Oral Ozanimod for Pediatric Ulcerative Colitis

Recruiting in Palo Alto (17 mi)

+109 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Recruiting

Sponsor: Bristol-Myers Squibb

Disqualifiers: Crohn's disease, Immunodeficiency, others

No Placebo Group

Prior Safety Data

Approved in 2 Jurisdictions

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}

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.

Trial 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 (Sphingosine 1-phosphate receptor modulator)

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

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Valley View Wellness Medical Center Garden Grove, CA

Local Institution Cleveland, OH

University of Massachusetts Chan Medical School Worcester, MA

Local Institution - 0096 Sacramento, CA

More Trial Locations

Who Is Running the Clinical Trial?

Bristol-Myers Squibb Lead Sponsor

References

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

Ozanimod Maintenance Therapy After Cyclosporine Induction in Acute Severe Ulcerative Colitis. [2022] Ozanimod has recently received US Food and Drug Administration approval for moderate-to-severe ulcerative colitis. Treatment of acute severe colitis remains a clinical challenge, and although many patients respond to cyclosporine therapy, there remains a relative paucity of maintenance options. This case report describes the first reported, successful use of ozanimod after hospitalization for acute severe ulcerative colitis and in tandem with cyclosporine. Although a longer follow-up is required, this report shows the feasibility of ozanimod in this clinical setting.

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] Ozanimod is a first-in-class Sphingosine-1-phosphate (S1P) receptor modulator approved for the treatment of moderately to severely active ulcerative colitis (UC). Real world data describing use of ozanimod are limited.

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

Ozanimod: A Review in Ulcerative Colitis. [2023] Ozanimod (Zeposia&#174;) is the first sphingosine-1-phosphate receptor (S1PR) modulator to be approved for the treatment of adults with moderately to severely active ulcerative colitis in the USA, and in adults with moderately to severely active ulcerative colitis who have had an inadequate or lost response to, or were intolerant of, either conventional therapy or a biologic in the EU. An oral agent, ozanimod is administered once daily as induction and maintenance therapy. In the randomized, double-blind, multinational phase&#160;2 Touchstone and phase&#160;3 True North clinical trials, ozanimod was effective in inducing clinical remission and maintaining remission relative to placebo in adults with moderately to severely active ulcerative colitis. Ozanimod was generally well tolerated in these studies, with manageable or transient adverse events (AEs). Current data from the Touchstone and True North open-label extensions are consistent with the primary studies with respect to therapeutic efficacy and tolerability, with no new safety signals observed. Although further data will be beneficial, ozanimod expands the treatment options for adults with moderately to severely active ulcerative colitis.

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

Update on ozanimod for ulcerative colitis. [2022] Treating moderate to severe ulcerative colitis (UC) has been enriched by the increasing number of drugs available for this disease. However, failure of conventional therapies, an incomplete response, or loss of response to biologics is experienced in many UC patients. Thus, there is still a growing need for new drugs in the therapeutic arsenal for UC. Ozanimod is a sphingosine-1-phosphate (S1P) receptor modulator which has been recently approved for UC therapy. In this review, we focus on the mechanism of action of ozanimod hydrochloride in preclinical studies of intestinal inflammation as well as its clinical effectiveness and safety in moderate to severe UC patients. In this population, ozanimod was shown to be significantly more effective than placebo to induce clinical remission. Additionally, in terms of clinical response, corticosteroid-free remission, endoscopic improvement and mucosal healing, ozanimod performed significantly better than placebo in this population. No significant safety concerns about ozanimod emerged from clinical trials in UC.

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] To review the pharmacological and clinical profile of ozanimod in the treatment of ulcerative colitis (UC).

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

Clinician's Guide to Using Ozanimod for the Treatment of Ulcerative Colitis. [2023] The emergence of advanced therapies (eg, biologics, Janus kinase inhibitors) over the past few decades has revolutionized the treatment of ulcerative colitis. However, the limitations of these therapies leave an unmet need for safer and more effective or convenient treatment options. There is growing interest in the development of novel oral small molecule therapies for the treatment of ulcerative colitis. Ozanimod is an oral small molecule therapy that is approved in the United States, the European Union, and other countries as the first sphingosine 1-phosphate receptor modulator for the treatment of moderately to severely active ulcerative colitis in adults. This review provides guidance for ozanimod use for the treatment of ulcerative colitis based on the prescribing information, clinical trial and real-world data, and the authors' clinical experiences. This guidance outlines patient characteristics to consider when deciding if ozanimod treatment is suitable and describes how to educate patients on risks and best practices. It also details the nature and frequency of monitoring during treatment, which should be adapted to the individual patient based on preexisting risk factors and events that possibly occur during treatment. This review also provides insights into the patient characteristics and clinical scenarios best suited for ozanimod treatment based on its efficacy and safety profile and the comparative risks with other therapies.

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

A Literature Review of Ozanimod Therapy in Inflammatory Bowel Disease: From Concept to Practical Application. [2022] Inflammatory bowel disease (IBD), namely Ulcerative Colitis (UC) and Crohn's Disease (CD), is believed to be due to a dysregulation of the innate immune response. The complexity of the immune cascade offers both a challenge and an opportunity to researchers seeking out new treatments for IBD, as various points along the inflammatory pathways can be targeted for interruption. Sphinogosine-1-phosphate (S1P) is a phospholipid molecule with wide ranging biological effects caused by binding five known S1P receptor subtypes. Ozanimod is a small molecule drug that selectively targets S1P receptors 1 and 5 which play a crucial role in lymphocyte trafficking. In clinical trials for both UC and CD, it has been shown to induce a reversible lymphopenia which correlates with response to therapy. Reported adverse events include infection, anemia, and elevated liver enzymes. Rare instances of bradycardia, heart block, and macular edema were also reported. As a newly available therapy approved for UC patients, we aim to summarize ozanimod's novel mechanism of action, pre-clinical and clinical trial results, and to give context to this newly available drug that gastroenterologists may utilize in their treatment algorithm.

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] Ulcerative colitis (UC) is a chronic inflammatory condition affecting the colon and rectum. Long-term therapy is generally required to achieve and maintain disease control.1 In May 2021 the US Food and Drug Administration approved the use of ozanimod in patients with moderate to severe UC. We describe the first report of the use of ozanimod in real-world clinical practice.