MD1003 (High-Dose Biotin) for the Treatment of Progressive Multiple Sclerosis: A Randomised, Double-Blind, Placebo-Controlled Study

Mult Scler. 2016 Nov;22(13):1719-1731. doi: 10.1177/1352458516667568. Epub 2016 Sep 1.

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.

Keywords: MD1003; Multiple sclerosis; clinical trial; disability progression; high-dose biotin; primary progressive multiple sclerosis; secondary progressive multiple sclerosis.

Publication types

  • Randomized Controlled Trial

MeSH terms

  • Adult
  • Biotin / administration & dosage
  • Biotin / adverse effects
  • Biotin / pharmacology*
  • Disease Progression*
  • Double-Blind Method
  • Female
  • Humans
  • Male
  • Middle Aged
  • Multiple Sclerosis, Chronic Progressive / drug therapy*
  • Outcome Assessment, Health Care*
  • Vitamin B Complex / administration & dosage
  • Vitamin B Complex / adverse effects
  • Vitamin B Complex / pharmacology*

Substances

  • Vitamin B Complex
  • Biotin

Associated data

  • EudraCT/2013-002113-35