Safety and Efficacy Trial of RPC1063 for Moderate to Severe Ulcerative Colitis

Study Overview
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Brief Summary
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The purpose of this study is to determine whether RPC1063 is effective in the treatment of Ulcerative Colitis (UC).

Detailed Description
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The trial is composed of 2 periods: Induction and Maintenance. In the Induction Period (IP), patients will be entered into the trial in 2 separate cohorts (Cohort 1 and Cohort 2).Patients from Cohort 1 and 2 in clinical response at the end of the IP will proceed through to the Maintenance Period (MP). Patients from Cohort 1 and 2 who participate in this trial may also qualify to participate in an optional Open-Label Extension (OLE) trial.

Official Title
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A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled Trial of Oral RPC1063 as Induction and Maintenance Therapy for Moderate to Severe Ulcerative Colitis

Conditions
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Ulcerative Colitis

Intervention / Treatment
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* Drug: RPC1063
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Aged 18 to 75 years (at screening for Cohort 1 and 2) UC confirmed on endoscopy Moderately to severely active UC (May score 6-12) Currently receiving treatment with aminosalisylate, prednisone, or budesonide Can be receiving azathioprine, mercaptopurine, or methotrexate, but treatment will be stopped prior to randomization Exclusion Criteria: Have severe extensive colitis as evidence by: Physician judgment that the patient is likely to require colectomy or ileostomy within 12 weeks of baseline. Current or recent (within 3 months) evidence of fulminant colitis, toxic megacolon, or bowel perforation. Diagnosis of CD, indeterminate colitis, or the presence of fistula consistent with CD or microscopic colitis, radiation colitis, or ischemic colitis Clinically relevant cardiovascular conditions or other relevant diseases that could impact the implementation or interpretation of the trial, or put the patient at risk History of uveitis or unknown macular edema Pregnancy, lactation, or a positive serum β-human chorionic gonadotropin (β-hCG) measured during screening

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 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: RPC1063 (Ozanimod)<br>1mg, daily oral administration during Induction and Maintenance periods. | Drug: RPC1063<br> <br> |
| Placebo Comparator: Placebo<br>Daily oral administration during Induction and Maintenance periods. | Drug: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants in Clinical Remission at 10 Weeks | Percentage of participants that are in Clinical remission at 10 weeks | At 10 Weeks | 
| Percentage of Participants in Clinical Remission at 52 Weeks | Percentage of participants that are in Clinical remission at 52 weeks | At 52 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants With Clinical Response at 10 Weeks | Percentage of participants that are in Clinical response at 10 weeks | At 10 Weeks | 
| Percentage of Participants With Endoscopic Improvement at 10 Weeks | Percentage of participants with endoscopic improvement at 10 weeks | At 10 Weeks | 
| Percentage of Participants With Mucosal Healing at 10 Weeks | Percentage of participants with mucosal healing at 10 weeks | At 10 Weeks | 
| Percentage of Participants in Clinical Response at 52 Weeks | Percentage of participants that are in Clinical response at 52 weeks | At 52 Weeks | 
| Percentage of Participants With Endoscopic Improvement at 52 Weeks | Percentage of participants with endoscopic improvement at 52 weeks | At 52 Weeks | 
| Percentage of Participants in Clinical Remission at Week 52 Who Were in Remission at Week 10 | Percentage of participants in clinical remission at week 52 who were in clinical remission at week 10 | At 52 Weeks | 
| Percentage of Participants With Corticosteroid Free Remission at 52 Weeks | Percentage of participants with corticosteroid free remission at 52 weeks | At 52 Weeks | 
| Percentage of Participants With Mucosal Healing at 52 Weeks | Percentage of participants with Mucosal Healing at 52 weeks | At 52 Weeks | 
| Percentage of Participants With Durable Clinical Remission at 52 Weeks | Percentage of participants with durable clinical remission at 52 weeks | At 52 Weeks |