A Phase 3 Study With an Open-Label Extension in Pediatric Participants to Evaluate the Safety, Efficacy, and Pharmacokinetics of Diroximel Fumarate and Dimethyl Fumarate for the Treatment of Relapsing Forms of Multiple Sclerosis

Official Summary

In this study, researchers will learn more about the study drugs diroximel fumarate (DRF) and dimethyl fumarate (DMF) in children with MS who may be experiencing relapses. Participants will be divided into 2 groups based on their weight: * Group A will include children who weigh 40 kilograms (kg) or less. They will receive DRF in both Part 1 and Part 2 of the study. * Group B will include children who weigh more than 40 kg. They will be randomly assigned to receive DRF, DMF, or fingolimod. Fingolimod is a drug already used to treat MS in adults. In Part 2, they will receive DRF. This is a 2-part study. Part 1 treatment will last 96 weeks. Participants who complete Part 1 may move to Part 2. Part 2 is an extension period. Treatment will last another 96 weeks and will help researchers learn about the long-term safety and effects of treatment. The main goal of the study is to learn about the safety of DRF and DMF and compare their effect on relapses and brain lesions with fingolimod. The main questions researchers want to answer are: * How many participants have adverse events and serious adverse events? * How often do participants relapse after treatment with DRF and DMF compared to fingolimod? Researchers will take brain imaging scans to check for any new areas of brain inflammation and compare the brain lesions before and after treatment. Researchers will also measure the amount of drug in the blood to understand how the body processes it. To check safety, they will monitor participants' growth and development, and compare changes in heart tests, vital signs, and lab tests. They will also use rating scales to monitor depression symptoms. The study will be done as follows: Part 1 (Treatment Period) * After screening, participants will join Part 1 and be divided into 2 groups based on their weight. * Participants in Group A will receive DRF. * Participants in Group B will be randomly assigned to receive either DRF, DMF, or fingolimod. * Neither the research

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: QUADRUPLE
• Enrollment: 185 participants

Interventions

• DRUG: Diroximel Fumarate — Oral capsule
• DRUG: Dimethyl Fumarate — Oral capsule
• DRUG: Fingolimod — Oral capsule
• DRUG: Placebo matching DRF — Oral capsule
• DRUG: Placebo matching DMF — Oral capsule

Primary Outcomes

• TP Cohort A and OLE Period: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and AEs Leading to Treatment Discontinuation (Cohort A: From first dose of study drug up to end of follow-up (up to Week 104), OLE: Baseline (Week 96) up to the end of OLE period (up to Week 196))
• TP Cohort B: Annualized Relapse Rate (ARR) Through Week 96 (Baseline (Day 1) up to Week 96)

Secondary Outcomes

• TP Cohorts A and B: Maximum Observed Concentration at Steady State (Cmax,ss) of Monomethyl Fumarate (MMF) (Predose and at multiple timepoints post dose up to Week 96)
• TP Cohorts A and B: Minimum Observed Concentration at Steady State (Cmin,ss) of MMF (Predose and at multiple timepoints post dose up to Week 96)
• TP Cohorts A and B: Cmax,ss of 2-Hydroxyethyl Succinimide (HES) (Predose and at multiple timepoints post dose up to Week 96)
• TP Cohorts A and B: Cmin,ss of HES (Predose and at multiple timepoints post dose up to Week 96)
• TP Cohorts A, B and OLE Period: Number of Participants with Potentially Clinically Serious (PCS) Change from Baseline in Vital Sign Parameters (Cohorts A and B: Baseline up to Week 96, OLE: Baseline (Week 96) up to Week 196)

More Relapsing Forms of Multiple Sclerosis Trials

View all Relapsing Forms of Multiple Sclerosis clinical trials