Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Oral Ozanimod in Pediatric Participants with Moderately to Severely Active Crohn's Disease with an Inadequate Response to Conventional Therapy
IMV047-023
What is the goal of the study?
Despite recent progress in Crohn's disease (CD) treatment, there remains an unmet need for oral agents that are safe and convenient and that can provide effective induction and long-term maintenance of clinical remission. Ozanimod is a small molecule compound which selectively binds with high affinity to sphingosine 1-phosphate (S1P) receptors 1 and 5. Many cell types express S1P1, including vascular endothelial cells, brain cells, and lymphocytes. Stimulation (agonism) of this receptor results in biological activities that includes lymphocyte retention in peripheral lymphoid organs (eg, lymph nodes and gastrointestinal Peyer's patches), resulting in reversible systemic reduction in circulating lymphocytes. Given the immune-mediated inflammation in CD, prevention of circulation of disease-exacerbating, self-reactive lymphocytes to the gut is likely to have salutary immunomodulatory effects with a consequent dampening of disease processes. The benefit/risk profile for ozanimod has been evaluated for the indication of CD in adult participants. In an open-label Phase 2 study in adult participants with moderately to severely active CD (RPC01-2201), ozanimod resulted in endoscopic, clinical, and histologic improvement at Weeks 12 and 52 as measured by centrally read Simple Endoscopic Score for Crohn's Disease (SES-CD) scores and histopathology, Crohn's Disease Activity Index (CDAI) and 2-component patient-reported outcome (abdominal pain and stool frequency). Results of biomarkers, C-reactive protein, and fecal calprotectin (FCP) were highly variable; however, median FCP values were less than Baseline at Week 12 and Week 52. Ozanimod is also currently being studied in Phase 3 studies in adult participants with moderate to severe CD as part of a comprehensive inflammatory bowel disease program. A futility analysis was conducted on approximately 300 participants, with a similar proportion of biologic naïve to biologic exposed population as that in the overall study population. The data were reviewed by an independent Data Monitoring Committee, which recommended continuation of the study without modification
Who can participate in the study?
Please contact the study team listed below to learn more.