Background: No therapies have been formally approved by the Food and Drug Administration for use in pediatric multiple sclerosis, a rare disease.
Objective: We evaluated the safety, efficacy, and pharmacokinetics of dimethyl fumarate in pediatric patients with multiple sclerosis.
Methods: FOCUS, a phase 2, multicenter study of patients aged 10 to 17 years with relapsing-remitting multiple sclerosis, comprised an eight-week baseline and 24-week treatment period; during treatment, patients received dimethyl fumarate (120 mg twice daily on days one to seven; 240 mg twice a day thereafter). Magnetic resonance imaging scans were obtained at week -8, day 0, week 16, and week 24. The primary end point was the change in T2 hyperintense lesion incidence from the baseline period to the final 8 weeks of treatment. Secondary end points were pharmacokinetic parameters and adverse event incidence.
Results: Twenty of 22 enrolled patients completed the study. There was a significant reduction in T2 hyperintense lesion incidence from baseline to the final eight weeks of treatment (P = 0.009). Adverse events (most commonly gastrointestinal events and flushing) and pharmacokinetic parameters were consistent with adult findings. No serious adverse events were considered dimethyl fumarate related.
Conclusions: Dimethyl fumarate treatment was associated with a reduction in magnetic resonance imaging activity in pediatric patients; pharmacokinetic and safety profiles were consistent with those in adults. Dimethyl fumarate is a potential treatment for pediatric multiple sclerosis.
Trial registration: ClinicalTrials.gov NCT02410200.
Keywords: Dimethyl fumarate; Efficacy; Pediatric; Pharmacokinetics; Relapsing-remitting multiple sclerosis; Safety.
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