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.

SUBMITTER: Tourbah A 

PROVIDER: S-EPMC5098693 | biostudies-literature | 2016 Nov

REPOSITORIES: biostudies-literature