Placebo-Controlled Study of the Efficacy and Safety of BG00012 in Pediatric Subjects With Relapsing-Remitting Multiple Sclerosis (RRMS)

Study Overview
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Brief Summary
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The primary objective of the study is to assess the efficacy of oral BG00012 as compared with placebo in pediatric subjects with relapsing-remitting multiple sclerosis (RRMS). The secondary objectives of this study are to evaluate the safety and tolerability of BG00012 and to compare the effect of BG00012 with placebo on additional clinical and radiological measures of disease activity.

Official Title
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A Randomized, Placebo-Controlled, Parallel-Group Study in Pediatric Subjects Ages 10 Through 17 Years to Evaluate the Efficacy and Safety of BG00012 for the Treatment of Relapsing-Remitting Forms of Multiple Sclerosis

Conditions
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Relapsing-Remitting Multiple Sclerosis, Relapsing Forms of Multiple Sclerosis

Intervention / Treatment
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* Drug: dimethyl fumarate
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Informed consent and assent as appropriate Must have a body weight of ≥30 kg Must have a diagnosis of RRMS as defined by the revised consensus definition for pediatric multiple sclerosis (MS) Must be ambulatory with a converted Krutzke Baseline Expanded Disability Status Scale (EDSS) score between 0 and 5.0, inclusive Key Exclusion Criteria: Primary progressive, secondary progressive, or progressive relapsing MS. History of disorders mimicking MS, such as other demyelinating disorders (e.g., acute disseminated encephalomyelitis), systemic autoimmune disorders (e.g., Sjögren disease and lupus erythematosus), metabolic disorders (e.g., dystrophies), and infectious disorders. History of severe allergic or anaphylactic reactions, or known drug hypersensitivity to dimethyl fumarate (DMF) or fumaric acid esters. Prior treatment with any of the following medications within 12 months prior to randomization: mitoxantrone, cyclophosphamide, rituximab. Prior treatment with any of the following medications or procedures within 6 months prior to randomization: fingolimod, teriflunomide, natalizumab, cyclosporine, azathioprine, methotrexate, mycophenolate mofetil, laquinimod, intravenous (IV) immunoglobulin, plasmapheresis or cytapheresis. NOTE: Other protocol defined inclusion/exclusion criteria may apply.

Ages Eligible for Study
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Minimum Age: 10 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BG00012<br>Participants will receive 120 mg capsule(s) BG00012 taken orally. | Drug: dimethyl fumarate<br>* enteric-coated microtablets<br>* Other names: DMF;|
| Experimental: Placebo<br>Participants will receive matching placebo capsule(s) taken orally. | Drug: Placebo<br>* enteric-coated microtablets<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to first multiple sclerosis (MS) relapse | Relapses are defined as new or recurrent neurologic symptoms not associated with fever or infection, lasting at least 24 hours, and accompanied by new objective neurological findings upon examination by the Examining Neurologist. | Up to week 104 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants that experience adverse events (AEs) and serious adverse events (SAEs) |  | Up to week 104 | 
| Number of new or newly enlarging T2 Hyperintense Lesions on Brain magnetic resonance imaging (MRI) scans |  | Weeks 24, 48, 72 and 96 | 
| Number of gadolinium-enhancing Lesions |  | Baseline, and weeks 24, 48, 72 and 96 | 
| Annualized MS relapse rate |  | weeks 48 and 96 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Safety, Randomized, Ages 10-17, Pediatrics, Placebo controlled, Relapse-Remitting, Multiple Sclerosis, Efficacy, BG00012