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Clinical Trial Results:
A Double-blind, Randomized, Controlled Trial Comparing the Safety and Efficacy of AMDC-USR with Placebo in Female Subjects with Stress Urinary Incontinence

Summary
EudraCT number	2014-002919-41
Trial protocol	DE BE
Global end of trial date	10 Nov 2020

Results information
Results version number	v1(current)
This version publication date	27 Oct 2022
First version publication date	27 Oct 2022
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

13-003

ISRCTN number

US NCT number

NCT01893138

WHO universal trial number (UTN)

Sponsor organisation name

Cook MyoSite, Inc.

Sponsor organisation address

105 Delta Drive, Pittsburgh, PA, United States, 15238

Public contact

Kelly Cardello, Cook MyoSite, Inc., +1 412-963-7380, Kelly.Cardello@CookMyoSite.com

Scientific contact

Ron Jankowski, PhD, Cook MyoSite, Inc., +1 412-963-7380, Ron.Jankowski@CookMyoSite.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

10 Nov 2020

Is this the analysis of the primary completion data?

Yes

Primary completion date

10 Nov 2020

Global end of trial reached?

Yes

Global end of trial date

10 Nov 2020

Was the trial ended prematurely?

Main objective of the trial

To investigate the efficacy and safety of Autologous Muscle Derived Cells (AMDC, generic name iltamiocel, preparation of a patient's own cells) compared to a placebo (vehicle) control dose in the treatment of stress urinary incontinence (SUI) in adult female patients.

Protection of trial subjects

The study was conducted in accordance with the ethical principles that have their origins in the Declaration of Helsinki, including the International Council for Harmonization (ICH) Guideline for Good Clinical Practice and applicable regulations in the United States, Germany, and Belgium where the study took place.

Background therapy

Evidence for comparator

Actual start date of recruitment

21 Nov 2013

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Number of subjects enrolled per country

Country: Number of subjects enrolled

Belgium: 3

Country: Number of subjects enrolled

Germany: 7

Country: Number of subjects enrolled

United States: 287

Worldwide total number of subjects

297

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)

241

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

Subjects were screened and enrolled at 30 sites globally; in the United States, Germany, and Belgium.

Screening details

297 subjects were enrolled (underwent biopsy procedure) and 297 subjects received study treatment (iltamiocel injection). At randomization, participants were stratified by presence or absence of prior incontinence surgery and by < 10 or ≥10 stress incontinence episodes over 3-day diary at screening.

Period 1 title

Double-blind Period

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator, Monitor, Data analyst, Carer, Assessor

Are arms mutually exclusive

Yes

Iltamiocel

Arm description

AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intraurethral injection of 150 x 10^6 cells.

Arm type

Experimental

Investigational medicinal product name

Iltamiocel

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Intramuscular use

Dosage and administration details

Single intraurethral injection of 150 x 10^6 cells.

Placebo

Arm description

Placebo control is the vehicle solution used for the study product. Single intraurethral injection of vehicle control.

Arm type

Placebo

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Intramuscular use

Dosage and administration details

Single intraurethral injection of placebo.

Number of subjects in period 1	Iltamiocel	Placebo
Started	199	98
Iltamiocel Injection	199	98
1 Month Follow-Up	199	97
3 Month Follow-Up	199	97
6 Month Follow-Up	198	97
12 Month Follow-Up	198	97
Completed	198	97
Not completed	1	1
Consent withdrawn by subject	1	1

Period 2 title

Open Label - Unblinded Period

Is this the baseline period?

Allocation method

Non-randomised - controlled

Blinding used

Not blinded

Are arms mutually exclusive

Yes

Iltamiocel

Arm description

AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. No additional treatment for this arm in the open label period.

Arm type

No intervention

Investigational medicinal product name

No investigational medicinal product assigned in this arm

Placebo

Arm description

Subjects originally randomized to placebo received single, open-label iltamiocel injection of 150x10^6 cells.

Arm type

Experimental

Investigational medicinal product name

Iltamiocel

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Intramuscular use

Dosage and administration details

Single intraurethral injection of 150 x 10^6 cells.

Number of subjects in period 2	Iltamiocel	Placebo
Started	198	97
Open Label Period	166	97
Iltamiocel Injection	0 ^[1]	92
2 Year Follow-Up	166	87
Completed	166	87
Not completed	32	10
Consent withdrawn by subject	18	7
Adverse event, non-fatal	1	-
Lost to follow-up	4	3
Lack of efficacy	9	-

Notes
[1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
Justification: No additional treatment for iltamiocel arm in the open-label period.

Baseline characteristics

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Reporting group title

Iltamiocel

Reporting group description

AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intraurethral injection of 150 x 10^6 cells.

Reporting group title

Placebo

Reporting group description

Placebo control is the vehicle solution used for the study product. Single intraurethral injection of vehicle control.

Reporting group values	Iltamiocel	Placebo	Total
Number of subjects	199	98	297
Age categorical
Units: Subjects
In utero			0
Preterm newborn infants (gestational age < 37 wks)			0
Newborns (0-27 days)			0
Infants and toddlers (28 days-23 months)			0
Children (2-11 years)			0
Adolescents (12-17 years)			0
Adults (18-64 years)			0
From 65-84 years			0
85 years and over			0
Age continuous
Units: years
arithmetic mean (standard deviation)	54.1 ± 10.9	55.0 ± 10.7	-
Gender categorical
Units: Subjects
Female	199	98	297
Male	0	0	0
Stress Incontinence Episodes Over 3 Day Diary
Participant recorded number of stress leaks over 3 days in electronic diary.
Units: stress leaks
arithmetic mean (standard deviation)	14.2 ± 9.8	15.0 ± 14.2	-
24 Hour Pad Test Weight
A test in which the participant wears specific absorbent pads dispensed by the clinic. The pads are weighed to record the pre-test weight prior to dispensing to the participant. The participant must wear the absorbent pads for 24 hours. The participant returns the used absorbent pads to the clinic, and the clinic records the post-test weight to determine urinary leakage for the 24 hour period. The difference in weights represent the amount of urine leaked in which 24 hour pad weight = total weight of pads after test period - total weight of pads prior to test period.
Units: grams
arithmetic mean (standard deviation)	47.6 ± 91.8	40.9 ± 52.8	-
Incontinence Quality of Life (IQOL) Assessment -Total Score
The I-QOL questionnaire is a validated, 22-item tool used to assess quality of life (QOL) of women with urinary incontinence, focused on avoidance and limiting behavior, psychosocial impacts, and social embarrassment. Score scale is 0-100; increased score indicates improvement.. Scored 0 to100, with higher scores indicating a better QOL.
Units: scores on scales
arithmetic mean (standard deviation)	59.0 ± 21.0	59.5 ± 20.2	-
7-Item Incontinence Impact Questionnaire – Short Form (IIQ-7)
The IIQ-7 questionnaire is a validated, 7-item tool used to assess the impact of urinary incontinence on health-related quality of life, focused on physical activity, social relationships, travel, and emotional health. Scored 0 to 100, lower scores indicate better quality of life (QOL).
Units: scores on scales
arithmetic mean (standard deviation)	44.4 ± 20.9	43.7 ± 21.8	-
6-Item Urogenital Distress Inventory Score – Short Form (UDI-6)
This questionnaire consists of 6 questions focused on symptoms related to stress urinary incontinence, detrusor overactivity, and bladder outlet obstruction. Score scale is 0-75; decreased score indicates improvement.
Units: scores on scales
arithmetic mean (standard deviation)	38.5 ± 16.0	40.5 ± 18.4	-
Global Quality of Life Assessment (GQOL)
This questionnaire consists of 1 question focused on satisfaction with condition. Score scale is 0-6; decreased score indicates improvement.
Units: scores on scales
arithmetic mean (standard deviation)	5.7 ± 1.0	5.6 ± 1.1	-
Incontinence Severity Index (ISI)
The ISI is a questionnaire consisting of two questions which assesses the frequency and quantity of urine leakage. Score scale is 0-12; decreased score indicates improvement.
Units: scores on scales
arithmetic mean (standard deviation)	7.6 ± 2.4	7.4 ± 2.8	-

End points

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Reporting group title

Iltamiocel

Reporting group description

AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intraurethral injection of 150 x 10^6 cells.

Reporting group title

Placebo

Reporting group description

Placebo control is the vehicle solution used for the study product. Single intraurethral injection of vehicle control.

Reporting group title

Iltamiocel

Reporting group description

AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. No additional treatment for this arm in the open label period.

Reporting group title

Placebo

Reporting group description

Subjects originally randomized to placebo received single, open-label iltamiocel injection of 150x10^6 cells.

Subject analysis set title

Durability of 50% Reduction in SIEF at Month 24

Subject analysis set type

Sub-group analysis

Subject analysis set description

The number of subjects with 50% reduction of stress incontinence episode frequency (SIEF) at 12 months and diary data at Month 24.

Subject analysis set title

Durability of 75% Reduction in SIEF at Month 24

Subject analysis set type

Sub-group analysis

Subject analysis set description

The number of subjects with 75% reduction of stress incontinence episode frequency (SIEF) at 12 months and diary data at Month 24.

Subject analysis set title

Durability of 0 to 1 SIEF at Month 24

Subject analysis set type

Sub-group analysis

Subject analysis set description

The number of subjects with 0 to 1 stress incontinence episode frequency (SIEF) at 12 months and diary data at Month 24.

Subject analysis set title

Durability of 50% Reduction in SIEF at Month 24(Prior Surgery)

Subject analysis set type

Sub-group analysis

Subject analysis set description

The number of subjects with prior surgery with 50% reduction of stress incontinence episode frequency (SIEF) at 12 months and diary data at Month 24.

Subject analysis set title

Durability of 75% Reduction in SIEF at Month 24(Prior Surgery)

Subject analysis set type

Sub-group analysis

Subject analysis set description

The number of subjects with prior surgery with 75% reduction of stress incontinence episode frequency (SIEF) at 12 months and diary data at Month 24.

Subject analysis set title

Durability of 0 to 1 SIEF at Month 24 (Prior Surgery)

Subject analysis set type

Sub-group analysis

Subject analysis set description

The number of subjects with prior surgery with 0 to 1 stress incontinence episode frequency (SIEF) at 12 months and diary data at Month 24.

Primary: Participants With ≥ 50% Reduction in Stress Incontinence Episode Frequency From Baseline to 12 Months Post-treatment; as Assessed by 3 Day Diary

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End point title

Participants With ≥ 50% Reduction in Stress Incontinence Episode Frequency From Baseline to 12 Months Post-treatment; as Assessed by 3 Day Diary

End point description

All participants with baseline and 12 month 3 day diary data.

End point type

Primary

End point timeframe

12 months

End point values	Iltamiocel	Placebo
Number of subjects analysed	198	97
Units: participants	103	52

Chi-square test between iltamiocel & placebo arms

Comparison groups

Iltamiocel v Placebo

Number of subjects included in analysis

295

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.7978

Method

Chi-squared

Confidence interval

Secondary: Participants With at ≥ 75% Reduction in Stress Incontinence Episodes From Baseline at 12 Months

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End point title

Participants With at ≥ 75% Reduction in Stress Incontinence Episodes From Baseline at 12 Months

End point description

All participants with baseline and 12 month 3 day diary data.

End point type

Secondary

End point timeframe

12 months

End point values	Iltamiocel	Placebo
Number of subjects analysed	198	97
Units: participants	73	30

No statistical analyses for this end point

Secondary: Participants With 0 or 1 Stress Incontinence Episodes Based on 3 Day Diary Records at 12 Months

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End point title

Participants With 0 or 1 Stress Incontinence Episodes Based on 3 Day Diary Records at 12 Months

End point description

All participants with baseline and 12 month diary data.

End point type

Secondary

End point timeframe

12 months

End point values	Iltamiocel	Placebo
Number of subjects analysed	198	97
Units: participants	53	22

No statistical analyses for this end point

Secondary: Improvement (Reduction) in the Frequency of Stress Incontinence Episodes From Baseline at 12 Months

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End point title

Improvement (Reduction) in the Frequency of Stress Incontinence Episodes From Baseline at 12 Months

End point description

All participants with baseline and 12 month 3 day diary data.

End point type

Secondary

End point timeframe

12 months

End point values	Iltamiocel	Placebo
Number of subjects analysed	198	97
Units: change in number of stress leaks
arithmetic mean (standard deviation)	-5.8 ± 11	-4.9 ± 13.5

No statistical analyses for this end point

Other pre-specified: Association of Quality of Life Improvement With Stress Incontinence Episode Reduction

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End point title

Association of Quality of Life Improvement With Stress Incontinence Episode Reduction

End point description

All participants with baseline and 12 month diary data; Spearman's correlation used for analysis. Association of change in questionnaires with change in stress incontinence episode frequency (SIEF) at 12 months.

End point type

Other pre-specified

End point timeframe

12 months

End point values	Iltamiocel	Placebo
Number of subjects analysed	198	96
Units: correlation coefficient
number (not applicable)
Change in IQOL vs change in SIEF	-0.556	-0.456
Change in IIQ-7 vs change in SIEF	0.522	0.442
Change in UDI-6 vs change in SIEF	0.408	0.433
Change in GQOL vs change in SIEF	0.537	0.422
Change in ISI vs change in SIEF	0.529	0.599

No statistical analyses for this end point

Other pre-specified: Treatment Durability at 24 Months

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End point title

Treatment Durability at 24 Months

End point description

Treatment durability defined as iltamiocel-treated participants with categorical reduction in stress incontinence episode frequency (SIEF) at 12 months who maintained response at 24 months. All participants with 12 month and 24 month stress incontinence episode frequency (SIEF) diary data.

End point type

Other pre-specified

End point timeframe

12 months to 24 months after injection with iltamiocel

End point values	Durability of 50% Reduction in SIEF at Month 24	Durability of 75% Reduction in SIEF at Month 24	Durability of 0 to 1 SIEF at Month 24
Number of subjects analysed	89	65	45
Units: participants	80	50	36

No statistical analyses for this end point

Post-hoc: Participants With at ≥ 75% Reduction in Stress Incontinence Episodes From Baseline at 12 Months (Prior Surgery Participants Only)

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End point title

Participants With at ≥ 75% Reduction in Stress Incontinence Episodes From Baseline at 12 Months (Prior Surgery Participants Only)

End point description

Participants with a history of prior SUI surgery with baseline and 12 month diary data.

End point type

Post-hoc

End point timeframe

12 months

End point values	Iltamiocel	Placebo
Number of subjects analysed	50	25
Units: participants	20	4

No statistical analyses for this end point

Post-hoc: Participants With 0 or 1 Stress Incontinence Episodes Based on 3 Day Diary Records at 12 Months (Prior Surgery Participants Only)

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End point title

Participants With 0 or 1 Stress Incontinence Episodes Based on 3 Day Diary Records at 12 Months (Prior Surgery Participants Only)

End point description

Participants with a history of prior SUI surgery with baseline and 12 month diary data.

End point type

Post-hoc

End point timeframe

12 months

End point values	Iltamiocel	Placebo
Number of subjects analysed	50	25
Units: participants	14	3

No statistical analyses for this end point

Post-hoc: Improvement (Reduction) in the Frequency of Stress Incontinence Episodes From Baseline at 12 Months (Prior Surgery Participants Only)

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End point title

Improvement (Reduction) in the Frequency of Stress Incontinence Episodes From Baseline at 12 Months (Prior Surgery Participants Only)

End point description

Participants with a history of prior SUI surgery with baseline and 12 month diary data.

End point type

Post-hoc

End point timeframe

12 months

End point values	Iltamiocel	Placebo
Number of subjects analysed	50	25
Units: change in number of stress leaks
arithmetic mean (standard deviation)	-6.7 ± 14.5	-4.5 ± 11.5

No statistical analyses for this end point

Post-hoc: Association of Quality of Life Improvement With Stress Incontinence Episode Reduction (Prior Surgery Participants Only)

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End point title

Association of Quality of Life Improvement With Stress Incontinence Episode Reduction (Prior Surgery Participants Only)

End point description

All prior surgery participants with baseline and 12 month diary data; Spearman's correlation used for analysis. Association of change in questionnaires with change in stress incontinence episode frequency (SIEF) at 12 months.

End point type

Post-hoc

End point timeframe

12 months

End point values	Iltamiocel	Placebo
Number of subjects analysed	50	25
Units: correlation coefficient
number (not applicable)
Change in IQOL vs change in SIEF	-0.489	-0.460
Change in IIQ-7 vs change in SIEF	0.427	0.542
Change in UDI-6 vs change in SIEF	0.424	0.604
Change in GQOL vs change in SIEF	0.561	0.190
Change in ISI vs change in SIEF	0.426	0.476

No statistical analyses for this end point

Post-hoc: Treatment Durability at 24 Months (Prior Surgery Participants)

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End point title

Treatment Durability at 24 Months (Prior Surgery Participants)

End point description

Treatment durability defined as iltamiocel-treated participants with reduction in stress incontinence episode frequency (SIEF) at 12 months who maintained response at 24 months. Prior surgery participants with 12 month and 24 month stress incontinence episode frequency (SIEF) diary data.

End point type

Post-hoc

End point timeframe

12 months to 24 months after injection with iltamiocel

End point values	Durability of 50% Reduction in SIEF at Month 24(Prior Surgery)	Durability of 75% Reduction in SIEF at Month 24(Prior Surgery)	Durability of 0 to 1 SIEF at Month 24 (Prior Surgery)
Number of subjects analysed	22	18	13
Units: participants	19	12	11

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

12 month double-blind period

Adverse event reporting additional description

Collection at injection, 1 month, 3 months, 6 months, and 12 months post-treatment

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

16.1

Reporting group title

Iltamiocel

Reporting group description

AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intraurethral injection of 150 x 10^6 cells.

Reporting group title

Placebo

Reporting group description

Placebo control is the vehicle solution used for the study product. Single intraurethral injection of vehicle control.

Serious adverse events	Iltamiocel	Placebo
Total subjects affected by serious adverse events
subjects affected / exposed	11 / 199 (5.53%)	9 / 98 (9.18%)
number of deaths (all causes)	0	0
number of deaths resulting from adverse events	0	0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Papillary thyroid cancer
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Injury, poisoning and procedural complications
Meniscus injury
subjects affected / exposed	0 / 199 (0.00%)	1 / 98 (1.02%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Pelvic fracture
subjects affected / exposed	0 / 199 (0.00%)	1 / 98 (1.02%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Procedural Pain
subjects affected / exposed	2 / 199 (1.01%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 2	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Cardiac disorders
Acute myocardial infarction
subjects affected / exposed	0 / 199 (0.00%)	1 / 98 (1.02%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Coronary artery dissection
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Nervous system disorders
Transient ischaemic attack
subjects affected / exposed	0 / 199 (0.00%)	1 / 98 (1.02%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
General disorders and administration site conditions
Chest pain
subjects affected / exposed	0 / 199 (0.00%)	1 / 98 (1.02%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Systemic inflammatory response syndrome
subjects affected / exposed	0 / 199 (0.00%)	1 / 98 (1.02%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Immune system disorders
Hypersensitivity
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Gastrointestinal disorders
Colitis
subjects affected / exposed	0 / 199 (0.00%)	1 / 98 (1.02%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Gastritis
subjects affected / exposed	0 / 199 (0.00%)	1 / 98 (1.02%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Pancreatitis
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Atelectasis
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Psychiatric disorders
Major depression
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Suicide attempt
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Renal and urinary disorders
Nephrolithiasis
subjects affected / exposed	0 / 199 (0.00%)	1 / 98 (1.02%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Musculoskeletal and connective tissue disorders
Intervertebral disc degeneration
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Osteoarthritis
subjects affected / exposed	0 / 199 (0.00%)	1 / 98 (1.02%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Pain in extremity
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Scoliosis
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Spondylolisthesis
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Infections and infestations
Pneumonia
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Pyelonephritis
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Sepsis
subjects affected / exposed	1 / 199 (0.50%)	1 / 98 (1.02%)
occurrences causally related to treatment / all	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Staphylococcal infection
subjects affected / exposed	2 / 199 (1.01%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 2	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Urinary tract infection
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Viral infection
subjects affected / exposed	1 / 199 (0.50%)	0 / 98 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0

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

Non-serious adverse events	Iltamiocel	Placebo
Total subjects affected by non serious adverse events
subjects affected / exposed	84 / 199 (42.21%)	28 / 98 (28.57%)
General disorders and administration site conditions
Injection site pain
subjects affected / exposed	10 / 199 (5.03%)	1 / 98 (1.02%)
occurrences all number	11	1
Renal and urinary disorders
Dysuria
subjects affected / exposed	20 / 199 (10.05%)	7 / 98 (7.14%)
occurrences all number	20	8
Musculoskeletal and connective tissue disorders
Back pain
subjects affected / exposed	7 / 199 (3.52%)	5 / 98 (5.10%)
occurrences all number	8	6
Infections and infestations
Sinusitis
subjects affected / exposed	11 / 199 (5.53%)	1 / 98 (1.02%)
occurrences all number	11	1
Urinary tract infection
subjects affected / exposed	36 / 199 (18.09%)	14 / 98 (14.29%)
occurrences all number	53	19

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Were there any global substantial amendments to the protocol? Yes

03 Jun 2014

Substantial amendment to protocol (version 13-003-02, dated 03 June 2014) for use in United States.

03 Nov 2015

Substantial amendment 01 to protocol (version 13-003-04-BE, dated 06 October 2015) for use in Belgium.

30 Nov 2015

Substantial amendment to protocol (version 13-003-03, dated 02 November 2015) for use in United States.

01 Feb 2016

Substantial amendment to protocol (version 13-003-04, dated 14 January 2016) for use in United States.

08 Mar 2016

Substantial amendment to protocol (version 13-003-05, dated 18 February 2016) for use in United States.

27 Jan 2017

Substantial amendment to protocol (version 13-003-05-BE, dated 19 January 2017) for use in Belgium.

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 Double-blind, Randomized, Controlled Trial Comparing the Safety and Efficacy of AMDC-USR with Placebo in Female Subjects with Stress Urinary Incontinence

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Participants With ≥ 50% Reduction in Stress Incontinence Episode Frequency From Baseline to 12 Months Post-treatment; as Assessed by 3 Day Diary

Primary: Participants With ≥ 50% Reduction in Stress Incontinence Episode Frequency From Baseline to 12 Months Post-treatment; as Assessed by 3 Day Diary

Secondary: Participants With at ≥ 75% Reduction in Stress Incontinence Episodes From Baseline at 12 Months

Secondary: Participants With at ≥ 75% Reduction in Stress Incontinence Episodes From Baseline at 12 Months

Secondary: Participants With 0 or 1 Stress Incontinence Episodes Based on 3 Day Diary Records at 12 Months

Secondary: Participants With 0 or 1 Stress Incontinence Episodes Based on 3 Day Diary Records at 12 Months

Secondary: Improvement (Reduction) in the Frequency of Stress Incontinence Episodes From Baseline at 12 Months

Secondary: Improvement (Reduction) in the Frequency of Stress Incontinence Episodes From Baseline at 12 Months

Other pre-specified: Association of Quality of Life Improvement With Stress Incontinence Episode Reduction

Other pre-specified: Association of Quality of Life Improvement With Stress Incontinence Episode Reduction

Other pre-specified: Treatment Durability at 24 Months

Other pre-specified: Treatment Durability at 24 Months

Post-hoc: Participants With at ≥ 75% Reduction in Stress Incontinence Episodes From Baseline at 12 Months (Prior Surgery Participants Only)

Post-hoc: Participants With at ≥ 75% Reduction in Stress Incontinence Episodes From Baseline at 12 Months (Prior Surgery Participants Only)

Post-hoc: Participants With 0 or 1 Stress Incontinence Episodes Based on 3 Day Diary Records at 12 Months (Prior Surgery Participants Only)

Post-hoc: Participants With 0 or 1 Stress Incontinence Episodes Based on 3 Day Diary Records at 12 Months (Prior Surgery Participants Only)

Post-hoc: Improvement (Reduction) in the Frequency of Stress Incontinence Episodes From Baseline at 12 Months (Prior Surgery Participants Only)

Post-hoc: Improvement (Reduction) in the Frequency of Stress Incontinence Episodes From Baseline at 12 Months (Prior Surgery Participants Only)

Post-hoc: Association of Quality of Life Improvement With Stress Incontinence Episode Reduction (Prior Surgery Participants Only)

Post-hoc: Association of Quality of Life Improvement With Stress Incontinence Episode Reduction (Prior Surgery Participants Only)

Post-hoc: Treatment Durability at 24 Months (Prior Surgery Participants)

Post-hoc: Treatment Durability at 24 Months (Prior Surgery Participants)

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

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Spain
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Outside EU/EEA

Clinical trials

Clinical Trial Results: A Double-blind, Randomized, Controlled Trial Comparing the Safety and Efficacy of AMDC-USR with Placebo in Female Subjects with Stress Urinary Incontinence

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Participants With ≥ 50% Reduction in Stress Incontinence Episode Frequency From Baseline to 12 Months Post-treatment; as Assessed by 3 Day Diary

Primary: Participants With ≥ 50% Reduction in Stress Incontinence Episode Frequency From Baseline to 12 Months Post-treatment; as Assessed by 3 Day Diary

Secondary: Participants With at ≥ 75% Reduction in Stress Incontinence Episodes From Baseline at 12 Months

Secondary: Participants With at ≥ 75% Reduction in Stress Incontinence Episodes From Baseline at 12 Months

Secondary: Participants With 0 or 1 Stress Incontinence Episodes Based on 3 Day Diary Records at 12 Months

Secondary: Participants With 0 or 1 Stress Incontinence Episodes Based on 3 Day Diary Records at 12 Months

Secondary: Improvement (Reduction) in the Frequency of Stress Incontinence Episodes From Baseline at 12 Months

Secondary: Improvement (Reduction) in the Frequency of Stress Incontinence Episodes From Baseline at 12 Months

Other pre-specified: Association of Quality of Life Improvement With Stress Incontinence Episode Reduction

Other pre-specified: Association of Quality of Life Improvement With Stress Incontinence Episode Reduction

Other pre-specified: Treatment Durability at 24 Months

Other pre-specified: Treatment Durability at 24 Months

Post-hoc: Participants With at ≥ 75% Reduction in Stress Incontinence Episodes From Baseline at 12 Months (Prior Surgery Participants Only)

Post-hoc: Participants With at ≥ 75% Reduction in Stress Incontinence Episodes From Baseline at 12 Months (Prior Surgery Participants Only)

Post-hoc: Participants With 0 or 1 Stress Incontinence Episodes Based on 3 Day Diary Records at 12 Months (Prior Surgery Participants Only)

Post-hoc: Participants With 0 or 1 Stress Incontinence Episodes Based on 3 Day Diary Records at 12 Months (Prior Surgery Participants Only)

Post-hoc: Improvement (Reduction) in the Frequency of Stress Incontinence Episodes From Baseline at 12 Months (Prior Surgery Participants Only)

Post-hoc: Improvement (Reduction) in the Frequency of Stress Incontinence Episodes From Baseline at 12 Months (Prior Surgery Participants Only)

Post-hoc: Association of Quality of Life Improvement With Stress Incontinence Episode Reduction (Prior Surgery Participants Only)

Post-hoc: Association of Quality of Life Improvement With Stress Incontinence Episode Reduction (Prior Surgery Participants Only)

Post-hoc: Treatment Durability at 24 Months (Prior Surgery Participants)

Post-hoc: Treatment Durability at 24 Months (Prior Surgery Participants)

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
A Double-blind, Randomized, Controlled Trial Comparing the Safety and Efficacy of AMDC-USR with Placebo in Female Subjects with Stress Urinary Incontinence