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Clinical Trial Results:
A Mulitcenter, Open-Label PK Study of Mirikizumab in Pediatric Patients with Moderately to Severely Active Ulcerative Colitis

Summary
EudraCT number	2019-001298-96
Trial protocol	Outside EU/EEA
Global end of trial date	15 Mar 2023

Results information
Results version number	v1(current)
This version publication date	28 Sep 2023
First version publication date	28 Sep 2023
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

I6T-MC-AMBU

ISRCTN number

US NCT number

NCT04004611

WHO universal trial number (UTN)

Other trial identifiers

Trial Number: 17410

Sponsor organisation name

Eli Lilly and Company

Sponsor organisation address

Lilly Corporate Center, Indianapolis, IN, United States, 46285

Public contact

Available Mon ‐ Fri 9 AM ‐ 5 PM EST, Eli Lilly and Company, 1 877‐CTLilly,

Scientific contact

Available Mon ‐ Fri 9 AM ‐ 5 PM EST, Eli Lilly and Company, 1 877‐285‐4559,

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

Yes

EMA paediatric investigation plan number(s)

EMEA-002208-PIP01-17

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

15 Mar 2023

Is this the analysis of the primary completion data?

Global end of trial reached?

Yes

Global end of trial date

15 Mar 2023

Was the trial ended prematurely?

Main objective of the trial

This study was designed to evaluate how the body processes and removes mirikizumab. The study also evaluated safety and disease response in pediatric participants with UC taking mirikizumab. The study lasted about 52 weeks and included up to 18 visits.

Protection of trial subjects

This study was conducted in accordance with International Conference on Harmonization (ICH) Good Clinical Practice, and the principles of the Declaration of Helsinki, in addition to following the laws and regulations of the country or countries in which a study is conducted.

Background therapy

Evidence for comparator

Actual start date of recruitment

18 May 2020

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Number of subjects enrolled per country

Country: Number of subjects enrolled

Japan: 4

Country: Number of subjects enrolled

Korea, Republic of: 4

Country: Number of subjects enrolled

United States: 17

Country: Number of subjects enrolled

Israel: 1

Worldwide total number of subjects

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

Mirikizumab (Miri) dose groups to which pediatric participants are assigned at Week (wk) 0 (for induction period) and at wk 12 (for maintenance period) are dependent on participant' s weight and their clinical response status at wk 12 for maintenance period.

Screening details

All participants who achieved a modified Mayo score (MMS) clinical response at wk 12 or wk 24 [non-responders (NR) at wk 12 who received extended intravenous (IV) induction dosing for 12 more wks] were eligible for the maintenance period.

Period 1 title

OL Induction Period (Wk 0-12)

Is this the baseline period?

Yes

Allocation method

Non-randomised - controlled

Blinding used

Not blinded

Are arms mutually exclusive

Yes

OL Induction Period: 5 milligram per kilogram (mg/kg) Miri IV

Arm description

Participants (≤40 kg weight) received 5 mg/kg mirikizumab given as an IV infusion every 4 weeks (Q4W) on weeks 0, 4, 8 for 12 weeks.

Arm type

Experimental

Investigational medicinal product name

5 mg/kg mirikizumab

Investigational medicinal product code

LY3074828

Other name

Pharmaceutical forms

Injection

Routes of administration

Intravenous use

Dosage and administration details

Participants (≤40 kg weight) received 5 mg/kg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks.

OL Induction Period: 10 mg/kg Miri IV

Arm description

Participants (≤40 kg weight) received 10 mg/kg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks.

Arm type

Experimental

Investigational medicinal product name

10 mg/kg mirikizumab

Investigational medicinal product code

LY3074828

Other name

Pharmaceutical forms

Injection

Routes of administration

Intravenous use

Dosage and administration details

Participants (≤40 kg weight) received 10 mg/kg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks.

OL Induction Period: 300 mg Miri IV

Arm description

Participants (>40 kg weight) received 300 mg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks.

Arm type

Experimental

Investigational medicinal product name

300 mg mirikizumab

Investigational medicinal product code

LY3074828

Other name

Pharmaceutical forms

Injection

Routes of administration

Subcutaneous use

Dosage and administration details

Participants (>40 kg weight) received 300 mg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks.

Number of subjects in period 1	OL Induction Period: 5 milligram per kilogram (mg/kg) Miri IV	OL Induction Period: 10 mg/kg Miri IV	OL Induction Period: 300 mg Miri IV
Started	10	5	11
Completed	10	5	11

Period 2 title

OL Maintenance Period (Wk 12-52)

Is this the baseline period?

Allocation method

Non-randomised - controlled

Blinding used

Not blinded

Are arms mutually exclusive

Yes

OL Maintenance Period: 50 mg Miri Subcutaneous (SC)

Arm description

Participants (≤20 kg weight) who were responders to mirikizumab at week 12 in induction received 50 mg SC Q4W from week 12 through week 48 or until loss of response was confirmed.

Arm type

Experimental

Investigational medicinal product name

50 mg mirikizumab

Investigational medicinal product code

LY3074828

Other name

Pharmaceutical forms

Injection

Routes of administration

Subcutaneous use

Dosage and administration details

Participants (≤20 kg weight) who were responders to mirikizumab at week 12 in induction received 50 mg SC Q4W from week 12 through week 48 or until loss of response was confirmed.

OL Maintenance Period: 100 mg Miri SC

Arm description

Participants (>20 to ≤40 kg weight) who were responders to mirikizumab at week 12 in induction received 100 mg SC Q4W from week 12 through week 48 or until loss of response was confirmed.

Arm type

Experimental

Investigational medicinal product name

100 mg mirikizumab

Investigational medicinal product code

LY3074828

Other name

Pharmaceutical forms

Injection

Routes of administration

Subcutaneous use

Dosage and administration details

Participants (>20 to ≤40 kg weight) who were responders to mirikizumab at week 12 in induction received 100 mg SC Q4W from week 12 through week 48 or until loss of response was confirmed.

OL Maintenance Period: 200 mg Miri SC

Arm description

Participants (>40 kg weight) who were responders to mirikizumab at week 12 in induction received 200 mg SC Q4W from week 12 through week 48 or until loss of response was confirmed.

Arm type

Experimental

Investigational medicinal product name

200 mg mirikizumab

Investigational medicinal product code

LY3074828

Other name

Pharmaceutical forms

Injection

Routes of administration

Subcutaneous use

Dosage and administration details

Participants (>40 kg weight) who were responders to mirikizumab at week 12 in induction received 200 mg SC Q4W from week 12 through week 48 or until loss of response was confirmed.

OL Maintenance Period: NR: 10 mg/kg Miri IV/50 mg Miri SC

Arm description

Participants (≤40 kg) who were non responders to miri at week 12 in induction received 10 mg SC Q4W for 12 weeks or discontinued after repeat induction, and then received 50 mg miri (≤20 kg weight) SC Q4W through week 48 or until loss of response was confirmed.

Arm type

Experimental

Investigational medicinal product name

50 mg mirikizumab

Investigational medicinal product code

LY3074828

Other name

Pharmaceutical forms

Injection

Routes of administration

Subcutaneous use

Dosage and administration details

OL Maintenance Period: NR: 10 mg/kg Miri IV/100 mg Miri SC

Arm description

Participants (≤40 kg) who were non responders to miri at week 12 in induction received 10 mg SC Q4W for 12 weeks or discontinued after repeat induction, and then received 100 mg miri (>20 to ≤40 kg weight) SC Q4W through week 48 or until loss of response was confirmed.

Arm type

Experimental

Investigational medicinal product name

100 mg mirikizumab

Investigational medicinal product code

LY3074828

Other name

Pharmaceutical forms

Injection

Routes of administration

Subcutaneous use

Dosage and administration details

OL Maintenance Period: NR: 300 mg Miri IV /200 mg Miri SC

Arm description

Participants (>40 kg) who were non responders to miri at week 12 in induction received 300 mg SC Q4W for 12 weeks or discontinued after repeat induction, then received 200 mg miri SC Q4W through week 48 or until loss of response was confirmed.

Arm type

Experimental

Investigational medicinal product name

200 mg mirikizumab

Investigational medicinal product code

LY3074828

Other name

Pharmaceutical forms

Injection

Routes of administration

Subcutaneous use

Dosage and administration details

Number of subjects in period 2	OL Maintenance Period: 50 mg Miri Subcutaneous (SC)	OL Maintenance Period: 100 mg Miri SC	OL Maintenance Period: 200 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/50 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/100 mg Miri SC	OL Maintenance Period: NR: 300 mg Miri IV /200 mg Miri SC
Started	1	8	9	1	1	6
Completed	1	7	8	0	1	2
Not completed	0	1	1	1	0	4
Adverse event, non-fatal	-	-	-	-	-	1
Withdrawal by Parent or Guardian	-	1	-	-	-	-
Lack of efficacy	-	-	1	1	-	3

Period 3 title

OL Induction and Maintenance Period

Is this the baseline period?

Allocation method

Non-randomised - controlled

Blinding used

Not blinded

Are arms mutually exclusive

OL Induction and Maintenance Period: 5 mg/kg Miri IV

Arm description

Participants (≤40 kg weight) received 5 mg/kg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks followed by IV or SC mirikizumab based on their week 12 response status and weight through week 48 or until loss of response was confirmed.

Arm type

Experimental

Investigational medicinal product name

5 mg/kg mirikizumab

Investigational medicinal product code

LY3074828

Other name

Pharmaceutical forms

Injection

Routes of administration

Intravenous use

Dosage and administration details

OL Induction and Maintenance Period: 10 mg/kg Miri IV

Arm description

Participants (≤40 kg weight) received 10 mg/kg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks followed by IV or SC mirikizumab based on their week 12 response status and weight through week 48 or until loss of response was confirmed.

Arm type

Experimental

Investigational medicinal product name

10 mg/kg mirikizumab

Investigational medicinal product code

LY3074828

Other name

Pharmaceutical forms

Injection

Routes of administration

Intravenous use

Dosage and administration details

OL Induction and Maintenance Period: 300 mg Miri IV

Arm description

Participants (>40 kg weight) received 300 mg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks followed by IV or SC mirikizumab based on their week 12 response status and weight through week 48 or until loss of response was confirmed.

Arm type

Experimental

Investigational medicinal product name

300 mg mirikizumab

Investigational medicinal product code

LY3074828

Other name

Pharmaceutical forms

Injection

Routes of administration

Subcutaneous use

Dosage and administration details

Number of subjects in period 3	OL Induction and Maintenance Period: 5 mg/kg Miri IV	OL Induction and Maintenance Period: 10 mg/kg Miri IV	OL Induction and Maintenance Period: 300 mg Miri IV
Started	10	5	11
Completed	9	4	6
Not completed	1	1	5
Adverse event, non-fatal	-	-	1
Withdrawal by Parent or Guardian	-	1	-
Lack of efficacy	1	-	4

Baseline characteristics

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Reporting group title

OL Induction Period: 5 milligram per kilogram (mg/kg) Miri IV

Reporting group description

Participants (≤40 kg weight) received 5 mg/kg mirikizumab given as an IV infusion every 4 weeks (Q4W) on weeks 0, 4, 8 for 12 weeks.

Reporting group title

OL Induction Period: 10 mg/kg Miri IV

Reporting group description

Participants (≤40 kg weight) received 10 mg/kg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks.

Reporting group title

OL Induction Period: 300 mg Miri IV

Reporting group description

Participants (>40 kg weight) received 300 mg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks.

Reporting group values	OL Induction Period: 5 milligram per kilogram (mg/kg) Miri IV	OL Induction Period: 10 mg/kg Miri IV	OL Induction Period: 300 mg Miri IV	Total
Number of subjects	10	5	11	26
Age categorical
Units: Subjects
Age continuous
All randomized participants.
Units: years
arithmetic mean (standard deviation)	9.6 ± 4.22	11.6 ± 1.14	14.0 ± 1.26	-
Gender categorical
All randomized participants.
Units: Subjects
Female	6	3	6	15
Male	4	2	5	11
Ethnicity (NIH/OMB)
All randomized participants.
Units: Subjects
Hispanic or Latino	3	0	0	3
Not Hispanic or Latino	2	3	8	13
Unknown or Not Reported	5	2	3	10
Race (NIH/OMB)
All randomized participants.
Units: Subjects
American Indian or Alaska Native	0	0	0	0
Asian	4	2	1	7
Native Hawaiian or Other Pacific Islander	0	0	0	0
Black or African American	0	0	0	0
White	5	3	9	17
More than one race	1	0	0	1
Unknown or Not Reported	0	0	1	1
Region of Enrollment
All randomized participants.
Units: Subjects
Israel	0	0	1	1
Japan	1	2	1	4
South Korea	4	0	0	4
United States	5	3	9	17

End points

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Reporting group title

OL Induction Period: 5 milligram per kilogram (mg/kg) Miri IV

Reporting group description

Participants (≤40 kg weight) received 5 mg/kg mirikizumab given as an IV infusion every 4 weeks (Q4W) on weeks 0, 4, 8 for 12 weeks.

Reporting group title

OL Induction Period: 10 mg/kg Miri IV

Reporting group description

Participants (≤40 kg weight) received 10 mg/kg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks.

Reporting group title

OL Induction Period: 300 mg Miri IV

Reporting group description

Participants (>40 kg weight) received 300 mg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks.

Reporting group title

OL Maintenance Period: 50 mg Miri Subcutaneous (SC)

Reporting group description

Participants (≤20 kg weight) who were responders to mirikizumab at week 12 in induction received 50 mg SC Q4W from week 12 through week 48 or until loss of response was confirmed.

Reporting group title

OL Maintenance Period: 100 mg Miri SC

Reporting group description

Participants (>20 to ≤40 kg weight) who were responders to mirikizumab at week 12 in induction received 100 mg SC Q4W from week 12 through week 48 or until loss of response was confirmed.

Reporting group title

OL Maintenance Period: 200 mg Miri SC

Reporting group description

Participants (>40 kg weight) who were responders to mirikizumab at week 12 in induction received 200 mg SC Q4W from week 12 through week 48 or until loss of response was confirmed.

Reporting group title

OL Maintenance Period: NR: 10 mg/kg Miri IV/50 mg Miri SC

Reporting group description

Reporting group title

OL Maintenance Period: NR: 10 mg/kg Miri IV/100 mg Miri SC

Reporting group description

Reporting group title

OL Maintenance Period: NR: 300 mg Miri IV /200 mg Miri SC

Reporting group description

Reporting group title

OL Induction and Maintenance Period: 5 mg/kg Miri IV

Reporting group description

Reporting group title

OL Induction and Maintenance Period: 10 mg/kg Miri IV

Reporting group description

Reporting group title

OL Induction and Maintenance Period: 300 mg Miri IV

Reporting group description

Subject analysis set title

Mirikizumab

Subject analysis set type

Per protocol

Subject analysis set description

Participants weighing >40 kg received a mirikizumab induction dose of 300 mg via IV infusion and participants weighing ≤40 kg received induction doses of 5 mg/kg or 10 mg/kg via IV infusion at Weeks 0, 4, and 8 for 12 weeks.

Primary: Pharmacokinetics (PK): Clearance of Mirikizumab

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End point title

Pharmacokinetics (PK): Clearance of Mirikizumab ^[1]

End point description

Clearance of mirikizumab was evaluated. The PK of mirikizumab is characterized at interim analysis points using mixed-effect (population PK) modelling approaches using the available induction and maintenance mirikizumab concentration data. Analysis population description (APD) included all randomized participants who received at least one dose of study drug and had evaluable PK data.

End point type

Primary

End point timeframe

Predose on week 4, 8, 12,16, 24, 36, 52 and post dose on week 0 and 8

Notes
[1] - 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 inferential statistics was planned for this endpoint.

End point values	Mirikizumab
Number of subjects analysed	26
Units: Liters per hour per kilogram (L/hr/kg)
geometric mean (geometric coefficient of variation)	0.000190 ± 59.74

No statistical analyses for this end point

Secondary: Percentage of Participants in Clinical Remission

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End point title

Percentage of Participants in Clinical Remission

End point description

Clinical remission defined as achieving a 9-point modified Mayo score (MMS) for rectal bleeding (RB) = 0, stool frequency (SF) = 0 or 1 and endoscopy (ES) = 0 or 1 (excluding friability). The MMS is a composite score of ulcerative colitis disease activity calculated as the sum of three subscores: SF subscore, based on the participant's diary and scored from 0 (normal number of stools) to 3 (5 or more stools than normal); RB subscore, based on the participant's diary and scored from 0 (no blood seen) to 3 (blood alone passed); ES subscore, based on colonoscopy or sigmoidoscopy and scored from 0 (normal or inactive disease) to 3 (severe disease, spontaneous bleeding,ulceration. APD included Modified Intention-to-treat population (mITT):All randomized participants who received at least one dose of study drug and who had clinical remission measured correctly at baseline. Participants were analysed per their assigned treatment arm regardless of the treatment they actually received.

End point type

Secondary

End point timeframe

Week 52

End point values	OL Maintenance Period: 50 mg Miri Subcutaneous (SC)	OL Maintenance Period: 100 mg Miri SC	OL Maintenance Period: 200 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/50 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/100 mg Miri SC	OL Maintenance Period: NR: 300 mg Miri IV /200 mg Miri SC
Number of subjects analysed	1	8	9	1	1	6
Units: percentage of participants
number (confidence interval 95%)	0.0 (0.0 to 79.3)	62.5 (30.6 to 86.3)	55.6 (26.7 to 81.1)	0.0 (0.0 to 79.3)	0.0 (0.0 to 79.3)	0.0 (0.0 to 39.0)

No statistical analyses for this end point

Secondary: Percentage of Participants in Clinical Response

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End point title

Percentage of Participants in Clinical Response

End point description

Clinical response defined as a decrease in the 9-point MMS [RB, SF and the ES findings] inclusive of ≥ 2 points and ≥30% from baseline with either a decrease of RB subscore of ≥ or RB subscore of 0 or 1. The MMS is a composite score of ulcerative colitis disease activity calculated as the sum of three subscores: SF subscore, based on the participant's diary and scored from 0 (normal number of stools) to 3 (5 or more stools than normal); RB subscore, based on the participant's diary and scored from 0 (no blood seen) to 3 (blood alone passed); ES subscore, based on colonoscopy or sigmoidoscopy and scored from 0 (normal or inactive disease) to 3 (severe disease, spontaneous bleeding, ulceration). APD included mITT: All randomized participants who received at least one dose of study drug and who had the clinical response measured correctly at baseline. Participants were analysed per their assigned treatment arm regardless of the treatment they actually received.

End point type

Secondary

End point timeframe

Week 52

End point values	OL Maintenance Period: 50 mg Miri Subcutaneous (SC)	OL Maintenance Period: 100 mg Miri SC	OL Maintenance Period: 200 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/50 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/100 mg Miri SC	OL Maintenance Period: NR: 300 mg Miri IV /200 mg Miri SC
Number of subjects analysed	1	8	9	1	1	6
Units: percentage of participants
number (confidence interval 95%)	0.0 (0.0 to 79.3)	75.0 (40.9 to 92.9)	88.9 (56.5 to 98.0)	0.0 (0.0 to 79.3)	0.0 (0.0 to 79.3)	0.0 (0.0 to 39.0)

No statistical analyses for this end point

Secondary: Percentage of Participants Who are in MMS Clinical Remission Without the Use of Corticosteroids

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End point title

Percentage of Participants Who are in MMS Clinical Remission Without the Use of Corticosteroids

End point description

Corticosteroid-free clinical remission was defined as an SF subscore = 0 or 1, RB subscore = 0, ES ≤ 1 (excluding friability), and have not received corticosteroids for ≥ 12 weeks in the 52-Week Treatment Period. Each component subscore ranged from 0 to 3 and total score range of the MMS was from 0 to 9, with higher scores indicating more severe disease. APD included mITT: All randomized participants who received at least one dose of study drug and who had the modified mayo score clinical remission without the use of corticosteroids measured correctly at baseline. Participants were analysed per their assigned treatment arm regardless of the treatment they actually received.

End point type

Secondary

End point timeframe

Week 52

End point values	OL Maintenance Period: 50 mg Miri Subcutaneous (SC)	OL Maintenance Period: 100 mg Miri SC	OL Maintenance Period: 200 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/50 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/100 mg Miri SC	OL Maintenance Period: NR: 300 mg Miri IV /200 mg Miri SC
Number of subjects analysed	1	8	9	1	1	6
Units: percentage of participants
number (confidence interval 95%)	0.0 (0.0 to 79.3)	62.5 (30.6 to 86.3)	55.6 (26.7 to 81.1)	0.0 (0.0 to 79.3)	0.0 (0.0 to 79.3)	0.0 (0.0 to 39.0)

No statistical analyses for this end point

Secondary: Percentage of Participants in Clinical Remission Based on the Pediatric Ulcerative Colitis Activity Index (PUCAI)

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End point title

Percentage of Participants in Clinical Remission Based on the Pediatric Ulcerative Colitis Activity Index (PUCAI)

End point description

The PUCAI is a clinician-administered, 6-item questionnaire that measures: abdominal pain; RB; stool consistency; number of stools; nocturnal stools; and activity level. For PUCAI score all items are answered as an average over the ‘past 2 days’. A total disease activity score is calculated from 0 to 85, with Severe 65-85; Moderate:35-60; Mild:10-30, and None:<10. The clinician will record the participant or caregiver/legal guardian responses for the PUCAI electronically as source data in the tablet device at appropriate visits. PUCAI clinical remission is defined as a PUCAI score of <10 points. APD included mITT: All randomized participants who received at least one dose of study drug and who had the clinical remission based on the PUCAI measured correctly at baseline. Participants were analysed per their assigned treatment arm regardless of the treatment they actually received.

End point type

Secondary

End point timeframe

Week 52

End point values	OL Maintenance Period: 50 mg Miri Subcutaneous (SC)	OL Maintenance Period: 100 mg Miri SC	OL Maintenance Period: 200 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/50 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/100 mg Miri SC	OL Maintenance Period: NR: 300 mg Miri IV /200 mg Miri SC
Number of subjects analysed	1	8	9	1	1	6
Units: percentage of participants
number (confidence interval 95%)	0.0 (0.0 to 79.3)	75.0 (40.9 to 92.9)	77.8 (45.3 to 93.7)	0.0 (0.0 to 79.3)	0.0 (0.0 to 79.3)	0.0 (0.0 to 39.0)

No statistical analyses for this end point

Secondary: Percentage of Participants in Clinical Response Based on the PUCAI

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End point title

Percentage of Participants in Clinical Response Based on the PUCAI

End point description

PUCAI clinical response is defined as a reduction in baseline PUCAI score of ≥20 points. APD included mITT: All randomized participants who received at least one dose of study drug and who had the clinical response based on the PUCAI measured correctly at baseline. Participants were analysed per their assigned treatment arm regardless of the treatment they actually received.

End point type

Secondary

End point timeframe

Week 52

End point values	OL Maintenance Period: 50 mg Miri Subcutaneous (SC)	OL Maintenance Period: 100 mg Miri SC	OL Maintenance Period: 200 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/50 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/100 mg Miri SC	OL Maintenance Period: NR: 300 mg Miri IV /200 mg Miri SC
Number of subjects analysed	1	8	9	1	1	6
Units: percentage of participants
number (confidence interval 95%)	0.0 (0.0 to 79.3)	75.0 (40.9 to 92.9)	88.9 (56.5 to 98.0)	0.0 (0.0 to 79.3)	0.0 (0.0 to 79.3)	0.0 (0.0 to 39.0)

No statistical analyses for this end point

Secondary: Percentage of Participants in Endoscopic Remission

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End point title

Percentage of Participants in Endoscopic Remission

End point description

Endoscopic remission at week 52 is defined as achieving a Mayo endoscopic subscore of 0 or 1 (excluding friability) at Week 52. ES subscore is based on colonoscopy or sigmoidoscopy and scored from 0 (normal or inactive disease) to 3 (severe disease, spontaneous bleeding, ulceration). APD included mITT: All randomized participants who received at least one dose of study drug and who had the endoscopic remission measured correctly at baseline. Participants were analysed per their assigned treatment arm regardless of the treatment they actually received.

End point type

Secondary

End point timeframe

Week 52

End point values	OL Maintenance Period: 50 mg Miri Subcutaneous (SC)	OL Maintenance Period: 100 mg Miri SC	OL Maintenance Period: 200 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/50 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/100 mg Miri SC	OL Maintenance Period: NR: 300 mg Miri IV /200 mg Miri SC
Number of subjects analysed	1	8	9	1	1	6
Units: percentage of participants
number (confidence interval 95%)	0.0 (0.0 to 79.3)	62.5 (30.6 to 86.3)	55.6 (26.7 to 81.1)	0.0 (0.0 to 79.3)	0.0 (0.0 to 79.3)	0.0 (0.0 to 39.0)

No statistical analyses for this end point

Secondary: Percentage of Participants in Symptomatic Remission

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End point title

Percentage of Participants in Symptomatic Remission

End point description

Symptomatic remission at week 52 is defined as a Mayo score for RB=0, SF=0 or 1 with ≥ 1 point decrease from baseline. SF subscore, based on the participant's diary and scored from 0 (normal number of stools) to 3 (5 or more stools than normal). RB subscore, based on the participant's diary and scored from 0 (no blood seen) to 3 (blood alone passed). APD included mITT: All randomized participants who received at least one dose of study drug and who had the symptomatic remission measured correctly at baseline. Participants were analysed per their assigned treatment arm regardless of the treatment they actually received.

End point type

Secondary

End point timeframe

Week 52

End point values	OL Maintenance Period: 50 mg Miri Subcutaneous (SC)	OL Maintenance Period: 100 mg Miri SC	OL Maintenance Period: 200 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/50 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/100 mg Miri SC	OL Maintenance Period: NR: 300 mg Miri IV /200 mg Miri SC
Number of subjects analysed	1	8	9	1	1	6
Units: percentage of participants
number (confidence interval 95%)	0.0 (0.0 to 79.3)	75.0 (40.9 to 92.9)	66.7 (35.4 to 87.9)	0.0 (0.0 to 79.3)	0.0 (0.0 to 79.3)	0.0 (0.0 to 39.0)

No statistical analyses for this end point

Secondary: Height Velocity (in Centimeters/Year)

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End point title

Height Velocity (in Centimeters/Year)

End point description

Observed height velocity by gender and age group was calculated. Age groups for which this was summarized were 2 to <8, 8 to <12, and 12 to <18. Observed height velocity by gender and age group was calculated at baseline according to the following formula: (Present Height [cm] - Previous Height [cm])/Interval (months) Between Measurements × 12. 9999=Data Not Available (N/A) and individual values are provided. APD included mITT: All randomized participants who received at least one dose of study drug and who had the height velocity measured correctly at baseline. Participants were analysed per their assigned treatment arm regardless of the treatment they actually received.

End point type

Secondary

End point timeframe

Week 52

End point values	OL Induction and Maintenance Period: 5 mg/kg Miri IV	OL Induction and Maintenance Period: 10 mg/kg Miri IV	OL Induction and Maintenance Period: 300 mg Miri IV
Number of subjects analysed	10 ^[2]	5 ^[3]	11
Units: centimeter per year (cm/year)
arithmetic mean (standard deviation)
Female:2-<8 years (n=1,0,0)	9999 ± 9999	0 ± 0	0 ± 0
Female:8-<12 years (n=2,0,0)	8.65 ± 3.2	0 ± 0	0 ± 0
Female:12-<18 years (n=3,2,2)	4.54 ± 4.2	4.14 ± 1.3	2.97 ± 0.2
Male:2-<8 years (n=1,0,0)	9999 ± 9999	0 ± 0	0 ± 0
Male:8-<12 years (n=0,1,0)	0 ± 0	9999 ± 9999	0 ± 0
Male:12-<18 years (n=2,1,4)	12.67 ± 1.0	9999 ± 9999	3.65 ± 4.3

Notes
[2] - Female:2-<8 years: Individual value= 7.85; Male: 2 - <8 years: Individual value=7.72
[3] - Male: 8-<12 years, Male:12 - <8 years: Individual value= 3.64

No statistical analyses for this end point

Secondary: Change from Baseline in Body Weight

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End point title

Change from Baseline in Body Weight

End point description

Change from Baseline in body weight by gender and age group was calculated. 9999=Data Not Available (N/A) and individual values are provided. APD included mITT: All randomized participants who received at least one dose of study drug and who had the body weight measured correctly at baseline. Participants were analysed per their assigned treatment arm regardless of the treatment they actually received.

End point type

Secondary

End point timeframe

Baseline, Week 52

End point values	OL Induction and Maintenance Period: 5 mg/kg Miri IV	OL Induction and Maintenance Period: 10 mg/kg Miri IV	OL Induction and Maintenance Period: 300 mg Miri IV
Number of subjects analysed	10 ^[4]	5 ^[5]	11
Units: kg
arithmetic mean (standard deviation)
Female:2-<8 years(n=1,0,0)	9999 ± 9999	0 ± 0	0 ± 0
Female:8-<12 years(n=2,0,0)	8 ± 1.1	0 ± 0	0 ± 0
Female:12-<18 years(n=3,2,2)	9 ± 7.1	9 ± 3.5	8 ± 2.7
Male:2-<8 years(n=1,0,0)	9999 ± 9999	0 ± 0	0 ± 0
Male:8-<12 years(n=0,1,0)	0 ± 0	9999 ± 9999	0 ± 0
Male:12-<18 years(n=2,1,4)	13 ± 11.6	9999 ± 9999	2 ± 3.2

Notes
[4] - Female:2-<8 years: Individual value = 5.1; Male:2-<8 years: Individual value = 1.3
[5] - Male: 8 - <12 years: Individual value = 3; Male: 12 - <18 years: Individual value = 3.6

No statistical analyses for this end point

Secondary: Percentage of Participants with Histologic-Endoscopic Mucosal Remission

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End point title

Percentage of Participants with Histologic-Endoscopic Mucosal Remission

End point description

Histologic-endoscopic mucosal remission is defined as achieving both histologic remission and endoscopic remission. Histologic remission is defined as Geboes histological subscores of 0 for parameters: 2B (neutrophils in lamina propria), 3 (neutrophils in epithelium), 4 (crypt destruction), and 5 (erosion or ulceration). APD included mITT: All randomized participants who received at least one dose of study drug and who had the histologic-endoscopic mucosal remission measured correctly at baseline. Participants were analysed per their assigned treatment arm regardless of the treatment they actually received.

End point type

Secondary

End point timeframe

Week 52

End point values	OL Maintenance Period: 50 mg Miri Subcutaneous (SC)	OL Maintenance Period: 100 mg Miri SC	OL Maintenance Period: 200 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/50 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/100 mg Miri SC	OL Maintenance Period: NR: 300 mg Miri IV /200 mg Miri SC
Number of subjects analysed	1	8	9	1	1	6
Units: percentage of participants
number (confidence interval 95%)	0.0 (0.0 to 79.3)	62.5 (30.6 to 86.3)	44.4 (18.9 to 73.3)	0.0 (0.0 to 79.3)	0.0 (0.0 to 79.3)	0.0 (0.0 to 39.0)

No statistical analyses for this end point

Secondary: Change from baseline in 7-day average of Abdominal Pain Numeric Rating Scale (NRS) score at Week 12

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End point title

Change from baseline in 7-day average of Abdominal Pain Numeric Rating Scale (NRS) score at Week 12

End point description

The Abdominal Pain NRS is a single participant-reported item that measures the “worst abdominal pain in the past 24 hours” using a 6-point scale ranging from 0 (no pain) to 5 (worst possible pain) for 8-11 years old, and 11-point NRS ranging from 0 (no pain) to 10 (worst possible pain) for children < 8 years old as completed by a caregiver and those 12-17 years old. Abdominal pain NRS Score calculated by averaging data from all available daily diary entries of Abdominal Pain NRS for a 7 day period. A negative change from baseline indicates improvement in the participant's Abdominal Pain NRS. 9999=Data Not Available (N/A) and individual values are provided. APD included mITT: All randomized participants who received at least one dose of study drug and who had the 7-day average of Abdominal Pain NRS measured correctly at baseline. Participants were analysed per their assigned treatment arm regardless of the treatment they actually received.

End point type

Secondary

End point timeframe

Baseline, Week 12

End point values	OL Induction Period: 5 milligram per kilogram (mg/kg) Miri IV	OL Induction Period: 10 mg/kg Miri IV	OL Induction Period: 300 mg Miri IV
Number of subjects analysed	10	5 ^[6]	11
Units: score on a scale
arithmetic mean (standard deviation)
2-<8 years(n=3,0,0)	-3 ± 2.1	0 ± 0	0 ± 0
8-<12 years(n=2,1,0)	-3 ± 2.1	9999 ± 9999	0 ± 0
12-<18 years(n=5,3,10)	-4 ± 2.7	-2 ± 1.5	-1 ± 2.8

Notes
[6] - 8-<12 years: Individual value=-2

No statistical analyses for this end point

Secondary: Change From Baseline in 7-day Average of Abdominal Pain NRS Score at Week 52

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End point title

Change From Baseline in 7-day Average of Abdominal Pain NRS Score at Week 52

End point description

The Abdominal Pain NRS is a single participant-reported item that measures the "worst abdominal pain in the past 24 hours" using a 6-point scale ranging from 0 (no pain) to 5 (worst possible pain) for 8-11 years old, and 11-point NRS ranging from 0 (no pain) to 10 (worst possible pain) for children < 8 years old as completed by a caregiver and those 12-17 years old. Abdominal Pain NRS Score is calculated by averaging data from all available daily diary entries of abdominal pain NRS for a 7 day period. A negative change from baseline indicates improvement in the participant's Abdominal Pain NRS. 9999=Data Not Available (N/A) and individual values are provided. APD included all randomized participants who received at least one dose of study drug and who had the 7-day average of Abdominal Pain NRS measured correctly at baseline. Participants were analysed per their assigned treatment arm regardless of the treatment they actually received.

End point type

Secondary

End point timeframe

Baseline, Week 52

End point values	OL Maintenance Period: 50 mg Miri Subcutaneous (SC)	OL Maintenance Period: 100 mg Miri SC	OL Maintenance Period: 200 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/50 mg Miri SC	OL Maintenance Period: NR: 10 mg/kg Miri IV/100 mg Miri SC	OL Maintenance Period: NR: 300 mg Miri IV /200 mg Miri SC
Number of subjects analysed	1	8	9	1	1	6
Units: score on a scale
arithmetic mean (standard deviation)
2-<8 years(n=1,1,0,1,0,0)	9999 ± 9999	9999 ± 9999	0 ± 0	0 ± 0	0 ± 0	0 ± 0
8-<12 years(n=0,3,0,0,0,0)	0 ± 0	-2 ± 1.0	0 ± 0	0 ± 0	0 ± 0	0 ± 0
12-<18 years(n=0,3,8,0,1,6)	0 ± 0	-5 ± 3.1	-4 ± 2.4	0 ± 0	9999 ± 9999	0 ± 2.6

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

Induction Period (Up to 12 Weeks), Induction and Maintenance Period (Up to 52 Weeks)

Adverse event reporting additional description

All randomized participants who received at least one dose of study drug. Participants were analyzed according to the weight/treatment dose group to which they were assigned. Adverse event data is reported for the induction period and combined induction and maintenance periods as per planned analysis.

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

25.1

Reporting group title

OL Induction Period: 5 mg/kg Miri IV

Reporting group description

Participants (≤40 kg weight) received 5 mg/kg mirikizumab given as an IV infusion every 4 Q4W on weeks 0, 4, 8 for 12 weeks.

Reporting group title

OL Induction and Maintenance Period: 5 mg/kg Miri IV

Reporting group description

Reporting group title

OL Induction Period: 10 mg/kg Miri IV

Reporting group description

Participants (≤40 kg weight) received 10 mg/kg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks.

Reporting group title

OL Induction and Maintenance Period: 10 mg/kg Miri IV

Reporting group description

Reporting group title

OL Induction Period: 300 mg Miri IV

Reporting group description

Participants (>40 kg weight) received 300 mg mirikizumab given as an IV infusion Q4W on weeks 0, 4, 8 for 12 weeks.

Reporting group title

OL Induction and Maintenance Period: 300 mg Miri IV

Reporting group description

Serious adverse events	OL Induction Period: 5 mg/kg Miri IV	OL Induction and Maintenance Period: 5 mg/kg Miri IV	OL Induction Period: 10 mg/kg Miri IV	OL Induction and Maintenance Period: 10 mg/kg Miri IV	OL Induction Period: 300 mg Miri IV	OL Induction and Maintenance Period: 300 mg Miri IV
Total subjects affected by serious adverse events
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	2 / 5 (40.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
number of deaths (all causes)	0	0	0	0	0	0
number of deaths resulting from adverse events	0	0	0	0	0	0
Gastrointestinal disorders
appendicitis noninfective
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	1 / 5 (20.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences causally related to treatment / all	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
colitis ulcerative
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	1 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Musculoskeletal and connective tissue disorders
pseudarthrosis
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	1 / 5 (20.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences causally related to treatment / all	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

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

Non-serious adverse events	OL Induction Period: 5 mg/kg Miri IV	OL Induction and Maintenance Period: 5 mg/kg Miri IV	OL Induction Period: 10 mg/kg Miri IV	OL Induction and Maintenance Period: 10 mg/kg Miri IV	OL Induction Period: 300 mg Miri IV	OL Induction and Maintenance Period: 300 mg Miri IV
Total subjects affected by non serious adverse events
subjects affected / exposed	8 / 10 (80.00%)	9 / 10 (90.00%)	2 / 5 (40.00%)	5 / 5 (100.00%)	8 / 11 (72.73%)	11 / 11 (100.00%)
General disorders and administration site conditions
administration site oedema
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	1	1	0	0	0	0
catheter site induration
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	1	1	0	0	0	0
fatigue
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	1	1	0	0	1	1
feeling abnormal
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	0	0	0
infusion site reaction
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	1	1	0	0	0	0
injection site erythema
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	1 / 5 (20.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	1	0	0
injection site oedema
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	0	0	0
injection site pain
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	2 / 10 (20.00%)	0 / 5 (0.00%)	1 / 5 (20.00%)	0 / 11 (0.00%)	3 / 11 (27.27%)
occurrences all number	0	5	0	7	0	22
injection site pruritus
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	1 / 5 (20.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	1	0	0
injection site reaction
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	1 / 5 (20.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	2	0	2	0	0
non-cardiac chest pain
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	0	1
pyrexia
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	2 / 10 (20.00%)	2 / 10 (20.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	2 / 11 (18.18%)
occurrences all number	2	2	0	0	1	2
puncture site pain
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	1	2	0	0	0	0
puncture site oedema
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	0	0	0
Immune system disorders
immunosuppression
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	1	1
seasonal allergy
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	0	0	0
Reproductive system and breast disorders
dysmenorrhoea
alternative dictionary used: MedDRA 25.1
subjects affected / exposed ^[1]	0 / 6 (0.00%)	0 / 6 (0.00%)	0 / 3 (0.00%)	0 / 3 (0.00%)	1 / 6 (16.67%)	1 / 6 (16.67%)
occurrences all number	0	0	0	0	1	1
Respiratory, thoracic and mediastinal disorders
cough
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	1	1	0	0	0	0
rhinorrhoea
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	0	0	0
oropharyngeal pain
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	1 / 5 (20.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	1	1	0	1	0	0
pharyngeal swelling
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	0	1
throat irritation
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	1	1
Psychiatric disorders
insomnia
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	1	1
procedural anxiety
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	0	1
Investigations
alanine aminotransferase increased
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	2	3	0	0	0	0
aspartate aminotransferase increased
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	0	0	0
Injury, poisoning and procedural complications
arthropod sting
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	1	1	0	0	0	0
infusion related reaction
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	2	2
Nervous system disorders
burning sensation
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	1	1
dizziness
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	1	1
headache
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	3 / 10 (30.00%)	3 / 10 (30.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	2 / 11 (18.18%)
occurrences all number	7	8	0	0	1	2
migraine
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	0	0	0
tremor
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	1	1
Blood and lymphatic system disorders
anaemia
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	1 / 5 (20.00%)	1 / 5 (20.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	0	1	1	0	0
blood loss anaemia
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	1	1	0	0	0	0
Ear and labyrinth disorders
ear pain
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	0	1
motion sickness
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	0	0	0
Gastrointestinal disorders
abdominal pain upper
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences all number	1	1	0	0	0	1
abdominal discomfort
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	0	0	0
anal incontinence
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	1	1	0	0	0	0
constipation
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	1	2	0	0	1	1
dyspepsia
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	1 / 5 (20.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	0	0	1	0	0
food poisoning
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	1	1	0	0	0	0
gastritis
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	0	0	0
gastrooesophageal reflux disease
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	0	1
haemorrhoids
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	0	1
lip blister
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	1	1
nausea
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	2 / 11 (18.18%)
occurrences all number	0	1	0	0	1	2
vomiting
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	2 / 10 (20.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	1	4	0	0	1	1
Skin and subcutaneous tissue disorders
alopecia
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	1	1	0	0	0	0
dermatitis
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	1	1	0	0	0	0
rash maculo-papular
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	1	1
rash
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	2 / 10 (20.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	2 / 11 (18.18%)	2 / 11 (18.18%)
occurrences all number	0	2	0	0	2	2
pruritus
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	1 / 10 (10.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	1	1	0	0	0	0
urticaria
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	1	1
Renal and urinary disorders
pollakiuria
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	1 / 5 (20.00%)	1 / 5 (20.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	0	1	1	0	0
Endocrine disorders
growth hormone deficiency
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	1 / 5 (20.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	0	0	1	0	0
Musculoskeletal and connective tissue disorders
back pain
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	0	1
arthralgia
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	1 / 11 (9.09%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	1	1
fracture nonunion
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	1 / 5 (20.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	0	0	1	0	0
muscle spasms
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	2 / 11 (18.18%)
occurrences all number	0	0	0	0	0	3
Infections and infestations
covid-19
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	2 / 10 (20.00%)	1 / 5 (20.00%)	2 / 5 (40.00%)	0 / 11 (0.00%)	2 / 11 (18.18%)
occurrences all number	0	2	1	2	0	2
asymptomatic covid-19
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	2 / 11 (18.18%)
occurrences all number	0	1	0	0	0	2
gastroenteritis
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	0	0	0
impetigo
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	0	0	0
oral herpes
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	0	1
sinusitis
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	0	1
viral upper respiratory tract infection
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	2 / 10 (20.00%)	0 / 5 (0.00%)	1 / 5 (20.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	2	0	1	0	1
upper respiratory tract infection
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1	0	0	0	1
tooth abscess
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	1 / 10 (10.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	0 / 11 (0.00%)
occurrences all number	0	1	0	0	0	0
tinea infection
alternative dictionary used: MedDRA 25.1
subjects affected / exposed	0 / 10 (0.00%)	0 / 10 (0.00%)	0 / 5 (0.00%)	0 / 5 (0.00%)	0 / 11 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	0	0	0	0	1

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: Gender specific events only occurring in male or female participants have had the number of participants at Risk adjusted accordingly.

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Were there any global substantial amendments to the protocol? Yes

26 Aug 2019

- Updated the definition of mucosal healing; - Exploratory endpoint has been added; -Removed text referring to randomization; - Clarification provided in schedule of activities; - Updated intestinal dysplasia language; - Added description of UCEIS; - Inclusion and exclusion criteria were revised; - Clarifications on treatments administered, physical examination, clinical laboratory tests, prohibited medications, Permitted medications with dose stabilization.

30 Apr 2021

- Secondary endpoint has been updated; - Clarification provided in schedule of activities; - Updated age for hormone collection; - Clarified overall design; wording; - Inclusion and exclusion criteria were revised; - Clarifications on treatments administered, physical examination, clinical laboratory tests, prohibited medications.

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 Mulitcenter, Open-Label PK Study of Mirikizumab in Pediatric Patients with Moderately to Severely Active Ulcerative Colitis

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Pharmacokinetics (PK): Clearance of Mirikizumab

Primary: Pharmacokinetics (PK): Clearance of Mirikizumab

Secondary: Percentage of Participants in Clinical Remission

Secondary: Percentage of Participants in Clinical Remission

Secondary: Percentage of Participants in Clinical Response

Secondary: Percentage of Participants in Clinical Response

Secondary: Percentage of Participants Who are in MMS Clinical Remission Without the Use of Corticosteroids

Secondary: Percentage of Participants Who are in MMS Clinical Remission Without the Use of Corticosteroids

Secondary: Percentage of Participants in Clinical Remission Based on the Pediatric Ulcerative Colitis Activity Index (PUCAI)

Secondary: Percentage of Participants in Clinical Remission Based on the Pediatric Ulcerative Colitis Activity Index (PUCAI)

Secondary: Percentage of Participants in Clinical Response Based on the PUCAI

Secondary: Percentage of Participants in Clinical Response Based on the PUCAI

Secondary: Percentage of Participants in Endoscopic Remission

Secondary: Percentage of Participants in Endoscopic Remission

Secondary: Percentage of Participants in Symptomatic Remission

Secondary: Percentage of Participants in Symptomatic Remission

Secondary: Height Velocity (in Centimeters/Year)

Secondary: Height Velocity (in Centimeters/Year)

Secondary: Change from Baseline in Body Weight

Secondary: Change from Baseline in Body Weight

Secondary: Percentage of Participants with Histologic-Endoscopic Mucosal Remission

Secondary: Percentage of Participants with Histologic-Endoscopic Mucosal Remission

Secondary: Change from baseline in 7-day average of Abdominal Pain Numeric Rating Scale (NRS) score at Week 12

Secondary: Change from baseline in 7-day average of Abdominal Pain Numeric Rating Scale (NRS) score at Week 12

Secondary: Change From Baseline in 7-day Average of Abdominal Pain NRS Score at Week 52

Secondary: Change From Baseline in 7-day Average of Abdominal Pain NRS Score at Week 52

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

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Clinical Trial Results:
A Mulitcenter, Open-Label PK Study of Mirikizumab in Pediatric Patients with Moderately to Severely Active Ulcerative Colitis