High-dose pharmaceutical grade biotin (MD1003) in amyotrophic lateral sclerosis: A pilot study

EClinicalMedicine
Raul Juntas-MoralesWilliam Camu

Abstract

Oligodendrocytes (OGs) provide metabolic support to motor neurons (MNs) and are implicated in the pathophysiology of amyotrophic lateral sclerosis (ALS). MD1003, or high-dose Pharmaceutical grade Biotin (hdPB), may improve disability in progressive multiple sclerosis patients via augmentation of OG or MN energy levels. Here, we assessed the safety and efficacy of MD1003 in ALS patients. This single centre, randomised, double-blind, placebo-controlled trial included patients aged 25-80 years with probable or definite ALS. Patients were assigned (2:1), using a computer-generated randomisation list, to receive oral MD1003 (300 mg/day) or placebo treatment for 24 weeks. The primary outcome, safety, was analysed in all patients who received at least one dose of study drug. This study, registered with ClinicalTrials.gov, NCT03114215, has been completed. Between June and December 2016, 30 patients were enrolled (MD1003, n = 20; placebo, n = 10). Baseline characteristics were representative of the ALS population. MD1003 and placebo groups were not well balanced at screening, with the MD1003-treated group having a higher rate of ALSFRS-R decline prior to screening versus placebo (-6·0 IQR [-8·5, -5·0] vs. -5·0 IQR [-5·0, -3·0]) and a pr...Continue Reading

Associated Clinical Trials

Citations

Apr 18, 2020·Expert Opinion on Investigational Drugs·Pablo Villoslada, Lawrence Steinman
Apr 4, 2021·International Journal of Molecular Sciences·Elisabeth TraiffortAmina Zahaf

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Methods Mentioned

BETA
transgenic

Clinical Trials Mentioned

NCT03114215

Software Mentioned

SAS
R

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Amyotrophic Lateral Sclerosis (ALS) is a progressive nervous system disease associated with the death of neurons that control voluntary muscles. Discover the latest research on ALS here.