics

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Reporting group title

Cohort 1: Placebo, MultiStem 300

Reporting group description

Subjects received a single dose (Day 1) or 3 doses (Day 1, Week 1, Week 2) of placebo infusion, followed by a single dose of MultiStem 300 Million Cells infusion at Week 8.

Reporting group title

Cohort 2: Placebo, MultiStem 750

Reporting group description

Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

Reporting group title

Cohort 1: MultiStem 300, Placebo

Reporting group description

Subjects received a single dose of MultiStem 300 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Reporting group title

Cohort 1: MultiStem 300 (x3), Placebo

Reporting group description

Subjects received up to 3 doses of MultiStem 300 Million Cells infusion (Day 1, Week, Week 2), followed by a single dose of placebo infusion at Week 8.

Reporting group title

Cohort 2: MultiStem 750, Placebo

Reporting group description

Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Reporting group title

Cohort 3: MultiStem 750, MultiStem 750

Reporting group description

Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

Reporting group title

Cohort 3: MultiStem 750, Placebo

Reporting group description

Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Reporting group title

Cohort 3: Placebo, MultiStem 750

Reporting group description

Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

Reporting group title

Cohort 3: Placebo, Placebo

Reporting group description

Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Reporting group values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo	Total
Number of subjects	2	3	2	4	6	23	25	19	21	105
Age categorical
Units: Subjects
In Utero	0	0	0	0	0	0	0	0	0	0
Pre-term newborn - gestational age <37 wk	0	0	0	0	0	0	0	0	0	0
Newborns (0-27 days)	0	0	0	0	0	0	0	0	0	0
Infants and toddlers (28 days-23 months)	0	0	0	0	0	0	0	0	0	0
Children (2-11 years)	0	0	0	0	0	0	0	0	0	0
Adolescents (12-17 years)	0	0	0	0	0	0	0	0	0	0
Adults (18-64 years)	1	3	1	4	6	21	23	18	18	95
Elderly (From 65-84 years)	1	0	1	0	0	2	2	1	3	10
Elderly 85 years and over	0	0	0	0	0	0	0	0	0	0
Age Continuous \|
Units: years
arithmetic mean (standard deviation)	61 ( 7.1 )	52 ( 7 )	58 ( 19.8 )	45 ( 11.1 )	40 ( 14 )	43.4 ( 12.8 )	38.8 ( 13.4 )	39.8 ( 12.5 )	42.5 ( 15.7 )	-
Gender, Male/Female
Units: participants
Male	2	2	2	3	1	16	13	16	14	69
Female	0	1	0	1	5	7	12	3	7	36

End points

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Reporting group title

Cohort 1: Placebo, MultiStem 300

Reporting group description

Subjects received a single dose (Day 1) or 3 doses (Day 1, Week 1, Week 2) of placebo infusion, followed by a single dose of MultiStem 300 Million Cells infusion at Week 8.

Reporting group title

Cohort 2: Placebo, MultiStem 750

Reporting group description

Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

Reporting group title

Cohort 1: MultiStem 300, Placebo

Reporting group description

Subjects received a single dose of MultiStem 300 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Reporting group title

Cohort 1: MultiStem 300 (x3), Placebo

Reporting group description

Subjects received up to 3 doses of MultiStem 300 Million Cells infusion (Day 1, Week, Week 2), followed by a single dose of placebo infusion at Week 8.

Reporting group title

Cohort 2: MultiStem 750, Placebo

Reporting group description

Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Reporting group title

Cohort 3: MultiStem 750, MultiStem 750

Reporting group description

Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

Reporting group title

Cohort 3: MultiStem 750, Placebo

Reporting group description

Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Reporting group title

Cohort 3: Placebo, MultiStem 750

Reporting group description

Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

Reporting group title

Cohort 3: Placebo, Placebo

Reporting group description

Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Reporting group title

Pooled MultiStem

Reporting group description

Reporting group title

Pooled Placebo

Reporting group description

Primary: Change From Baseline in Endoscopic Score (as Measured by Modified Baron Score) at Week 8

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End point title

Change From Baseline in Endoscopic Score (as Measured by Modified Baron Score) at Week 8

End point description

Modified Baron Score is an instrument designed to measure endoscopic activity of ulcerative colitis. It classifies the mucosal inflammation in 4 grades (0=normal, 1=granular mucosa with an abnormal vascular pattern, 2=friable mucosa, 3=microulceration with spontaneous bleeding, 4=gross ulceration with spontaneous bleeding).

End point type

Primary

End point timeframe

Baseline and Week 8

End point values	Pooled MultiStem	Pooled Placebo
Number of subjects analysed	48	40
Units: scores on a scale
arithmetic mean (standard deviation)
Baseline	3.13 ( 1.104 )	3.1 ( 1.128 )
Change at Week 8	0.1 ( 1.134 )	-0.3 ( 1.091 )

Change From Baseline in Endoscopic Score (Week 8)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo

Comparison groups

Pooled MultiStem v Pooled Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.96 ^[1]

Method

ANCOVA

Parameter type

Mean difference (final values)

Point estimate

0.4

Confidence interval

level

80%

sides

2-sided

lower limit

0.12

upper limit

0.69

Variability estimate

Standard error of the mean

Dispersion value

0.223

Notes
[1] - 1-sided p-value

Primary: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 4

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End point title

Change From Baseline in Rectal Bleeding Mayo Subscore at Week 4

End point description

Mayo Score is an instrument designed to measure disease activity of ulcerative colitis. Mayo subscores for rectal bleeding range from 0 to 3, with higher scores indicating more severe disease.

End point type

Primary

End point timeframe

Baseline and Week 4

End point values	Pooled MultiStem	Pooled Placebo
Number of subjects analysed	48	40
Units: scores on a scale
arithmetic mean (standard deviation)
Baseline	1.42 ( 0.942 )	1.23 ( 0.832 )
Change at Week 4	-0.44 ( 0.943 )	-0.38 ( 0.705 )

Rectal Bleeding Mayo Subscore (Week 4)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 4)

Comparison groups

Pooled MultiStem v Pooled Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.54 ^[2]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

0.02

Confidence interval

level

80%

sides

2-sided

lower limit

-0.19

upper limit

0.22

Variability estimate

Standard error of the mean

Dispersion value

0.161

Notes
[2] - 1-sided p-value

Primary: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 8

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End point title

Change From Baseline in Rectal Bleeding Mayo Subscore at Week 8

End point description

Mayo Score is an instrument designed to measure disease activity of ulcerative colitis. Mayo subscores for rectal bleeding range from 0 to 3, with higher scores indicating more severe disease.

End point type

Primary

End point timeframe

Baseline and Week 8

End point values	Pooled MultiStem	Pooled Placebo
Number of subjects analysed	48	40
Units: scores on a scale
arithmetic mean (standard deviation)	-0.46 ( 1.051 )	-0.43 ( 0.874 )

Rectal Bleeding Mayo Subscore (Week 8)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 8)

Comparison groups

Pooled MultiStem v Pooled Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.67 ^[3]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

0.08

Confidence interval

level

80%

sides

2-sided

lower limit

-0.15

upper limit

0.3

Variability estimate

Standard error of the mean

Dispersion value

0.174

Notes
[3] - 1-sided p-value

Primary: Number of Subjects with Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

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End point title

Number of Subjects with Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) ^[4]

End point description

An AE was any untoward medical occurrence in a subject who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 30 days after last dose that were absent before treatment or that worsened relative to pre-treatment state. AEs included both SAEs and non-SAEs.

End point type

Primary

End point timeframe

Baseline up to Week 52

Notes
[4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No statistical analysis was planned for this primary endpoint. Adverse events were reported in accordance with the sponsor reporting standards.

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: subjects
AEs	2	3	0	4	5	20	18	16	19
SAEs	1	0	0	3	0	7	4	5	4

No statistical analyses for this end point

Primary: Incidence of Treatment-Emergent AEs by System Organ Class (SOC)

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End point title

Incidence of Treatment-Emergent AEs by System Organ Class (SOC) ^[5]

End point description

End point type

Primary

End point timeframe

Baseline up to Week 52

Notes
[5] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No statistical analysis was planned for this primary endpoint. Adverse events were reported in accordance with the sponsor reporting standards.

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: subjects
BLOOD AND LYMPHATIC SYSTEM DISORDERS	0	0	0	0	0	5	0	3	4
CARDIAC DISORDERS	0	0	0	0	0	1	0	1	1
EAR AND LABYRINTH DISORDERS	0	0	0	0	0	1	0	1	1
ENDOCRINE DISORDERS	0	0	0	0	0	0	1	1	0
EYE DISORDERS	0	0	0	0	0	1	0	0	3
GASTROINTESTINAL DISORDERS	2	1	0	2	4	13	10	11	12
GENERAL DISORDERS	2	1	0	3	1	8	5	5	6
IMMUNE SYSTEM DISORDERS	0	0	0	1	0	0	2	0	0
INFECTIONS AND INFESTATIONS	1	1	0	1	1	10	9	9	7
INJURY, POISONING AND PROCEDURAL COMPLICATIONS	0	1	0	1	0	2	2	2	1
INVESTIGATIONS	0	1	0	0	1	4	1	5	3
METABOLISM AND NUTRITION DISORDERS	0	0	0	1	0	2	0	1	1
MUSCULOSKELETAL AND CONNECTIVE TISSUE DISORDERS	0	2	0	0	2	6	5	2	1
NEOPLASMS BENIGN, MALIGNANT AND UNSPECIFIED	0	0	0	0	0	1	0	0	0
NERVOUS SYSTEM DISORDERS	0	1	0	1	1	1	3	9	6
PSYCHIATRIC DISORDERS	0	0	0	0	0	1	1	3	2
RENAL AND URINARY DISORDERS	0	0	0	0	0	1	1	0	0
RESPIRATORY, THORACIC AND MEDIASTINAL DISORDERS	1	1	0	0	0	5	5	1	4
SKIN AND SUBCUTANEOUS TISSUE DISORDERS	0	0	0	1	1	4	4	3	2
SURGICAL AND MEDICAL PROCEDURES	0	0	0	0	0	1	0	1	0
VASCULAR DISORDERS	0	0	0	0	2	1	1	2	1

No statistical analyses for this end point

Primary: Number of Treatment-Emergent AEs by Severity

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End point title

Number of Treatment-Emergent AEs by Severity ^[6]

End point description

The intensity grades were defined as follows: mild=does not interfere with subject's usual function; moderate=interferes to some extent with subject's usual function; severe=interferes significantly with subject's usual function.

End point type

Primary

End point timeframe

Baseline up to Week 52

Notes
[6] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No statistical analysis was planned for this primary endpoint. Adverse events were reported in accordance with the sponsor reporting standards.

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: adverse events
Mild AEs	6	16	0	8	13	61	35	56	48
Moderate AEs	4	0	0	5	2	33	31	21	17
Severe AEs	1	0	0	4	0	13	8	4	3

No statistical analyses for this end point

Secondary: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 12 and Week 16

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End point title

Change From Baseline in Rectal Bleeding Mayo Subscore at Week 12 and Week 16

End point description

Mayo Score is an instrument designed to measure disease activity of ulcerative colitis. Mayo subscores for rectal bleeding range from 0 to 3, with higher scores indicating more severe disease.

End point type

Secondary

End point timeframe

Baseline, Week 12, Week 16

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: scores on a scale
arithmetic mean (standard deviation)
Change at Week 12 (n=0,0,0,0,0,22,25,17,19)	99999 ( 99999 )	99999 ( 99999 )	99999 ( 99999 )	99999 ( 99999 )	99999 ( 99999 )	-0.77 ( 0.869 )	-0.64 ( 1.114 )	-0.53 ( 0.874 )	-0.47 ( 0.905 )
Change at Week 16 (n=2,3,2,4,5,21,24,17,19)	-1.5 ( 0.707 )	-1 ( 1 )	0 ( 0 )	0 ( 0.816 )	-0.6 ( 1.14 )	-0.57 ( 0.978 )	-0.92 ( 1.1 )	-0.47 ( 0.874 )	-0.58 ( 0.838 )

No statistical analyses for this end point

Secondary: Number of Subjects With Laboratory Test Abnormalities

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End point title

Number of Subjects With Laboratory Test Abnormalities

End point description

The total number of subjects with laboratory test abnormalities with or without regard to baseline abnormality was assessed. Laboratory parameters included: hematology (hemoglobin, hematocrit, red blood cell [RBC] count, platelet count, white blood cell [WBC] count, total neutrophils, eosinophils, monocytes, basophils, lymphocytes, erythrocyte sedimentation rate); chemistry (blood urea nitrogen and creatinine, glucose, calcium, sodium, potassium, chloride, total bicarbonate, aspartate aminotransferase, alanine aminotransferase, total bilirubin, alkaline phosphatase, uric acid, albumin, total protein; urinalysis (pH, glucose, protein, blood, ketones, nitrites, leukocyte esterase, microscopy (only if urine dipstick was positive for blood, protein, nitrites or leukocyte esterase); other (follicle-stimulating hormone, human chorionic gonadotropin, stool microbiology, creatinine kinase, direct bilirubin, indirect bilirubin, gamma-glutamyl transferase, international normalized ratio.

End point type

Secondary

End point timeframe

Baseline up to Week 24

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: subjects
Normal/Abnormal Baseline(n=2,3,2,4,6,23,25,19,21)	2	2	1	4	4	14	20	15	17
Normal Baseline (n=2,3,2,4,6,23,25,19,21)	2	2	1	3	4	9	16	12	13
Abnormal Baseline (n=2,3,2,2,3,22,19,15,18)	1	1	1	1	2	7	4	4	6

No statistical analyses for this end point

Secondary: Number of Subjects With Potentially Clinically Significant Vital Signs Findings

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End point title

Number of Subjects With Potentially Clinically Significant Vital Signs Findings

End point description

Vital signs assessment included pulse rate, systolic blood pressure (SBP) and diastolic blood pressure (DBP). Criteria for vital sign values meeting potential clinical concern included: SBP <90 millimeters of mercury (mm Hg) and >=30 mm Hg increase/decrease from baseline, DBP <50 mm Hg and >=20 mm Hg increase/decrease from baseline, pulse rate <40 or >120 beats per minute (bpm),

End point type

Secondary

End point timeframe

Baseline up to Week 52

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: subjects
Supine SBP <90 mm Hg	0	0	0	0	2	0	0	0	0
Supine DBP <50 mm Hg	0	0	0	0	0	0	1	0	0
Supine Pulse Rate <40 bpm	0	0	0	0	0	0	0	0	0
Supine Pulse Rate >120 bpm	0	0	0	0	0	3	0	1	0
Supine SBP >=30 mm Hg Increase From Baseline	0	0	0	1	1	1	3	1	2
Supine DBP >=20 mm Hg Increase From Baseline	2	0	0	1	0	3	0	2	4
Supine SBP >=30 mm Hg Decrease From Baseline	0	0	0	1	0	1	1	1	1
Supine DBP >=20 mm Hg Decrease From Baseline	1	1	0	0	0	6	1	3	0

No statistical analyses for this end point

Secondary: Fold Change From Baseline in Fecal Calprotectin at Weeks 4, 8, 12, and 16

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End point title

Fold Change From Baseline in Fecal Calprotectin at Weeks 4, 8, 12, and 16

End point description

Fecal calprotectin, a very stable marker, is a 36kDa calcium and zinc binding protein which is neutrophil-derived. It represents 60% of cytosolic proteins in granulocytes and is a measurement of neutrophil migration to the gastrointestinal tract.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12 and 16

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: milligrams per kilogram (mg/kg)
geometric mean (geometric coefficient of variation)
Baseline (n=2,3,1,4,5,21,23,19,20)	690.7549 ( 17 )	1189.8169 ( 22 )	739.23 ( 99999 )	898.2758 ( 89 )	874.1363 ( 1462 )	576.5796 ( 237 )	476.1504 ( 222 )	513.2139 ( 208 )	821.1953 ( 220 )
Change at Week 4 (n=2,3,1,3,5,20,21,18,19)	0.6297 ( 50 )	1.868 ( 103 )	2.0264 ( 99999 )	2.0549 ( 112 )	1.094 ( 91 )	1.2196 ( 158 )	0.8179 ( 314 )	1.0541 ( 166 )	0.6722 ( 193 )
Change at Week 8 (n=1,2,1,3,2,18,20,17,20)	2.4504 ( 99999 )	2.4559 ( 51 )	2.6199 ( 99999 )	0.5744 ( 52 )	0.3282 ( 2 )	1.0704 ( 220 )	0.7462 ( 235 )	0.8598 ( 227 )	0.3479 ( 242 )
Change at Week 12 (n=0,0,0,0,0,20,21,17,18)	99999 ( 99999 )	99999 ( 99999 )	99999 ( 99999 )	99999 ( 99999 )	99999 ( 99999 )	0.9947 ( 171 )	0.8269 ( 310 )	0.8788 ( 293 )	0.5279 ( 171 )
Change at Week 16 (n=2,3,1,4,4,18,18,16,17)	2.1672 ( 49 )	0.7679 ( 32 )	1.1956 ( 99999 )	0.4459 ( 139 )	0.2474 ( 4517 )	0.6473 ( 277 )	0.8486 ( 149 )	0.5473 ( 309 )	0.5275 ( 243 )

Fecal Calprotectin (Week 4)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 4)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.53 ^[7]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

1.02

Confidence interval

level

80%

sides

2-sided

lower limit

0.73

upper limit

1.42

Notes
[7] - 1-sided p-value

Fecal Calprotectin (Week 8)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 8)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.91 ^[8]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

1.48

Confidence interval

level

80%

sides

2-sided

lower limit

1.02

upper limit

2.14

Notes
[8] - 1-sided p-value

Fecal Calprotectin (Week 12)

Statistical analysis description

Cohort 3 MM versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.81 ^[9]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

1.38

Confidence interval

level

80%

sides

2-sided

lower limit

0.86

upper limit

2.23

Notes
[9] - 1-sided p-value

Fecal Calprotectin (Week 12)

Statistical analysis description

Cohort 3 MP versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.44 ^[10]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

0.94

Confidence interval

level

80%

sides

2-sided

lower limit

0.59

upper limit

1.51

Notes
[10] - 1-sided p-value

Fecal Calprotectin (Week 12)

Statistical analysis description

Cohort 3 PM versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.67 ^[11]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

1.18

Confidence interval

level

80%

sides

2-sided

lower limit

0.72

upper limit

1.94

Notes
[11] - 1-sided p-value

Fecal Calprotectin (Week 16)

Statistical analysis description

Cohort 3 MM versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.62 ^[12]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

1.12

Confidence interval

level

80%

sides

2-sided

lower limit

0.68

upper limit

1.84

Notes
[12] - 1-sided p-value

Fecal Calprotectin (Week 16)

Statistical analysis description

Cohort 3 MP versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.45 ^[13]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

0.95

Confidence interval

level

80%

sides

2-sided

lower limit

0.58

upper limit

1.57

Notes
[13] - 1-sided p-value

Fecal Calprotectin (Week 16)

Statistical analysis description

Cohort 3 PM versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.3 ^[14]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

0.81

Confidence interval

level

80%

sides

2-sided

lower limit

0.49

upper limit

1.36

Notes
[14] - 1-sided p-value

Secondary: Fold Change From Baseline in C-Reactive Protein (CRP) at Weeks 4, 8, 12, and 16

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End point title

Fold Change From Baseline in C-Reactive Protein (CRP) at Weeks 4, 8, 12, and 16

End point description

CRP is an acute-phase protein which provides an objective criterion of inflammatory activity. CRP has a short half-life (19 hours) and therefore rises early after the onset of inflammation and rapidly decreases after resolution of the inflammation. It is induced by interleukin-6, TNF-alpha and other pro-inflammatory cytokines that are produced within the intestinal lamina propria.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12 and 16

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: milligrams per deciliter (mg/dL)
geometric mean (geometric coefficient of variation)
Baseline (n=2,3,2,4,6,22,25,19,20)	1.272 ( 45 )	1.527 ( 118 )	0.229 ( 34 )	0.578 ( 37 )	0.18 ( 74 )	0.613 ( 339 )	0.542 ( 236 )	0.397 ( 157 )	0.453 ( 297 )
Change at Week 4 (n=2,3,2,3,6,21,25,18,20)	0.289 ( 9 )	0.555 ( 82 )	1.937 ( 42 )	1.285 ( 60 )	1.446 ( 54 )	0.891 ( 147 )	0.764 ( 140 )	0.999 ( 103 )	0.588 ( 132 )
Change at Week 8 (n=1,3,1,3,5,21,25,19,20)	1.39 ( 99999 )	0.745 ( 49 )	2.331 ( 99999 )	1.172 ( 20 )	1.555 ( 31 )	1.189 ( 146 )	0.759 ( 207 )	1.2 ( 113 )	0.623 ( 150 )
Change at Week 12 (n=0,0,0,0,0,21,24,17,18)	99999 ( 99999 )	99999 ( 99999 )	99999 ( 99999 )	99999 ( 99999 )	99999 ( 99999 )	0.937 ( 211 )	0.71 ( 365 )	0.782 ( 149 )	0.373 ( 155 )
Change at Week 16 (n=2,3,2,4,5,20,24,17,18)	0.956 ( 164 )	0.481 ( 56 )	0.606 ( 248 )	0.869 ( 14 )	1.184 ( 54 )	0.776 ( 156 )	0.425 ( 178 )	0.612 ( 133 )	0.417 ( 197 )

Fold Change From Baseline in CRP (Week 4)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 4)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.79 ^[15]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

1.17

Confidence interval

level

80%

sides

2-sided

lower limit

0.91

upper limit

1.51

Notes
[15] - 1-sided p-value

Fold Change From Baseline in CRP (Week 8)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 8)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.8 ^[16]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

1.21

Confidence interval

level

80%

sides

2-sided

lower limit

0.9

upper limit

1.63

Notes
[16] - 1-sided p-value

Fold Change From Baseline in CRP (Week 12)

Statistical analysis description

Cohort 3 MM versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.99 ^[17]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

2.75

Confidence interval

level

80%

sides

2-sided

lower limit

1.63

upper limit

4.64

Notes
[17] - 1-sided p-value

Fold Change From Baseline in CRP (Week 12)

Statistical analysis description

Cohort 3 MP versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.96 ^[18]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

1.96

Confidence interval

level

80%

sides

2-sided

lower limit

1.19

upper limit

3.25

Notes
[18] - 1-sided p-value

Fold Change From Baseline in CRP (Week 12)

Statistical analysis description

Cohort 3 PM versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.95 ^[19]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

1.98

Confidence interval

level

80%

sides

2-sided

lower limit

1.15

upper limit

3.41

Notes
[19] - 1-sided p-value

Fold Change From Baseline in CRP (Week 16)

Statistical analysis description

Cohort 3 MM versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.99 ^[20]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

2.3

Confidence interval

level

80%

sides

2-sided

lower limit

1.52

upper limit

3.48

Notes
[20] - 1-sided p-value

Fold Change From Baseline in CRP (Week 16)

Statistical analysis description

Cohort 3 MP versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.62 ^[21]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

1.1

Confidence interval

level

80%

sides

2-sided

lower limit

0.74

upper limit

1.63

Notes
[21] - 1-sided p-value

Fold Change From Baseline in CRP (Week 16)

Statistical analysis description

Cohort 3 PM versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.89 ^[22]

Method

Mixed models analysis

Parameter type

Geometric Mean Ratio

Point estimate

1.51

Confidence interval

level

80%

sides

2-sided

lower limit

0.98

upper limit

2.32

Notes
[22] - 1-sided p-value

Secondary: Percentage of Subjects With Rectal Bleeding Mayo Subscore of Zero at Weeks 4, 8, 12, and 16

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End point title

Percentage of Subjects With Rectal Bleeding Mayo Subscore of Zero at Weeks 4, 8, 12, and 16

End point description

Mayo subscores for rectal bleeding range from 0 to 3 (0=no blood seen; 1=streaks of blood with stool less than half the time; 2=obvious blood with stool most of the time; 3=blood alone passes).

End point type

Secondary

End point timeframe

Week 4, 8, 12 and 16

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: percentage of subjects
number (not applicable)
Week 4	50	66.7	50	33.3	40	52.2	28	31.6	42.9
Week 8	50	66.7	50	33.3	40	47.8	36	31.6	47.6
Week 12	99999	99999	99999	99999	99999	63.6	40	52.9	47.4
Week 16	50	100	50	50	40	52.4	50	41.2	57.9

Rectal Bleeding Mayo Subscore of Zero (Week 4)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 4)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.34 ^[23]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

1.21

Confidence interval

level

80%

sides

2-sided

lower limit

0.66

upper limit

2.19

Notes
[23] - 1-sided p-value

Rectal Bleeding Mayo Subscore of Zero (Week 8)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 8)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.33 ^[24]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

1.23

Confidence interval

level

80%

sides

2-sided

lower limit

0.68

upper limit

2.23

Notes
[24] - 1-sided p-value

Rectal Bleeding Mayo Subscore of Zero (Week 12)

Statistical analysis description

Cohort 3 MM versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.1 ^[25]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

2.37

Confidence interval

level

80%

sides

2-sided

lower limit

0.99

upper limit

5.67

Notes
[25] - 1-sided p-value

Rectal Bleeding Mayo Subscore of Zero (Week 12)

Statistical analysis description

Cohort 3 MP versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.74 ^[26]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

0.66

Confidence interval

level

80%

sides

2-sided

lower limit

0.28

upper limit

1.55

Notes
[26] - 1-sided p-value

Rectal Bleeding Mayo Subscore of Zero (Week 12)

Statistical analysis description

Cohort 3 PM versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.48 ^[27]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

1.04

Confidence interval

level

80%

sides

2-sided

lower limit

0.41

upper limit

2.64

Notes
[27] - 1-sided p-value

Rectal Bleeding Mayo Subscore of Zero (Week 16)

Statistical analysis description

Cohort 3 MM versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.58 ^[28]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

0.88

Confidence interval

level

80%

sides

2-sided

lower limit

0.38

upper limit

2.03

Notes
[28] - 1-sided p-value

Rectal Bleeding Mayo Subscore of Zero (Week 16)

Statistical analysis description

Cohort 3 MP versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.68 ^[29]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

0.74

Confidence interval

level

80%

sides

2-sided

lower limit

0.33

upper limit

1.66

Notes
[29] - 1-sided p-value

Rectal Bleeding Mayo Subscore of Zero (Week 16)

Statistical analysis description

Cohort 3 PM versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.84 ^[30]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

0.51

Confidence interval

level

80%

sides

2-sided

lower limit

0.21

upper limit

1.23

Notes
[30] - 1-sided p-value

Secondary: Percentage of Subjects in Endoscopic Remission at Week 8

Top of page

End point title

Percentage of Subjects in Endoscopic Remission at Week 8

End point description

Endoscopic remission is defined as modified Baron Endoscopic Score equal to 0.

End point type

Secondary

End point timeframe

Week 8

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: percentage of subjects
number (not applicable)	0	0	0	0	16.7	4.3	4	0	9.5

Endoscopic Remission (Week 8)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.57 ^[31]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

0.83

Confidence interval

level

80%

sides

2-sided

lower limit

0.22

upper limit

3.07

Notes
[31] - 1-sided p-value

Secondary: Percentage of Subjects in Clinical Remission at Week 8

Top of page

End point title

Percentage of Subjects in Clinical Remission at Week 8

End point description

Clinical remission is defined as a total Mayo score of 2 points or lower, with no individual subscores exceeding 1 point.

End point type

Secondary

End point timeframe

Week 8

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: percentage of subjects
number (not applicable)	0	0	0	0	0	0	8	0	19

Clinical Remission (Week 8)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.85 ^[32]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

0.39

Confidence interval

level

80%

sides

2-sided

lower limit

0.12

upper limit

1.23

Notes
[32] - 1-sided p-value

Secondary: Percentage of Subjects With Decrease From Baseline of at Least 1 Point in Rectal Bleeding Mayo Subscore at Weeks 4, 8, 12, and 16

Top of page

End point title

Percentage of Subjects With Decrease From Baseline of at Least 1 Point in Rectal Bleeding Mayo Subscore at Weeks 4, 8, 12, and 16

End point description

Mayo subscores for rectal bleeding range from 0 to 3 (0=no blood seen; 1=streaks of blood with stool less than half the time; 2=obvious blood with stool most of the time; 3=blood alone passes). A decrease from baseline score indicates improvement.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12 and 16

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: percentage of subjects
number (not applicable)
Week 4	100	66.7	0	0	40	43.5	48	21.1	38.1
Week 8	100	33.3	0	0	40	34.8	48	31.6	47.6
Week 12	99999	99999	99999	99999	99999	54.5	60	41.2	52.6
Week 16	100	66.7	0	25	60	47.6	58.3	35.3	47.4

Rectal Bleeding Mayo Subscore (Week 4)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 4)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.05 ^[33]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

2.27

Confidence interval

level

80%

sides

2-sided

lower limit

1.21

upper limit

4.24

Notes
[33] - 1-sided p-value

Rectal Bleeding Mayo Subscore (Week 8)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 8)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.43 ^[34]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

1.09

Confidence interval

level

80%

sides

2-sided

lower limit

0.61

upper limit

1.95

Notes
[34] - 1-sided p-value

Rectal Bleeding Mayo Subscore (Week 12)

Statistical analysis description

Cohort 3 MM versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.44 ^[35]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

1.11

Confidence interval

level

80%

sides

2-sided

lower limit

0.45

upper limit

2.76

Notes
[35] - 1-sided p-value

Rectal Bleeding Mayo Subscore (Week 12)

Statistical analysis description

Cohort 3 MP versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.38 ^[36]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

1.23

Confidence interval

level

80%

sides

2-sided

lower limit

0.5

upper limit

3.04

Notes
[36] - 1-sided p-value

Rectal Bleeding Mayo Subscore (Week 12)

Statistical analysis description

Cohort 3 PM versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.89 ^[37]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

0.38

Confidence interval

level

80%

sides

2-sided

lower limit

0.14

upper limit

1.04

Notes
[37] - 1-sided p-value

Rectal Bleeding Mayo Subscore (Week 16)

Statistical analysis description

Cohort 3 MM versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.47 ^[38]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

1.05

Confidence interval

level

80%

sides

2-sided

lower limit

0.45

upper limit

2.42

Notes
[38] - 1-sided p-value

Rectal Bleeding Mayo Subscore (Week 16)

Statistical analysis description

Cohort 3 MP versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.24 ^[39]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

1.58

Confidence interval

level

80%

sides

2-sided

lower limit

0.7

upper limit

3.57

Notes
[39] - 1-sided p-value

Rectal Bleeding Mayo Subscore (Week 16)

Statistical analysis description

Cohort 3 PM versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.81 ^[40]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

0.53

Confidence interval

level

80%

sides

2-sided

lower limit

0.22

upper limit

1.32

Notes
[40] - 1-sided p-value

Secondary: Percentage of Subjects With Endoscopic Response at Week 8

Top of page

End point title

Percentage of Subjects With Endoscopic Response at Week 8

End point description

Endoscopic response is defined as a decrease in modified Baron endoscopic score from baseline of at least 2 points.

End point type

Secondary

End point timeframe

Week 8

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: percentage of subjects
number (not applicable)	0	0	0	0	0	8.7	8	10.5	19

Endoscopic Response (Week 8)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.84 ^[41]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

0.49

Confidence interval

level

80%

sides

2-sided

lower limit

0.2

upper limit

1.24

Notes
[41] - 1-sided p-value

Secondary: Percentage of Subjects in Clinical Response at Week 8

Top of page

End point title

Percentage of Subjects in Clinical Response at Week 8

End point description

Clinical response is defined as a decrease in total Mayo score from baseline of at least 3 points and at least 30 percent (%), with an accompanying decrease in the subscore for rectal bleeding of at least 1 point or an absolute subscore for rectal bleeding of 0 or 1.

End point type

Secondary

End point timeframe

Week 8

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: percentage of subjects
number (not applicable)	50	33.3	0	0	0	4.3	24	15.8	42.9

Clinical Response (Week 8)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.96 ^[42]

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

0.3

Confidence interval

level

80%

sides

2-sided

lower limit

0.12

upper limit

0.73

Notes
[42] - 1-sided p-value

Secondary: Change From Baseline in Total Mayo Scores at Week 8

Top of page

End point title

Change From Baseline in Total Mayo Scores at Week 8

End point description

Mayo Score is an instrument designed to measure disease activity of ulcerative colitis, with total score ranging from 0 to 12. It consists of 4 subscores (stool frequency, rectal bleeding, findings of flexible proctosigmoidoscopy, and physician global assessment [PGA]), each graded from 0 to 3, with higher scores indicating more severe disease.

End point type

Secondary

End point timeframe

Baseline, Week 8

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: scores on a scale
arithmetic mean (standard deviation)
Baseline (n=2,3,2,4,6,23,25,19,21)	10 ( 2.828 )	7.33 ( 1.155 )	8.5 ( 0.707 )	7.75 ( 0.957 )	7.5 ( 2.258 )	8.74 ( 1.839 )	8.48 ( 2.143 )	8.47 ( 1.806 )	8.14 ( 2.2 )
Change at Week 8 (n=2,3,2,3,5,23,25,19,21)	-2 ( 1.414 )	0 ( 2.646 )	1 ( 1.414 )	0.33 ( 0.577 )	-0.6 ( 0.894 )	-0.78 ( 2.066 )	-1.2 ( 2.872 )	-0.89 ( 2.132 )	-2 ( 2.683 )

Total Mayo Scores (Week 8)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.87 ^[43]

Method

ANCOVA

Parameter type

Mean difference (final values)

Point estimate

0.58

Confidence interval

level

80%

sides

2-sided

lower limit

-0.08

upper limit

1.24

Variability estimate

Standard error of the mean

Dispersion value

0.513

Notes
[43] - 1-sided p-value

Secondary: Change From Baseline in Partial Mayo Scores at Weeks 4, 8, 12, and 16

Top of page

End point title

Change From Baseline in Partial Mayo Scores at Weeks 4, 8, 12, and 16

End point description

A Partial Mayo Score (Mayo score without endoscopy) ranges from 0 (normal or inactive disease) to 9 (severe disease) and calculated as the sum of 3 subscores (stool frequency, rectal bleeding and PGA) with each ranging from 0 to 3 (0=normal, 1=mild, 2=moderate, 3=severe). Higher scores indicate more severe disease.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12 and 16

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: scores on a scale
arithmetic mean (standard deviation)
Baseline (n=2,3,2,4,6,23,25,19,21)	7.5 ( 2.121 )	5.33 ( 0.577 )	6 ( 1.414 )	5.75 ( 0.5 )	5.5 ( 1.378 )	6.22 ( 1.347 )	6.08 ( 1.605 )	6 ( 1.202 )	5.71 ( 1.736 )
Change at Week 4 (n=2,3,2,3,5,23,25,19,21)	-3 ( 0 )	-2 ( 1.732 )	-0.5 ( 0.707 )	0.67 ( 1.528 )	-0.4 ( 1.14 )	-1 ( 1.414 )	-0.96 ( 1.791 )	-1.16 ( 1.675 )	-1.24 ( 1.895 )
Change at Week 8 (n=2,3,2,3,5,23,25,19,21)	-2 ( 1.414 )	-1 ( 1.732 )	0.5 ( 0.707 )	0.33 ( 0.577 )	-0.8 ( 1.095 )	-0.91 ( 1.756 )	-1.12 ( 2.472 )	-0.79 ( 1.653 )	-1.62 ( 2.037 )
Change at Week 12 (n=0,0,0,0,0,22,25,17,19)	99999 ( 99999 )	99999 ( 99999 )	99999 ( 99999 )	99999 ( 99999 )	99999 ( 99999 )	-1.91 ( 1.974 )	-1.52 ( 2.312 )	-2.06 ( 2.135 )	-2.11 ( 2.622 )
Change at Week 16 (2,3,2,4,5,21,24,17,19)	-1.5 ( 0.707 )	-3 ( 3 )	-1 ( 2.828 )	-1.25 ( 3.403 )	-2.2 ( 1.924 )	-1.86 ( 2.061 )	-2.54 ( 2.621 )	-2.41 ( 2.181 )	-2.63 ( 2.033 )

Partial Mayo Scores (Week 4)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 4)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.8 ^[44]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

0.29

Confidence interval

level

80%

sides

2-sided

lower limit

-0.15

upper limit

0.74

Variability estimate

Standard error of the mean

Dispersion value

0.345

Notes
[44] - 1-sided p-value

Partial Mayo Scores (Week 8)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 8)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.77 ^[45]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

0.3

Confidence interval

level

80%

sides

2-sided

lower limit

-0.23

upper limit

0.84

Variability estimate

Standard error of the mean

Dispersion value

0.418

Notes
[45] - 1-sided p-value

Partial Mayo Scores (Week 12)

Statistical analysis description

Cohort 3 MM versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.74 ^[46]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

0.44

Confidence interval

level

80%

sides

2-sided

lower limit

-0.42

upper limit

1.3

Variability estimate

Standard error of the mean

Dispersion value

0.666

Notes
[46] - 1-sided p-value

Partial Mayo Scores (Week 12)

Statistical analysis description

Cohort 3 MP versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.88 ^[47]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

0.76

Confidence interval

level

80%

sides

2-sided

lower limit

-0.08

upper limit

1.59

Variability estimate

Standard error of the mean

Dispersion value

0.645

Notes
[47] - 1-sided p-value

Partial Mayo Scores (Week 12)

Statistical analysis description

Cohort 3 PM versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.72 ^[48]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

0.42

Confidence interval

level

80%

sides

2-sided

lower limit

-0.5

upper limit

1.33

Variability estimate

Standard error of the mean

Dispersion value

0.705

Notes
[48] - 1-sided p-value

Partial Mayo Scores (Week 16)

Statistical analysis description

Cohort 3 MM versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.97 ^[49]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

1.21

Confidence interval

level

80%

sides

2-sided

lower limit

0.37

upper limit

2.06

Variability estimate

Standard error of the mean

Dispersion value

0.656

Notes
[49] - 1-sided p-value

Partial Mayo Scores (Week 16)

Statistical analysis description

Cohort 3 MP versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.75 ^[50]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

0.43

Confidence interval

level

80%

sides

2-sided

lower limit

-0.39

upper limit

1.25

Variability estimate

Standard error of the mean

Dispersion value

0.635

Notes
[50] - 1-sided p-value

Partial Mayo Scores (Week 16)

Statistical analysis description

Cohort 3 PM versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.85 ^[51]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

0.73

Confidence interval

level

80%

sides

2-sided

lower limit

-0.16

upper limit

1.63

Variability estimate

Standard error of the mean

Dispersion value

0.692

Notes
[51] - 1-sided p-value

Secondary: Change From Baseline in Biopsy Histology Scores at Week 8

Top of page

End point title

Change From Baseline in Biopsy Histology Scores at Week 8

End point description

A 15 to 25 centimeter (cm) biopsy sample of inflamed mucosal tissue was taken from the worst affected area and scored using the Riley Index. The Riley Index is a histologic scoring system for the assessment of the activity and severity of ulcerative colitis; it consists of 6 histologic features (acute inflammatory cell infiltrate, crypt abscesses, mucin depletion, surface epithelial integrity, chronic inflammatory cell infiltrate, and crypt architectural irregularities), all scored on a 4-point scale (higher scores indicate more severe disease).

End point type

Secondary

End point timeframe

Baseline and Week 8

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: scores on a scale
arithmetic mean (standard deviation)
Baseline(n=2,3,2,4,5,22,24,18,21)	9 ( 1.41 )	9.7 ( 3.79 )	11 ( 2.83 )	7 ( 3.92 )	10.8 ( 3.77 )	9.4 ( 4.37 )	10.4 ( 4.24 )	9.9 ( 4.17 )	9.7 ( 5.14 )
Change at Week 4 (n=2,3,1,3,5,21,24,15,20)	-3.5 ( 4.95 )	1.3 ( 1.15 )	-3 ( 99999 )	-1 ( 1.73 )	-0.4 ( 2.07 )	1.4 ( 4.63 )	-1 ( 3.76 )	0.9 ( 4.09 )	-1.5 ( 4.8 )

Biopsy Histology Scores (Week 8)

Statistical analysis description

Pooled MultiStem versus Pooled Placebo

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.75 ^[52]

Method

ANCOVA

Parameter type

Mean difference (final values)

Point estimate

0.52

Confidence interval

level

80%

sides

2-sided

lower limit

-0.46

upper limit

1.49

Variability estimate

Standard error of the mean

Dispersion value

0.753

Notes
[52] - 1-sided p-value

Secondary: Change From Baseline in Patient-Reported Rectal Bleeding up to Week 16

Top of page

End point title

Change From Baseline in Patient-Reported Rectal Bleeding up to Week 16

End point description

Patient-reported diary data assessed the number of bowel movements (BM) per day when not having a flare and the presence of blood in the stools (rectal bleeding [RB]), if any.

End point type

Secondary

End point timeframe

Baseline and Week 16

End point values	Cohort 1: Placebo, MultiStem 300	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300, Placebo	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Number of subjects analysed	2	3	2	4	6	23	25	19	21
Units: scores on a scale
arithmetic mean (standard deviation)
BM: Baseline(n=2,3,2,4,6,23,25,19,21)	12.5 ( 4.95 )	8 ( 2.784 )	6.75 ( 2.475 )	11.75 ( 10.506 )	8.42 ( 3.089 )	7.5 ( 3.49 )	7.78 ( 3.781 )	7.5 ( 3.571 )	6.88 ( 4.886 )
BM: Change at Week 1 (n=2,3,2,4,5,23,25,19,21)	-1.6 ( 2.263 )	-1.89 ( 1.786 )	4.15 ( 2.845 )	-0.6 ( 2.177 )	0.4 ( 1.3 )	-0.42 ( 1.509 )	-0.59 ( 1.657 )	-0.56 ( 1.215 )	-0.81 ( 3.421 )
BM: Change at Week 4 (n=2,2,2,3,5,23,23,16,19	-1.43 ( 3.435 )	-1.89 ( 5.101 )	2.36 ( 0.505 )	0.17 ( 2.038 )	1.31 ( 1.432 )	-0.81 ( 2.088 )	0.28 ( 3.956 )	-0.63 ( 1.856 )	-1.08 ( 2.575 )
BM: Change at Week 8 (n=2,3,2,3,6,23,23,16,19)	-1.67 ( 3.771 )	-0.86 ( 3.517 )	3.18 ( 0.758 )	0.17 ( 2.754 )	0.41 ( 2.131 )	-0.89 ( 2.864 )	-1.15 ( 2.833 )	0.18 ( 2.531 )	-1.41 ( 3.208 )
BM: Change at Week 12 (n=1,1,1,2,2,21,25,16,18)	0.14 ( 99999 )	1.43 ( 99999 )	1.57 ( 99999 )	-1.68 ( 2.374 )	0 ( 0 )	-0.67 ( 3.152 )	-0.59 ( 4.428 )	-1.77 ( 2.301 )	-2.02 ( 3.502 )
BM: Change at Week 16 (n=1,3,2,3,4,22,24,16,18)	0 ( 99999 )	-2.51 ( 3.716 )	1.72 ( 0.596 )	-0.12 ( 2.324 )	-2.45 ( 2.208 )	-1.81 ( 3.278 )	-2.17 ( 3.523 )	-1.68 ( 2.899 )	-1.86 ( 3.616 )
RB: Baseline (n=2,3,2,4,6,23,25,19,21)	1.25 ( 1.061 )	1.17 ( 1.258 )	1 ( 1.414 )	1 ( 0.816 )	1.33 ( 0.816 )	1.2 ( 0.974 )	1.46 ( 0.978 )	0.97 ( 0.95 )	1.05 ( 0.82 )
RB: Change at Week 1 (n=2,3,2,4,5,23,25,19,21)	-0.32 ( 0.253 )	-1 ( 1.167 )	-0.33 ( 0.471 )	-0.12 ( 0.158 )	-0.3 ( 0.415 )	-0.36 ( 0.697 )	-0.24 ( 0.805 )	-0.05 ( 0.574 )	-0.1 ( 0.408 )
RB: Change at Week 4 (n=2,2,2,3,5,23,23,16,19)	-0.75 ( 0.354 )	-0.29 ( 1.01 )	-0.25 ( 0.354 )	0.14 ( 0.378 )	-0.71 ( 0.99 )	-0.34 ( 0.863 )	-0.47 ( 0.84 )	-0.27 ( 0.738 )	-0.26 ( 0.604 )
RB: Change at Week 8 (n=2,3,2,3,6,22,23,13,20)	-0.75 ( 0.354 )	-0.5 ( 0.5 )	0 ( 0 )	0.33 ( 0.577 )	-0.47 ( 1.035 )	-0.4 ( 0.709 )	-0.51 ( 0.805 )	-0.19 ( 0.997 )	-0.51 ( 0.967 )
RB: Change at Week 12 (n=1,1,1,2,2,21,25,16,18)	-0.57 ( 99999 )	0 ( 99999 )	0 ( 99999 )	0 ( 0 )	0 ( 0 )	-0.6 ( 0.792 )	-0.49 ( 1.084 )	-0.48 ( 0.829 )	-0.39 ( 0.783 )
RB: Change at Week 16 (n=1,3,2,3,4,22,24,16,18)	-1 ( 99999 )	-1.17 ( 1.258 )	-0.1 ( 0.141 )	0.33 ( 0.577 )	-1.14 ( 0.865 )	-0.41 ( 0.936 )	-0.86 ( 1.106 )	-0.41 ( 0.838 )	-0.39 ( 0.874 )

Patient-Reported Rectal Bleeding up to Week 16

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 4)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.51 ^[53]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

Confidence interval

level

80%

sides

2-sided

lower limit

-0.18

upper limit

0.19

Variability estimate

Standard error of the mean

Dispersion value

0.144

Notes
[53] - 1-sided p-value

Patient-Reported Rectal Bleeding up to Week 16

Statistical analysis description

Pooled MultiStem versus Pooled Placebo (Week 8)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.62 ^[54]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

0.05

Confidence interval

level

80%

sides

2-sided

lower limit

-0.16

upper limit

0.25

Variability estimate

Standard error of the mean

Dispersion value

0.158

Notes
[54] - 1-sided p-value

Patient-Reported Rectal Bleeding up to Week 16

Statistical analysis description

Cohort 3 MM versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.29 ^[55]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

-0.12

Confidence interval

level

80%

sides

2-sided

lower limit

-0.39

upper limit

0.16

Variability estimate

Standard error of the mean

Dispersion value

0.213

Notes
[55] - 1-sided p-value

Patient-Reported Rectal Bleeding up to Week 16

Statistical analysis description

Cohort 3 MP versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.74 ^[56]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

0.13

Confidence interval

level

80%

sides

2-sided

lower limit

-0.14

upper limit

0.4

Variability estimate

Standard error of the mean

Dispersion value

0.209

Notes
[56] - 1-sided p-value

Patient-Reported Rectal Bleeding up to Week 16

Statistical analysis description

Cohort 3 PM versus Cohort 3 PP (Week 12)

Comparison groups

Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.36 ^[57]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

-0.08

Confidence interval

level

80%

sides

2-sided

lower limit

-0.37

upper limit

0.21

Variability estimate

Standard error of the mean

Dispersion value

0.226

Notes
[57] - 1-sided p-value

Patient-Reported Rectal Bleeding up to Week 16

Statistical analysis description

Cohort 3 MM versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: MultiStem 750, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.56 ^[58]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

0.04

Confidence interval

level

80%

sides

2-sided

lower limit

-0.26

upper limit

0.34

Variability estimate

Standard error of the mean

Dispersion value

0.234

Notes
[58] - 1-sided p-value

Patient-Reported Rectal Bleeding up to Week 16

Statistical analysis description

Cohort 3 MP versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: MultiStem 750, Placebo v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.18 ^[59]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

-0.21

Confidence interval

level

80%

sides

2-sided

lower limit

-0.51

upper limit

0.09

Variability estimate

Standard error of the mean

Dispersion value

0.231

Notes
[59] - 1-sided p-value

Patient-Reported Rectal Bleeding up to Week 16

Statistical analysis description

Cohort 3 PM versus Cohort 3 PP (Week 16)

Comparison groups

Cohort 3: Placebo, MultiStem 750 v Cohort 3: Placebo, Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.31 ^[60]

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

-0.12

Confidence interval

level

80%

sides

2-sided

lower limit

-0.45

upper limit

0.2

Variability estimate

Standard error of the mean

Dispersion value

0.25

Notes
[60] - 1-sided p-value

Adverse events

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Timeframe for reporting adverse events

Baseline up to 28 days after last study drug administration.

Adverse event reporting additional description

The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 subject and as non-serious in another, or 1 subject may have experienced both an AE and SAE during the study. All treated subjects were included in the analysis.

Assessment type

Non-systematic

Dictionary name

MedDRA

Dictionary version

17.1

Reporting group title

Cohort 1: Placebo, MultiStem 300

Reporting group description

Subjects received a single dose (Day 1) or 3 doses (Day 1, Week 1, Week 2) of placebo infusion, followed by a single dose of MultiStem 300 Million Cells infusion at Week 8.

Reporting group title

Cohort 1: MultiStem 300, Placebo

Reporting group description

Subjects received a single dose of MultiStem 300 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Reporting group title

Cohort 2: Placebo, MultiStem 750

Reporting group description

Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

Reporting group title

Cohort 1: MultiStem 300 (x3), Placebo

Reporting group description

Subjects received up to 3 doses of MultiStem 300 Million Cells infusion (Day 1, Week, Week 2), followed by a single dose of placebo infusion at Week 8.

Reporting group title

Cohort 3: MultiStem 750, MultiStem 750

Reporting group description

Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

Reporting group title

Cohort 2: MultiStem 750, Placebo

Reporting group description

Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Reporting group title

Cohort 3: MultiStem 750, Placebo

Reporting group description

Subjects received a single dose of MultiStem 750 Million Cells infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Reporting group title

Cohort 3: Placebo, MultiStem 750

Reporting group description

Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of MultiStem 750 Million Cells infusion at Week 8.

Reporting group title

Cohort 3: Placebo, Placebo

Reporting group description

Subjects received a single dose of placebo infusion on Day 1, followed by a single dose of placebo infusion at Week 8.

Serious adverse events	Cohort 1: Placebo, MultiStem 300	Cohort 1: MultiStem 300, Placebo	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Total subjects affected by serious adverse events
subjects affected / exposed	1 / 2 (50.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	3 / 4 (75.00%)	7 / 23 (30.43%)	0 / 6 (0.00%)	4 / 25 (16.00%)	5 / 19 (26.32%)	4 / 21 (19.05%)
number of deaths (all causes)	0	0	0	0	0	0	0	0	0
number of deaths resulting from adverse events	0	0	0	0	0	0	0	0	0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Adenocarcinoma of colon
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Vascular disorders
Diastolic hypertension
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Cardiac disorders
Ventricular tachycardia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	1 / 21 (4.76%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Nervous system disorders
Epilepsy
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Superior sagittal sinus thrombosis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	1 / 21 (4.76%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Blood and lymphatic system disorders
Anaemia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Pancytopenia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	1 / 1	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Immune system disorders
Hypersensitivity
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	1 / 4 (25.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	1 / 1	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Gastrointestinal disorders
Abdominal pain
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Colitis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	1 / 4 (25.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	1 / 25 (4.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Colitis ulcerative
subjects affected / exposed	1 / 2 (50.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	1 / 4 (25.00%)	5 / 23 (21.74%)	0 / 6 (0.00%)	1 / 25 (4.00%)	2 / 19 (10.53%)	3 / 21 (14.29%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0	0 / 1	0 / 7	0 / 0	0 / 1	0 / 2	0 / 3
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Nausea
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Vomiting
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Infections and infestations
Abdominal abscess
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Clostridium difficile colitis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	1 / 21 (4.76%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Clostridium difficile infection
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	1 / 25 (4.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Gastroenteritis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	1 / 25 (4.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Pelvic sepsis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Perirectal abscess
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Pneumonia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 5%

Non-serious adverse events	Cohort 1: Placebo, MultiStem 300	Cohort 1: MultiStem 300, Placebo	Cohort 2: Placebo, MultiStem 750	Cohort 1: MultiStem 300 (x3), Placebo	Cohort 3: MultiStem 750, MultiStem 750	Cohort 2: MultiStem 750, Placebo	Cohort 3: MultiStem 750, Placebo	Cohort 3: Placebo, MultiStem 750	Cohort 3: Placebo, Placebo
Total subjects affected by non serious adverse events
subjects affected / exposed	2 / 2 (100.00%)	0 / 2 (0.00%)	3 / 3 (100.00%)	3 / 4 (75.00%)	18 / 23 (78.26%)	5 / 6 (83.33%)	16 / 25 (64.00%)	15 / 19 (78.95%)	18 / 21 (85.71%)
Vascular disorders
Flushing
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	1 / 6 (16.67%)	0 / 25 (0.00%)	1 / 19 (5.26%)	1 / 21 (4.76%)
occurrences all number	0	0	0	0	0	1	0	2	1
Hot flush
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	1 / 6 (16.67%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Hypertension
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Surgical and medical procedures
Ileostomy
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
General disorders and administration site conditions
Asthenia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	1 / 4 (25.00%)	3 / 23 (13.04%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	1	4	0	0	0	0
Chest discomfort
subjects affected / exposed	1 / 2 (50.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	1 / 6 (16.67%)	0 / 25 (0.00%)	2 / 19 (10.53%)	1 / 21 (4.76%)
occurrences all number	1	0	0	0	0	1	0	2	1
Chest pain
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	1 / 25 (4.00%)	0 / 19 (0.00%)	1 / 21 (4.76%)
occurrences all number	0	0	1	0	0	0	1	0	2
Chills
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	2 / 4 (50.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	1	3	1	0	0	0	0
Fatigue
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	2 / 23 (8.70%)	0 / 6 (0.00%)	3 / 25 (12.00%)	1 / 19 (5.26%)	2 / 21 (9.52%)
occurrences all number	0	0	0	0	3	0	3	1	2
Influenza like illness
subjects affected / exposed	1 / 2 (50.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	1 / 25 (4.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	0	0	1	0	3	0	0
Oedema peripheral
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	2 / 21 (9.52%)
occurrences all number	0	0	0	0	0	0	0	1	3
Pain
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	1	0	1	0	0	1	0
Pyrexia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	3 / 4 (75.00%)	2 / 23 (8.70%)	0 / 6 (0.00%)	2 / 25 (8.00%)	1 / 19 (5.26%)	2 / 21 (9.52%)
occurrences all number	0	0	0	7	2	0	2	1	2
Respiratory, thoracic and mediastinal disorders
Cough
subjects affected / exposed	1 / 2 (50.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	3 / 23 (13.04%)	0 / 6 (0.00%)	3 / 25 (12.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	1	0	3	0	3	0	0
Dyspnoea
subjects affected / exposed	1 / 2 (50.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	1 / 21 (4.76%)
occurrences all number	1	0	0	0	1	0	0	0	1
Epistaxis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	1 / 21 (4.76%)
occurrences all number	0	0	0	0	0	0	0	1	2
Nasal congestion
subjects affected / exposed	1 / 2 (50.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	1 / 25 (4.00%)	0 / 19 (0.00%)	1 / 21 (4.76%)
occurrences all number	1	0	0	0	0	0	1	0	1
Oropharyngeal pain
subjects affected / exposed	1 / 2 (50.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	3 / 25 (12.00%)	0 / 19 (0.00%)	1 / 21 (4.76%)
occurrences all number	1	0	0	0	1	0	4	0	1
Psychiatric disorders
Anxiety
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	1 / 25 (4.00%)	3 / 19 (15.79%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	2	3	0
Confusional state
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Depression
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	2 / 21 (9.52%)
occurrences all number	0	0	0	0	0	0	0	0	2
Insomnia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	1 / 25 (4.00%)	1 / 19 (5.26%)	1 / 21 (4.76%)
occurrences all number	0	0	0	0	0	0	1	1	1
Investigations
Antibody test
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Bacterial test
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	1 / 6 (16.67%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Blood albumin decreased
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Blood bilirubin increased
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Blood glucose increased
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Blood urine present
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	1	0	0	0	0	0	0
C-reactive protein increased
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	5	0	0	2	0
Cold agglutinins
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Heart rate increased
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	1 / 6 (16.67%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Prostatic specific antigen increased
subjects affected / exposed ^[1]	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 16 (0.00%)	0 / 1 (0.00%)	0 / 13 (0.00%)	0 / 16 (0.00%)	1 / 14 (7.14%)
occurrences all number	0	0	0	0	0	0	0	0	1
Weight decreased
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	2 / 23 (8.70%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	1 / 21 (4.76%)
occurrences all number	0	0	0	0	2	0	0	2	1
Injury, poisoning and procedural complications
Contusion
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	1	0	0	0	0	1	0
Fall
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	1	0	1	0	0	0	0
Incision site pain
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	1 / 4 (25.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	1	0	0	0	0	0
Laceration
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	1 / 25 (4.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	1	1	0
Skeletal injury
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	1	0	0	0	0	0	0
Cardiac disorders
Tachycardia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	1	0	0	1	0
Nervous system disorders
Amnesia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Dizziness
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	0 / 23 (0.00%)	1 / 6 (16.67%)	1 / 25 (4.00%)	2 / 19 (10.53%)	0 / 21 (0.00%)
occurrences all number	0	0	1	0	0	1	1	2	0
Epilepsy
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	2	0
Headache
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	3 / 25 (12.00%)	7 / 19 (36.84%)	6 / 21 (28.57%)
occurrences all number	0	0	0	0	2	0	3	7	7
Lethargy
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	1 / 4 (25.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	1	0	0	0	0	0
Paraesthesia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	2 / 19 (10.53%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	3	0
Blood and lymphatic system disorders
Anaemia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	4 / 23 (17.39%)	0 / 6 (0.00%)	0 / 25 (0.00%)	2 / 19 (10.53%)	3 / 21 (14.29%)
occurrences all number	0	0	0	0	6	0	0	2	4
Lymphadenopathy
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Ear and labyrinth disorders
Vertigo
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	1 / 21 (4.76%)
occurrences all number	0	0	0	0	1	0	0	1	1
Gastrointestinal disorders
Abdominal pain
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	3 / 23 (13.04%)	0 / 6 (0.00%)	2 / 25 (8.00%)	1 / 19 (5.26%)	2 / 21 (9.52%)
occurrences all number	0	0	0	0	4	0	3	2	2
Abdominal pain upper
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	1 / 25 (4.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	1	1	0
Abdominal tenderness
subjects affected / exposed	1 / 2 (50.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	1 / 6 (16.67%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	0	0	0	1	0	0	0
Colitis ulcerative
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	5 / 23 (21.74%)	0 / 6 (0.00%)	2 / 25 (8.00%)	3 / 19 (15.79%)	5 / 21 (23.81%)
occurrences all number	0	0	0	0	8	0	3	4	5
Defaecation urgency
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	1 / 6 (16.67%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Diarrhoea
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	3 / 23 (13.04%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	3	0	0	0	0
Dyspepsia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	1 / 21 (4.76%)
occurrences all number	0	0	0	0	0	0	0	1	1
Gastritis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Gastrooesophageal reflux disease
subjects affected / exposed	1 / 2 (50.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	0	0	0	0	0	0	0
Haematochezia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	1 / 25 (4.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	1	0	0	0	0	0	0
Nausea
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	3 / 23 (13.04%)	2 / 6 (33.33%)	1 / 25 (4.00%)	1 / 19 (5.26%)	2 / 21 (9.52%)
occurrences all number	0	0	0	0	4	2	3	1	2
Painful defaecation
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Perianal erythema
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	1 / 4 (25.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	1	0	0	0	0	0
Rectal haemorrhage
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Tongue disorder
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	1	0	0	0	0	0	0
Vomiting
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	1 / 25 (4.00%)	2 / 19 (10.53%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	1	3	0
Skin and subcutaneous tissue disorders
Dermatitis contact
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	1 / 4 (25.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	1	0	0	0	0	0
Erythema
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Night sweats
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	1 / 6 (16.67%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Pruritus
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	2	0
Rash
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	2 / 25 (8.00%)	1 / 19 (5.26%)	1 / 21 (4.76%)
occurrences all number	0	0	0	0	1	0	2	1	1
Rash macular
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	1 / 4 (25.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	1	0	0	0	0	0
Skin lesion
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Urticaria
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	2	0
Endocrine disorders
Cushingoid
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	1 / 25 (4.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	1	1	0
Musculoskeletal and connective tissue disorders
Arthralgia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	2 / 25 (8.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	1	0	2	0	3	1	0
Back pain
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	3 / 23 (13.04%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	1 / 21 (4.76%)
occurrences all number	0	0	0	0	4	0	0	0	1
Costochondritis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	1	0	0	0	0	0	0
Muscle spasms
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	1 / 6 (16.67%)	1 / 25 (4.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	1	1	0	0
Muscle tightness
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	1 / 6 (16.67%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Myalgia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	3 / 23 (13.04%)	0 / 6 (0.00%)	1 / 25 (4.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	3	0	1	0	0
Osteoarthritis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Pain in extremity
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	1 / 25 (4.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	1	0	0	0	1	1	0
Pain in jaw
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	1 / 6 (16.67%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	1	0	0	0
Infections and infestations
Bacteriuria
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Bronchitis
subjects affected / exposed	1 / 2 (50.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	0	0	0	0	0	0	0
Cellulitis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Gastroenteritis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Influenza
subjects affected / exposed	1 / 2 (50.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	1	0	0	0	1	0	0	2	0
Nasopharyngitis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	2 / 23 (8.70%)	0 / 6 (0.00%)	1 / 25 (4.00%)	5 / 19 (26.32%)	4 / 21 (19.05%)
occurrences all number	0	0	0	0	2	0	1	7	4
Pharyngitis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	1	0	0	1	0
Sinusitis
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	0 / 4 (0.00%)	3 / 23 (13.04%)	0 / 6 (0.00%)	2 / 25 (8.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	1	0	3	0	3	0	0
Upper respiratory tract infection
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	1 / 3 (33.33%)	1 / 4 (25.00%)	2 / 23 (8.70%)	1 / 6 (16.67%)	2 / 25 (8.00%)	1 / 19 (5.26%)	1 / 21 (4.76%)
occurrences all number	0	0	2	1	3	1	3	1	1
Urinary tract infection
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	1 / 4 (25.00%)	2 / 23 (8.70%)	0 / 6 (0.00%)	2 / 25 (8.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	1	2	0	2	0	0
Metabolism and nutrition disorders
Dehydration
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	1 / 4 (25.00%)	1 / 23 (4.35%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	1	1	0	0	0	0
Hyperglycaemia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	0 / 23 (0.00%)	0 / 6 (0.00%)	0 / 25 (0.00%)	1 / 19 (5.26%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	0	0	0	1	0
Hypokalaemia
subjects affected / exposed	0 / 2 (0.00%)	0 / 2 (0.00%)	0 / 3 (0.00%)	0 / 4 (0.00%)	2 / 23 (8.70%)	0 / 6 (0.00%)	0 / 25 (0.00%)	0 / 19 (0.00%)	0 / 21 (0.00%)
occurrences all number	0	0	0	0	2	0	0	0	0

Notes
[1] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
Justification: This AE is gender specific event. The percentages of gender specific events are calculated using the corresponding gender count as denominator.

More information

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Were there any global substantial amendments to the protocol? Yes

05 Oct 2012

Interim analysis for futility was added.

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

None reported

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Clinical trials

Clinical Trial Results: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multi-Center Study to Investigate the Safety and Efficacy of MultiStem (PF-05285401) in Subjects With Moderate to Severe Ulcerative Colitis

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Change From Baseline in Endoscopic Score (as Measured by Modified Baron Score) at Week 8

Primary: Change From Baseline in Endoscopic Score (as Measured by Modified Baron Score) at Week 8

Primary: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 4

Primary: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 4

Primary: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 8

Primary: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 8

Primary: Number of Subjects with Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Primary: Number of Subjects with Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Primary: Incidence of Treatment-Emergent AEs by System Organ Class (SOC)

Primary: Incidence of Treatment-Emergent AEs by System Organ Class (SOC)

Primary: Number of Treatment-Emergent AEs by Severity

Primary: Number of Treatment-Emergent AEs by Severity

Secondary: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 12 and Week 16

Secondary: Change From Baseline in Rectal Bleeding Mayo Subscore at Week 12 and Week 16

Secondary: Number of Subjects With Laboratory Test Abnormalities

Secondary: Number of Subjects With Laboratory Test Abnormalities

Secondary: Number of Subjects With Potentially Clinically Significant Vital Signs Findings

Secondary: Number of Subjects With Potentially Clinically Significant Vital Signs Findings

Secondary: Fold Change From Baseline in Fecal Calprotectin at Weeks 4, 8, 12, and 16

Secondary: Fold Change From Baseline in Fecal Calprotectin at Weeks 4, 8, 12, and 16

Secondary: Fold Change From Baseline in C-Reactive Protein (CRP) at Weeks 4, 8, 12, and 16

Secondary: Fold Change From Baseline in C-Reactive Protein (CRP) at Weeks 4, 8, 12, and 16

Secondary: Percentage of Subjects With Rectal Bleeding Mayo Subscore of Zero at Weeks 4, 8, 12, and 16

Secondary: Percentage of Subjects With Rectal Bleeding Mayo Subscore of Zero at Weeks 4, 8, 12, and 16

Secondary: Percentage of Subjects in Endoscopic Remission at Week 8

Secondary: Percentage of Subjects in Endoscopic Remission at Week 8

Secondary: Percentage of Subjects in Clinical Remission at Week 8

Secondary: Percentage of Subjects in Clinical Remission at Week 8

Secondary: Percentage of Subjects With Decrease From Baseline of at Least 1 Point in Rectal Bleeding Mayo Subscore at Weeks 4, 8, 12, and 16

Secondary: Percentage of Subjects With Decrease From Baseline of at Least 1 Point in Rectal Bleeding Mayo Subscore at Weeks 4, 8, 12, and 16

Secondary: Percentage of Subjects With Endoscopic Response at Week 8

Secondary: Percentage of Subjects With Endoscopic Response at Week 8

Secondary: Percentage of Subjects in Clinical Response at Week 8

Secondary: Percentage of Subjects in Clinical Response at Week 8

Secondary: Change From Baseline in Total Mayo Scores at Week 8

Secondary: Change From Baseline in Total Mayo Scores at Week 8

Secondary: Change From Baseline in Partial Mayo Scores at Weeks 4, 8, 12, and 16

Secondary: Change From Baseline in Partial Mayo Scores at Weeks 4, 8, 12, and 16

Secondary: Change From Baseline in Biopsy Histology Scores at Week 8

Secondary: Change From Baseline in Biopsy Histology Scores at Week 8

Secondary: Change From Baseline in Patient-Reported Rectal Bleeding up to Week 16

Secondary: Change From Baseline in Patient-Reported Rectal Bleeding up to Week 16

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multi-Center Study to Investigate the Safety and Efficacy of MultiStem (PF-05285401) in Subjects With Moderate to Severe Ulcerative Colitis