A Study Investigating Oral Ozanimod (RPC1063) in Pediatric Participants With Moderate to Severe Active Ulcerative Colitis

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the effectiveness and safety of ozanimod (RPC1063) in achieving and maintaining clinical remission. Ozanimod will be administered orally to pediatric participants with moderate to severe active ulcerative colitis (UC) who have had an inadequate response to conventional therapy.

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 Subjects With Moderately to Severely Active Ulcerative Colitis With an Inadequate Response to Conventional Therapy

Conditions
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Colitis, Ulcerative

Intervention / Treatment
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* Drug: Ozanimod


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Moderately to severely active Ulcerative Colitis (UC) diagnosed prior to the Screening Visit Evidence of UC extending beyond the rectum, as determined by baseline endoscopy Has had an inadequate response, loss of response to, or is intolerant to at least 1 of the following treatments for UC: oral aminosalicylates, systemic corticosteroids, immunomodulators, biologic therapy Exclusion Criteria: Diagnosis of Crohn's disease or indeterminate colitis Has documentation of positive test for toxin producing Clostridium difficile, or polymerase chain reaction examination of the stool Apheresis within 2 weeks of randomization History of or currently active primary or secondary immunodeficiency, or participants with known genetic disorders as a cause for colitis 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 High Dose<br> | Drug: Ozanimod<br>* Specified dose on specified days<br>* Other names: RPC1063;|
| Experimental: Ozanimod Low Dose<br> | Drug: Ozanimod<br>* Specified dose on specified 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 clinical remission |  | At Week 52 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of participants who achieve clinical remission |  | At Week 10 | 
| Proportion of participants who achieve clinical response |  | At Week 52 | 
| Proportion of participants who achieve clinical response |  | At Week 10 | 
| Proportion of participants who achieve symptomatic remission |  | At Week 10 and Week 52 | 
| Time to achievement of symptomatic remission |  | Up to 6 years | 
| Proportion of participants who achieve endoscopic improvement |  | At Week 10 and Week 52 | 
| Proportion of participants who achieve corticosteroid free remission |  | At Week 52 | 
| Incidence of Adverse Events (AEs) |  | Up to 6 years | 
| Incidence of Serious Adverse Events |  | Up to 6 years | 
| Incidence of AEs leading to discontinuation from treatment |  | Up to 6 years | 
| Incidence of AEs of special interest (AESIs) |  | Up to 6 years | 
| Steady state systemic exposure of ozanimod and CC112273 |  | At Week 18 and throughout the study, up to 70 weeks | 
| Absolute change from baseline in Absolute Lymphocyte Count (ALC) |  | Up to 6 years | 
| Percent change from baseline in ALC |  | Up to 6 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Ulcerative Colitis, Ozanimod, Pediatric