title: MD1003 (high-dose biotin) for the treatment of progressive multiple sclerosis: A randomised, double-blind, placebo-controlled study.
abstract: Treatment with MD1003 (high-dose biotin) showed promising results in progressive multiple sclerosis (MS) in a pilot open-label study.
  
  
  
  To confirm the efficacy and safety of MD1003 in progressive MS in a double-blind, placebo-controlled study.
  
  
  
  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.
  
  
  
  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.
  
  
  
  MD1003 achieves sustained reversal of MS-related disability in a subset of patients with progressive MS and is well tolerated.
authors: Department of Neurology and Faculté de Médecine de Reims, CHU de Reims, URCA, Reims, France atourbah@chu-reims.fr.
authors: Ayman
authors: A
authors: Tourbah
authors: Department of Neurology and Faculté de Médecine de Nice-Sophia Antipolis, Nice, France.
authors: Christine
authors: C
authors: Lebrun-Frenay
authors: Service de Neurologie, CHU de Rennes, CICP 1414 INSERM, Rennes, France.
authors: Gilles
authors: G
authors: Edan
authors: Department of Neurology, CHU de Toulouse, Toulouse, France.
authors: Michel
authors: M
authors: Clanet
authors: Department of Neurology, GH Pitié Salpêtrière, Paris, France.
authors: Caroline
authors: C
authors: Papeix
authors: Department of Neurology A, Hospices Civils de Lyon, Lyon, France.
authors: Sandra
authors: S
authors: Vukusic
authors: Department of Neurology and Clinical Investigation Center, CHU de Strasbourg, INSERM 1434, Strasbourg, France.
authors: Jerome
authors: J
authors: De Sèze
authors: Department of Neurology, CHU de Nancy, Nancy, France.
authors: Marc
authors: M
authors: Debouverie
authors: Department of Neurology, Fondation Ophtalmologique Adolphe de Rothschild, Paris, France.
authors: Olivier
authors: O
authors: Gout
authors: Department of Neurology, CHU de Clermont-Ferrand, Clermont-Ferrand, France.
authors: Pierre
authors: P
authors: Clavelou
authors: Department of Neurology, CHU de Caen, Caen, France.
authors: Gilles
authors: G
authors: Defer
authors: Department of Neurology, CHU de Nantes, CIC015 INSERM, INSERM CR1064, Nantes, France.
authors: David-Axel
authors: DA
authors: Laplaud
authors: Department of Neurology, University Hospital of Dijon, Dijon, France.
authors: Thibault
authors: T
authors: Moreau
authors: Department of Neurology, CHU de Montpellier, Montpellier, France.
authors: Pierre
authors: P
authors: Labauge
authors: Department of Neurology, CHU de Bordeaux, Bordeaux, France.
authors: Bruno
authors: B
authors: Brochet
authors: MedDay Pharmaceuticals, Paris, France.
authors: Frédéric
authors: F
authors: Sedel
authors: APHM, Hôpital de la Timone, CNRS, CRMBM UMR 7339, CNRS, Department of Neurology and Aix-Marseille Université, Marseille, France.
authors: Jean
authors: J
authors: Pelletier
mesh_terms: D000328:Adult; D001710:Biotin / Q000008:administration & dosage / Q000009:adverse effects / Q000494:pharmacology*; D018450:Disease Progression; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D020528:Multiple Sclerosis, Chronic Progressive / Q000188:drug therapy*; D017063:Outcome Assessment, Health Care; D014803:Vitamin B Complex / Q000008:administration & dosage / Q000009:adverse effects / Q000494:pharmacology*
chemical_list: D014803:Vitamin B Complex; D001710:Biotin
keywords: MD1003; Multiple sclerosis; clinical trial; disability progression; high-dose biotin; primary progressive multiple sclerosis; secondary progressive multiple sclerosis