An Extension Study of RPC1063 as Therapy for Moderate to Severe Ulcerative Colitis

A Phase 3, Multicenter, Open-Label Extension Trial of Oral RPC1063 as Therapy for Moderate to Severe Ulcerative Colitis

The purpose of this study is to evaluate the long-term safety and efficacy of RPC1063 in participants with moderately to severely active ulcerative colitis. Only those participants who have previously participated in a trial of RPC1063, being either RPC01-3101 or completed at least 1 year of the open-label period of RPC01-202 will be eligible.

Study Overview

• Status: Completed
• Conditions: Ulcerative Colitis
• Intervention / Treatment: Drug: RPC1063

Detailed Description

This is an extension study trial. Eligible participants from the RPC01-3101 and RPC01-202 trials were able to roll-over in this trial to receive study medication until March 2023 or until the Sponsor discontinues the development program, whichever comes first.

• Study Type: Interventional
• Enrollment (Actual): 877
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Camperdown, New South Wales, Australia, 2050
      • Local Institution - 152
• Belarus
  • Vitebsk, Belarus, 210037
    • Local Institution - 751
• Belgium
  • Ghent, Belgium, 9000
    • Local Institution - 600
  • Leuven, Belgium, 3000
    • Local Institution - 601
• Bulgaria
  • Sofia, Bulgaria, 1336
    • Local Institution - 459
  • Sofia, Bulgaria, 1233
    • Local Institution - 450
  • Sofia, Bulgaria, 1606
    • Local Institution - 451
• Canada
  • Ontario
    • Lindsay, Ontario, Canada, K9V 5G6
      • Local Institution - 264
• Croatia
  • Osijek, Croatia, 31000
    • Local Institution - 611
• Czechia
  • Klatovy, Czechia, 399 01
    • Local Institution - 342
  • Prague, Czechia, 14021
    • Local Institution - 337
• Germany
  • Berlin, Germany, 13353
    • Local Institution - 525
  • Berlin, Germany, 12200
    • Local Institution - 535
  • Frankfurt, Germany, 60594
    • Local Institution - 545
• Greece
  • Athens, Greece, 10676
    • Local Institution - 643
• Hungary
  • Balatonfüred, Hungary, 8230
    • Local Institution - 816
  • Debrecen, Hungary, 4032
    • Local Institution - 808
• Israel
  • Rehovot, Israel, 76100
    • Local Institution - 505
• Italy
  • Tuscany
    • Florence, Tuscany, Italy, 50134
      • Local Institution - 567
• Moldova
  • Chisinau, Moldova, MD-2068
    • Local Institution - 658
  • Chisinau, Moldova, MD2025
    • Local Institution - 659
• Poland
  • Kłodzko, Poland, 57-300
    • Local Institution - 425
• Romania
  • Bucharest, Romania, 050098
    • Local Institution - 673
  • Bucharest, Romania, 010719
    • Local Institution - 677
• Slovakia
  • Bardejov, Slovakia, 08501
    • Local Institution - 910
• South Korea
  • Seongnam-si, South Korea, 13620
    • Local Institution - 364
  • Wŏnju, South Korea, 26426
    • Local Institution - 354
• Ukraine
  • Kharkiv, Ukraine, 61039
    • Local Institution - 954
  • Vinnytsia, Ukraine, 21018
    • Local Institution - 957
• United Kingdom
  • London, United Kingdom, SE1 9RT
    • Local Institution - 243
  • GREATER LONDON
    • London, GREATER LONDON, United Kingdom, E11 1NR
      • Local Institution - 236
• United States
  • Arizona
    • Tucson, Arizona, United States, 85712
      • Local Institution - 144
  • California
    • Anaheim, California, United States, 92801
      • Local Institution - 102
    • Mission Hills, California, United States, 91345
      • Local Institution - 117
  • Illinois
    • Oak Lawn, Illinois, United States, 60453
      • Local Institution - 112
  • Louisiana
    • Baton Rouge, Louisiana, United States, 70809
      • Local Institution - 119
  • North Carolina
    • Jacksonville, North Carolina, United States, 28546
      • Local Institution - 127
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73112
      • Local Institution - 290
  • Oregon
    • Portland, Oregon, United States, 97239
      • Local Institution - 143
  • Tennessee
    • Germantown, Tennessee, United States, 38138
      • Local Institution - 179
  • Texas
    • Dallas, Texas, United States, 75246
      • Local Institution - 122

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

For more information regarding Bristol-Myers Squibb Clinical Trial participation, please visit: www.BMSStudyConnect.com

Inclusion Criteria:

• Previously participated in a trial of RPC1063 and meets the criteria for participation in the open-label extension as outlined in the prior trial

Exclusion Criteria:

• Receiving treatment with breast cancer resistance protein inhibitors
• Clinically relevant cardiovascular conditions
• Liver function impairment

Other protocol-defined inclusion/exclusion criteria apply

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: RPC0163 (Ozanimod)	Drug: RPC1063; Other Names: Ozanimod

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Experiencing Treatment-Emergent Adverse Event (TEAEs)	Number of participants experiencing TEAEs, Serious TEAEs, TEAEs leading to discontinuation and TEAEs of special interest. TEAE is defined as any event with an onset date on or after the first dose date, or any ongoing event on the first dose date that worsens in severity on or after the first dose date, and until 90 days following the last dose of treatment with the study drug.	From first dose to 90 days post last dose (Up to approximately 92 months)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants With Clinical Remission	Clinical Remission is defined as Rectal bleeding subscore=0; stool frequency subscore <=1 (and a decrease of >=1 point from the baseline stool frequency subscore); and endoscopy subscore <=1. Rectal bleeding 0 = No blood seen Stool frequency 0 = Normal number of stools for this patient; 1 = 1 to 2 stools more than normal Endoscopy 0 = Normal or inactive disease; 1 = Mild disease (erythema, decreased vascular pattern, does not include friability).	Week 46, 94, 142, 190, 238
Percentage of Participants With Clinical Response	Clinical response is defined as reduction from baseline in the 9-point Mayo score of >=2 points and reduction from baseline in the 9-point Mayo score >=35%, and (reduction from baseline in the rectal bleeding subscore of >=1 point or a rectal bleeding subscore of <=1 point). 9 point Mayo score is defined as the sum of rectal bleeding subscore, stool frequency subscore, and the endoscopy subscore each ranging from (0=normal activity-3=worse activity) for a total score that ranges from 0 to 9 with higher score indicating worsening symptoms.	Week 46, 94, 142, 190, 238
Percentage of Participants With Endoscopic Improvement	Endoscopic Improvement is defined as endoscopy subscore of <=1 point. 0 = Normal or inactive disease; = Mild disease (erythema, decreased vascular pattern, does not include friability); = Moderate disease (marked erythema, lack of vascular pattern, friability, erosions); = Severe disease (spontaneous bleeding, ulceration)	Week 46, 94, 142, 190, 238
Percentage of Participants With Corticosteroid-free Remission	Corticosteroid-free remission is defined as clinical remission while off corticosteroids for ≥12 weeks. Clinical Remission is defined as Rectal bleeding subscore=0; stool frequency subscore <=1 (and a decrease of >=1 point from the baseline stool frequency subscore); and endoscopy subscore <=1. Rectal bleeding 0 = No blood seen Stool frequency 0 = Normal number of stools for this patient; 1 = 1 to 2 stools more than normal Endoscopy 0 = Normal or inactive disease; 1 = Mild disease (erythema, decreased vascular pattern, does not include friability).	Week 46, 94, 142, 190, 238
Percentage of Participants With Histologic Remission	Histologic remission is defined as Geboes index score < 2.0. The Geboes score is a validated histological grading system for UC that ranges from 0=no disease activity to 5=high disease activity.	Week 46, 94, 142, 190, 238
Percentage of Participants With Mucosal Healing	Mucosal Healing is defined as Endoscopy subscore of ≤ 1 point (0 = Normal or inactive disease; 1 = Mild disease (erythema, decreased vascular pattern, does not include friability)) and a Geboes index score < 2.0 (The Geboes score is a validated histological grading system for UC that ranges from 0=no disease activity to 5=high disease activity.)	Week 46, 94, 142, 190, 238
Change From Baseline in Complete Mayo Score	The Complete Mayo Score is a composite of four assessments, each rated from 0 to 3: Stool frequency: (0 = Normal number of stools for this patient; 1 = 1 to 2 stools more than normal; 2 = 3 to 4 stools more than normal; 3 = 5 or more stools more than normal); Rectal bleeding (0 = No blood seen; 1 = Streaks of blood with stool less than half the time; 2 = Obvious blood with stool most of the time; 3 = Blood alone passes); endoscopy (0 = Normal or inactive disease; 1 = Mild disease; 2 = Moderate disease; 3 = Severe disease); Physician's Global Assessment (0 = Normal; 1 = Mild disease; 2 = Moderate disease; 3 = Severe disease). The total score range is from 0-12, with higher score indicating worse disease activity. Baseline is the last measurement collected on or prior to the date of first dose in the 3102 OLE study.	Baseline, week 46, 94, 142, 190, 238, 286, 334, 382
Change From Baseline in Partial Mayo Score	The Partial Mayo Score is a composite of three assessments, each rated from 0 to 3: Stool frequency: (0 = Normal number of stools for this patient; 1 = 1 to 2 stools more than normal; 2 = 3 to 4 stools more than normal; 3 = 5 or more stools more than normal); Rectal bleeding (0 = No blood seen; 1 = Streaks of blood with stool less than half the time; 2 = Obvious blood with stool most of the time; 3 = Blood alone passes); Physician's Global Assessment (0 = Normal; 1 = Mild disease; 2 = Moderate disease; 3 = Severe disease). The total score range is from 0-9, with higher score indicating worse disease activity. Baseline is the last measurement collected on or prior to the date of first dose in the 3102 OLE study.	Baseline, week 46, 94, 142, 190, 238, 286, 334, 382
Change From Baseline in 9-Point Mayo Score	The 9-point Mayo Score is a composite of three assessments, each rated from 0 to 3: Stool frequency: (0 = Normal number of stools for this patient; 1 = 1 to 2 stools more than normal; 2 = 3 to 4 stools more than normal; 3 = 5 or more stools more than normal); Rectal bleeding (0 = No blood seen; 1 = Streaks of blood with stool less than half the time; 2 = Obvious blood with stool most of the time; 3 = Blood alone passes); Endoscopy (0 = Normal or inactive disease; 1 = Mild disease; 2 = Moderate disease; 3 = Severe disease) The total score range is from 0-9, with higher score indicating worse disease activity. Baseline is the last measurement collected on or prior to the date of first dose in the 3102 OLE study.	Baseline, week 46, 94, 142, 190, 238, 286, 334, 382
Percentage of Participants Who Had Previously Received Anti-TNF Therapy With Clinical Remission	Clinical Remission is defined as Rectal bleeding subscore=0; stool frequency subscore <=1 (and a decrease of >=1 point from the baseline stool frequency subscore); and endoscopy subscore <=1. Rectal bleeding 0 = No blood seen Stool frequency 0 = Normal number of stools for this patient; 1 = 1 to 2 stools more than normal Endoscopy 0 = Normal or inactive disease; 1 = Mild disease (erythema, decreased vascular pattern, does not include friability).	Week 46, 94, 142, 190, 238
Percentage of Participants Who Had Previously Received Anti-TNF Therapy With Clinical Response	Clinical response is defined as reduction from baseline in the 9-point Mayo score of >=2 points and reduction from baseline in the 9-point Mayo score >=35%, and (reduction from baseline in the rectal bleeding subscore of >=1 point or a rectal bleeding subscore of <=1 point). 9 point Mayo score is defined as the sum of rectal bleeding subscore, stool frequency subscore, and the endoscopy subscore each ranging from (0=normal activity-3=worse activity) for a total score that ranges from 0 to 9 with higher score indicating worsening symptoms.	Week 46, 94, 142, 190, 238
Percentage of Participants Who Had Previously Received Anti-TNF Therapy With Endoscopic Improvement	Endoscopic Improvement is defined as endoscopy subscore of <=1 point. 0 = Normal or inactive disease; 1 = Mild disease (erythema, decreased vascular pattern, does not include friability)	Week 46, 94, 142, 190, 238

Collaborators and Investigators

• Sponsor: Celgene
• Investigators: Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

General Publications

• Sandborn WJ, Feagan BG, Hanauer S, Vermeire S, Ghosh S, Liu WJ, Petersen A, Charles L, Huang V, Usiskin K, Wolf DC, D'Haens G. Long-Term Efficacy and Safety of Ozanimod in Moderately to Severely Active Ulcerative Colitis: Results From the Open-Label Extension of the Randomized, Phase 2 TOUCHSTONE Study. J Crohns Colitis. 2021 Jul 5;15(7):1120-1129. doi: 10.1093/ecco-jcc/jjab012.
• Yarur A, Irving P, Siegmund B, Dubinsky MC, Ananthakrishnan AN, Regueiro M, Ungaro RC, Ritter T, Nakase H, Liu Z, Mehra D, Osterman MT, Jain A, Rubin DT, Hibi T. Long-Term Ozanimod Therapy in Patients With Moderately Active Ulcerative Colitis After Failure of 5-Aminosalicylic Acid. Inflamm Bowel Dis. 2025 Sep 26:izaf195. doi: 10.1093/ibd/izaf195. Online ahead of print.
• Sands BE, Rubin DT, Loftus EV Jr, Wolf DC, Panaccione R, Colombel JF, Dignass A, Regueiro M, Vermeire S, Afzali A, Lawlor G, Ahmad HA, Wu H, Osterman MT, Jain A, D'Haens G. Impact of Prior Biologic Exposure on Ozanimod Efficacy and Safety in the Phase 3 True North Clinical Trial. Am J Gastroenterol. 2025 Oct 1;120(10):2339-2349. doi: 10.14309/ajg.0000000000003310. Epub 2025 Jan 8.
• Regueiro M, Siegmund B, Horst S, Moslin R, Charles L, Petersen A, Tatosian D, Wu H, Lawlor G, Fischer M, D'Haens G, Colombel JF. Concomitant Administration of Ozanimod and Serotonergic Antidepressants in Patients With Ulcerative Colitis or Relapsing Multiple Sclerosis. Inflamm Bowel Dis. 2025 Apr 10;31(4):1010-1017. doi: 10.1093/ibd/izae136.
• Sands BE, D'Haens G, Panaccione R, Regueiro M, Ghosh S, Hudesman D, Ahmad HA, Mehra D, Wu H, Jain A, Petersen A, Osterman MT, Afzali A, Danese S. Ozanimod in Patients With Moderate to Severe Ulcerative Colitis Naive to Advanced Therapies. Clin Gastroenterol Hepatol. 2024 Oct;22(10):2084-2095.e4. doi: 10.1016/j.cgh.2024.03.042. Epub 2024 May 8.
• Armuzzi A, Cross RK, Lichtenstein GR, Hou J, Deepak P, Regueiro M, Wolf DC, Akukwe L, Ahmad HA, Jain A, Kozinn M, Wu H, Petersen A, Charles L, Long M. Cardiovascular Safety of Ozanimod in Patients With Ulcerative Colitis: True North and Open-Label Extension Analyses. Clin Gastroenterol Hepatol. 2024 May;22(5):1067-1076.e3. doi: 10.1016/j.cgh.2023.11.018. Epub 2023 Nov 30.

Helpful Links

• BMS Clinical Trial Information
• BMS Clinical Trial Patient Recruiting

Study record dates

Study Major Dates

• Study Start (Actual): December 2, 2015
• Primary Completion (Actual): December 19, 2024
• Study Completion (Actual): December 19, 2024

Study Registration Dates

• First Submitted: August 20, 2015
• First Submitted That Met QC Criteria: August 21, 2015
• First Posted (Estimated): August 24, 2015

Study Record Updates

• Last Update Posted (Actual): December 31, 2025
• Last Update Submitted That Met QC Criteria: December 10, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Colonic Diseases; Gastroenteritis; Inflammatory Bowel Diseases; Colitis; Colitis, Ulcerative; Sphingosine 1 Phosphate Receptor Modulators; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; ozanimod
• Other Study ID Numbers: RPC01-3102; 2015-001600-64 (EudraCT Number); U1111-1218-0284 (Registry Identifier: WHO)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No