MD1003 (high-dose biotin) for the treatment of progressive multiple sclerosis: A randomised, double-blind, placebo-controlled study

Abstract : Background: Treatment with MD1003 (high-dose biotin) showed promising results in progressive multiple sclerosis (MS) in a pilot open-label study. Objective: To confirm the efficacy and safety of MD1003 in progressive MS in a double-blind, placebo-controlled study. Methods: Patients (n = 154) with a baseline Expanded Disability Status Scale (EDSS) score of 4.5-7 and evidence of disease worsening within the previous 2 years were randomised to 12-month MD1003 (100 mg biotin) or placebo thrice daily, followed by 12-month MD1003 for all patients. The primary endpoint was the proportion of patients with disability reversal at month 9, confirmed at month 12, defined as an EDSS decrease of ≥1 point (≥0.5 for EDSS 6-7) or a ≥20% decrease in timed 25-foot walk time compared with the best baseline among screening or randomisation visits. Results: A total of 13 (12.6%) MD1003-treated patients achieved the primary endpoint versus none of the placebo-treated patients (p = 0.005). MD1003 treatment also reduced EDSS progression and improved clinical impression of change compared with placebo. Efficacy was maintained over follow-up, and the safety profile of MD1003 was similar to that of placebo. Conclusion: MD1003 achieves sustained reversal of MS-related disability in a subset of patients with progressive MS and is well tolerated. © The Author(s), 2016.
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Submitted on : Monday, December 12, 2016 - 4:54:02 PM
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A. Tourbah, C. Lebrun-Frenay, G. Edan, M. Clanet, C. Papeix, et al.. MD1003 (high-dose biotin) for the treatment of progressive multiple sclerosis: A randomised, double-blind, placebo-controlled study. Multiple Sclerosis, 2016, 22 (13), pp.1719--1731. ⟨10.1177/1352458516667568⟩. ⟨hal-01414958⟩

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