Oral Ozanimod for Pediatric Ulcerative Colitis

Recruiting at 109 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

Trial Summary

What is the purpose of this trial?

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.

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.

What data supports the effectiveness of the drug Ozanimod for treating pediatric ulcerative colitis?

Ozanimod has been shown to be effective in adults with moderate to severe ulcerative colitis, helping to induce and maintain remission (a period without symptoms) better than a placebo (a substance with no therapeutic effect). It has been approved for use in adults, and while more data is needed for children, its success in adults suggests potential benefits for pediatric patients as well. ^{1 2 3 4 5}

Is ozanimod safe for use in humans?

Ozanimod has been shown to be generally safe in clinical trials for ulcerative colitis, with no significant safety concerns emerging. Some reported side effects include infections, anemia (low red blood cell count), elevated liver enzymes, and rare cases of slow heart rate, heart block, and eye swelling. ^{1 4 5 6 7}

What makes the drug Ozanimod unique for treating pediatric ulcerative colitis?

Ozanimod is unique because it is an oral medication that works by modulating the sphingosine-1-phosphate receptor, which helps reduce inflammation in the colon. It is the first of its kind approved for moderate to severe ulcerative colitis, offering a new option for patients who do not respond well to other treatments. ^{1 2 3 5 8}

Research Team

Bristol-Myers Squibb

Principal Investigator

Bristol-Myers Squibb

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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 (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 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 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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Ozanimod Maintenance Therapy After Cyclosporine Induction in Acute Severe Ulcerative Colitis. [2022]

2. United States 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. [2023]

3. New Zealand pubmed.ncbi.nlm.nih.gov

Ozanimod: A Review in Ulcerative Colitis. [2023]

4. Spain pubmed.ncbi.nlm.nih.gov

Update on ozanimod for ulcerative colitis. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

Ozanimod: A First-in-Class Sphingosine 1-Phosphate Receptor Modulator for the Treatment of Ulcerative Colitis. [2022]

6. England pubmed.ncbi.nlm.nih.gov

Clinician's Guide to Using Ozanimod for the Treatment of Ulcerative Colitis. [2023]

7. New Zealand pubmed.ncbi.nlm.nih.gov

A Literature Review of Ozanimod Therapy in Inflammatory Bowel Disease: From Concept to Practical Application. [2022]

8. United States pubmed.ncbi.nlm.nih.gov

Ozanimod in the Treatment of Ulcerative Colitis: Initial Real-World Data From a Large Tertiary Center. [2023]