A Study to Evaluate the Efficacy, Safety, and Drug Levels of Oral Ozanimod in Pediatric Participants With Moderately to Severely Active Crohn's Disease With an Inadequate Response to Conventional Therapy

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the efficacy, safety, drug levels, and drug effects of ozanimod in pediatric participants with moderately to severely active Crohn's Disease.

Official Title
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A Phase 2/3, Multicenter, Randomized, Double-blind Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Oral Ozanimod (RPC1063) in Pediatric Participants With Moderately to Severely Active Crohn's Disease With an Inadequate Response to Conventional Therapy

Conditions
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Crohn Disease

Intervention / Treatment
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* Drug: Ozanimod


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participants must have a Pediatric Crohn's Disease Activity Index (PCDAI) score ≥ 30 and a Simple Endoscopic Score for Crohn's Disease (SES-CD) score ≥ 6 (or SES-CD ≥ 4 in participants with isolated ileal disease) Participant has an inadequate response, intolerance, or loss of response to at least 1 of the following treatments for Crohn's Disease (CD): i) corticosteroids ii) immunomodulators iii) biologic therapy iv) other systemic immunomodulatory therapies for CD Exclusion Criteria: Participant is likely to require, in the physician's judgment, bowel resection within 12 weeks of entry into the study Participant has current stoma, ileal-anal pouch anastomosis, fistula that is likely to require, in the physician's judgment, surgical or medical intervention within 12 weeks of entry into the study or need for ileostomy or colostomy Participant has extensive small bowel resection (> 100 cm) or known diagnosis of short bowel syndrome or participant requires total parenteral nutrition Other protocol-defined inclusion/exclusion criteria apply.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Ozanimod Dose Level 1<br> | Drug: Ozanimod<br>* Specific dose on specific days<br>* Other names: RPC1063;|
| Experimental: Ozanimod Dose Level 2<br> | Drug: Ozanimod<br>* Specific dose on specific days<br>* Other names: RPC1063;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of participants who achieve Pediatric Crohn's Disease Activity Index (PCDAI) < 10 |  | At week 64 | 
| Proportion of participants achieving Simple Endoscopic Score for Crohn's Disease (SES-CD) ≤ 2 or SES-CD ≤ 4 points with no SES-CD subscore > 1 point |  | At week 64 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of participants who achieve PCDAI < 10 |  | At week 12 | 
| Proportion of participants who achieve SES-CD decrease from Baseline of ≥ 50% (ER-50) |  | At week 12 and week 64 | 
| Proportion of participants who achieve reduction in PCDAI score ≥ 12.5 |  | At week 12 and week 64 | 
| Proportion of participants who achieve total PCDAI score of < 30 points |  | At week 12 and week 64 | 
| Proportion of adolescents who achieve an average daily abdominal pain score ≤ 1 point |  | At week 12 and week 64 | 
| Proportion of adolescents who achieve an average daily stool frequency ≤ 3 points with abdominal pain |  | At week 12 and week 64 | 
| Proportion of adolescents who achieve stool frequency no worse than Baseline |  | At week 12 and week 64 | 
| Change from Baseline in stool frequency score over time |  | Up to 81 weeks | 
| Change from baseline in abdominal pain over time measured by Pediatric Crohn's Disease Activity Index (graded from 0-10) |  | Up to 81 weeks | 
| Proportion of adolescents who achieve Crohn's Disease Activity Index (CDAI) score < 150 |  | At week 12 and week 64 | 
| Proportion of adolescents who achieve CDAI reduction from Baseline of ≥ 100 points or CDAI score < 150 |  | At week 12 and week 64 | 
| Proportion of participants who achieve a PCDAI score < 10 while remaining corticosteroid free in the prior 12 weeks |  | At week 64 | 
| Proportion of participants who achieve a CDAI score < 150 while remaining corticosteroid free in the prior 12 weeks (adolescents only) |  | At week 64 | 
| Proportion of participants achieving SES-CD ≤ 2 or SES-CD ≤ 4 points with no SES-CD subscore > 1 point |  | At week 12 | 
| Steady state systemic exposures of ozanimod |  | At week 20 and up to 81 weeks | 
| Steady state systemic exposures of CC112273 |  | At week 20 and up to 81 weeks | 
| Absolute change from Baseline in absolute lymphocyte count (ALC) |  | At week 12, week 64, and up to 81 weeks | 
| Percent change from Baseline in ALC |  | At week 12, week 64, and up to 81 weeks | 
| Number of participants with Adverse Events (AEs) |  | Up to 81 weeks | 
| Number of participants with Serious Adverse Events (SAEs) |  | Up to 81 weeks |