IM047-001 Ozanimod (RPC1063) in pediatric subjects with ulcerative colitis
Research type
Research Study
Full title
A phase 2/3, multicenter, randomized, double-blind study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of oral ozanimod (RPC1063) in pediatric subjects with moderately to severely active ulcerative colitis with an inadequate response to conventional therapy.
IRAS ID
1006284
Contact name
GSM-CT Representative
Contact email
Sponsor organisation
Celgene International II Sàrl
Eudract number
2021-002308-11
Clinicaltrials.gov Identifier
Research summary
Ozanimod is approved in adults with moderate to severe ulcerative colitis (UC). The aim of this study is to assess the effectiveness and safety of ozanimod in children and adolescents who have moderate to severe UC. Ozanimod is thought to act on the immune system by binding to certain types of circulating white blood cells and as a result, keep them away from sites of inflammation.
The study will enrol 120 participants in different countries and includes 4 different periods (Screening, Double-Blind Treatment, Long-Term Extension and Follow-Up): An initial screening period to check suitability, then a double blinded treatment period (participants nor investigators will know the dose amount, which allows unbiased assessment) for up to 52 weeks. After that, there is an option to enter a long-term extension study, lasting up to 5 years. Participants will then complete 2 follow-up visits at the end of their study participation, mainly to check that the participant is well and there are no ill-effects from ozanimod or other study procedures.
Eligible participants will take ozanimod in capsule form, by mouth. For children who are not able to swallow capsules, a granule sprinkle formulation is in development. The two dose groups for age 12-17years are: High dose: 0.92 mg once a day OR Low dose: 0.46 mg once a day. There is an equal chance that participants will be assigned to either dose group. The doses to be assessed in the younger age groups (age 2-11) will depend on the information learned from the older age group(s).
Participants will have physicals (temperature, heart rate and blood pressure checked), ECGs, blood tests, urine tests, stool tests, low dose x-rays (that measure calcium in the bone and compare to other people their age, size, and gender) and procedures to examine their intestines/digestive tract (using a thin long flexible tube with a light and tiny camera attached). They will complete questionnaires and fill out electronic diaries each day.
REC name
London - Hampstead Research Ethics Committee
REC reference
23/LO/0079
Date of REC Opinion
15 Sep 2023
REC opinion
Further Information Favourable Opinion