Oral Ozanimod for Pediatric Ulcerative Colitis

Recruiting at 137 trial locations

Overseen By BMS Study Connect Contact Center www.BMSStudyConnect.com

Age: < 18

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Bristol-Myers Squibb

Disqualifiers: Crohn's disease, Immunodeficiency, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 8-12 weeks

120 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called ozanimod (also known as RPC1063) to determine if it can help children with moderate to severe ulcerative colitis feel better and maintain remission. The medication is taken orally, and the study will evaluate both low and high doses to identify the most effective option. Children suitable for this trial have been diagnosed with ulcerative colitis affecting more than just the rectum and have not responded to other treatments like certain pills or injections. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to important findings.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, it mentions that participants should have had an inadequate response to certain treatments, which might imply that you can continue them. Please consult with the trial coordinators for specific guidance.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that ozanimod has been tested for safety in people with ulcerative colitis (UC) and multiple sclerosis (MS). Studies have found that ozanimod is generally well-tolerated, with most people not experiencing serious side effects. In one study, some participants experienced side effects after starting treatment, occurring at a rate of 81.1 per 100 person-years, while serious side effects were less common, at 65.0 per 100 person-years.

The FDA has already approved ozanimod for treating relapsing forms of MS, indicating its safety for use in humans for that condition. These findings suggest it might also be safe for treating pediatric UC, although individual experiences can vary. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for ulcerative colitis, which typically include anti-inflammatory drugs like mesalamine or immune system suppressors such as corticosteroids, Ozanimod offers a unique approach. Ozanimod works by selectively modulating the S1P receptors, which play a role in immune cell movement, potentially reducing inflammation more precisely. Researchers are excited because this targeted action might lead to fewer side effects compared to broader immunosuppressive therapies. Additionally, Ozanimod is taken orally, making it a convenient alternative to treatments that require injections or infusions.

What evidence suggests that ozanimod might be an effective treatment for ulcerative colitis?

Research has shown that ozanimod may help treat ulcerative colitis (UC). In one study, 71.2% of patients experienced some improvement, and 54.7% had no symptoms by week 56, indicating significant relief. Another study found that 20.9% of patients had no symptoms by week 32. This trial will test two different doses of ozanimod, a low dose and a high dose, to evaluate its effectiveness in relieving UC symptoms. ^{1 2 4 6 7}

Who Is on the Research Team?

Bristol-Myers Squibb

Principal Investigator

Bristol-Myers Squibb

Are You a Good Fit for This Trial?

This trial is for children with moderate to severe active ulcerative colitis who haven't responded well to conventional treatments like oral aminosalicylates, corticosteroids, immunomodulators, or biologics. They must have UC beyond the rectum confirmed by endoscopy. Kids with positive tests for C. difficile, recent apheresis, immune deficiencies, genetic disorders causing colitis, or diagnosed with Crohn's disease can't participate.

Inclusion Criteria

I have been diagnosed with moderate to severe Ulcerative Colitis before the screening.

My UC didn't improve with treatments like steroids or biologics.

Evidence of UC extending beyond the rectum, as determined by baseline endoscopy

Exclusion Criteria

I have undergone apheresis within the last 2 weeks.

I have a history of immune system problems or genetic disorders causing colitis.

I have been diagnosed with Crohn's disease or indeterminate colitis.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral ozanimod to evaluate its effectiveness and safety in achieving and maintaining clinical remission

8-12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ozanimod

Trial Overview The study is testing the effectiveness and safety of an oral medication called Ozanimod in pediatric patients. It aims to see if this drug can help achieve and maintain clinical remission in kids who have not had success with standard therapies for their ulcerative colitis.

How Is the Trial Designed?

Group I: Ozanimod Low Dose Experimental Treatment 1 Intervention

Group II: Ozanimod High Dose Experimental Treatment 1 Intervention

Ozanimod is already approved in European Union, United States for the following indications:

Approved in European Union as Ozanimod for:

• Moderate to severe active ulcerative colitis

Approved in United States as Ozanimod for:

• Moderate to severe active ulcerative colitis

Who Is Running the Clinical Trial?

Bristol-Myers Squibb

Lead Sponsor

Trials

2,731

Recruited

4,127,000+

Headquarters

New York City, USA

Known For

Oncology & Cardiovascular

Top Products

Eliquis, Opdivo, Revlimid, Orencia

Christopher Boerner

Bristol-Myers Squibb

Chief Executive Officer since 2023

PhD in Business Administration from the Haas School of Business, University of California, Berkeley; BA in Economics and History from Washington University in St. Louis

Deepak L. Bhatt

Bristol-Myers Squibb

Chief Medical Officer since 2024

MD from Yale University; MSc in Clinical Epidemiology from the University of Pennsylvania

Published Research Related to This Trial

Ozanimod has been successfully used for the first time in a patient hospitalized for acute severe ulcerative colitis, demonstrating its potential as a treatment option in this challenging clinical scenario.

This case report suggests that ozanimod can be effectively combined with cyclosporine therapy, indicating a promising approach for managing patients who do not respond adequately to traditional treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ozanimod Maintenance Therapy After Cyclosporine Induction in Acute Severe Ulcerative Colitis. Cohen, NA., Dalal, SR., Choi, D., et al.[2022]

In a study of 45 patients with moderately to severely active ulcerative colitis treated with ozanimod, the drug showed a significant initial effectiveness with 58% achieving clinical response and 53% achieving remission by week 10, although these rates dropped to 25% by week 52.

The safety profile of ozanimod was favorable, with no reported cases of symptomatic bradycardia or new safety concerns, indicating it can be a safe option for patients with treatment-refractory ulcerative colitis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real World Clinical Effectiveness and Safety of Ozanimod in the Treatment of Ulcerative Colitis: 1-Year Follow-Up from a Tertiary Center. Cohen, NA., Choi, D., Garcia, N., et al.[2023]

Ozanimod was approved by the FDA in May 2021 for treating moderate to severe ulcerative colitis, indicating its potential as a new therapeutic option for this chronic condition.

This report presents the first real-world clinical experience with ozanimod, highlighting its application in managing ulcerative colitis in patients outside of clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ozanimod in the Treatment of Ulcerative Colitis: Initial Real-World Data From a Large Tertiary Center. Cohen, NA., Choi, D., Choden, T., et al.[2023]

Ozanimod has been shown to lead to clinical remission in a significantly higher proportion of patients with moderate to severe ulcerative colitis compared to placebo, based on the True North phase 3 trial.

While ozanimod offers a novel oral treatment option for ulcerative colitis, it comes with a unique safety profile that includes risks such as infections and liver enzyme elevations, necessitating careful monitoring during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ozanimod: A First-in-Class Sphingosine 1-Phosphate Receptor Modulator for the Treatment of Ulcerative Colitis. Choi, D., Stewart, AP., Bhat, S.[2022]

Ozanimod (Zeposia®) is the first approved oral medication for adults with moderately to severely active ulcerative colitis, showing effectiveness in inducing and maintaining clinical remission in phase 2 and phase 3 clinical trials.

The drug was generally well tolerated, with only manageable or transient adverse events reported, and no new safety concerns emerged in ongoing studies, indicating it is a safe treatment option for patients who have not responded to other therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ozanimod: A Review in Ulcerative Colitis. Paik, J.[2023]

Ozanimod, a recently approved S1P receptor modulator, has shown significant effectiveness in inducing clinical remission in patients with moderate to severe ulcerative colitis compared to placebo.

In addition to inducing remission, ozanimod demonstrated superior outcomes in clinical response, corticosteroid-free remission, endoscopic improvement, and mucosal healing, with no major safety concerns reported in clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Update on ozanimod for ulcerative colitis. Antonelli, EK., Del Sordo, R., Morelli, O., et al.[2022]

Ozanimod is the first oral small molecule therapy approved for treating moderately to severely active ulcerative colitis, offering a new option for patients who may not respond well to existing biologics or Janus kinase inhibitors.

The review emphasizes the importance of patient-specific factors in determining the suitability of ozanimod treatment, including monitoring for potential risks and educating patients on best practices during therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinician's Guide to Using Ozanimod for the Treatment of Ulcerative Colitis. Sands, BE., Schreiber, S., Blumenstein, I., et al.[2023]

Ozanimod is a new drug that selectively targets S1P receptors 1 and 5, which are important for controlling the movement of immune cells, and has been shown to effectively treat Ulcerative Colitis (UC) and Crohn's Disease (CD) by inducing a reversible decrease in lymphocytes, correlating with positive treatment responses.

While ozanimod is effective, it can cause side effects such as infections, anemia, and elevated liver enzymes, with rare cases of bradycardia and macular edema, highlighting the need for careful monitoring during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Literature Review of Ozanimod Therapy in Inflammatory Bowel Disease: From Concept to Practical Application. Becher, N., Swaminath, A., Sultan, K.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05076175

NCT05076175 | A Study Investigating Oral Ozanimod ... The purpose of this study is to evaluate the effectiveness and safety of ozanimod (RPC1063) in achieving and maintaining clinical remission.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8256627/

Long-Term Efficacy and Safety of Ozanimod in Moderately to ... Using the more conservative NRI analysis, 71.2% and 54.7% of patients achieved partial Mayo clinical response and remission, respectively, by OLE week 56, and ...

3. journals.lww.com journals.lww.com/ajg/fulltext/2015/10001/a_randomized,_double_blind,_placebo_controlled.1841.aspx

A Randomized, Double-Blind, Placebo-Controlled Trial of... Results: Of 197 patients in the induction period, 103 (52.3%) entered the MP and 91 (88.3%) completed. At Week 32 clinical remission occurred in 14/67 (20.9%), ...

4. crohnscolitisfoundation.org crohnscolitisfoundation.org/a-study-investigating-oral-ozanimod-rpc1063-pediatric-participants-with-moderate-to-severe-active-0

A Study Investigating Oral Ozanimod (RPC1063) in ... Study Objective: To evaluate the efficacy and safety of ozanimod in pediatric participants with moderately to severely active UC.

5. centerwatch.com centerwatch.com/clinical-trials/listings/NCT05076175/a-study-investigating-oral-ozanimod-rpc1063-in-pediatric-participants-with-moderate-to-severe-active-ulcerative-colitis

A Study Investigating Oral Ozanimod (RPC1063) in ... The purpose of this study is to evaluate the effectiveness and safety of ozanimod (RPC1063) in achieving and maintaining clinical remission.

6. nejm.org nejm.org/doi/full/10.1056/NEJMoa2033617

Ozanimod as Induction and Maintenance Therapy for ... Ozanimod was more effective than placebo as induction and maintenance therapy in patients with moderately to severely active ulcerative colitis.

7. gastroenterologyadvisor.com gastroenterologyadvisor.com/reports/safety-profile-of-ozanimod-confirmed-for-uc-rms-treatment/

Safety Profile of Ozanimod Confirmed for Treatment of UC, ... Ozanimod Demonstrates Consistent Long-Term Safety Profile in UC, Relapsing MS · Any TEAE: 81.1 per 100 PYs and 65.0 per 100 PYs; · Serious TEAEs: ...