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

Ayman Tourbah 1, * Christine Lebrun Frenay 2 Gilles Edan 3 Michel Clanet 4 Caroline Papeix 5 Sandra Vukusic 6 Jerome de Sèze 7 Marc Debouverie 8 Olivier Gout 9 Pierre Clavelou 10 Gilles Defer 11 David-Axel Laplaud 12, 13 Thibault Moreau 14 Pierre Labauge 15 Bruno Brochet 16 Frédéric Sedel 17 Jean Pelletier 18, 19
* Corresponding author
1 Service de neurologie [Reims]
2 Service de Neurologie [CHU Nice]
3 Service de Neurologie [Rennes]
4 Services de Neurologie [CHU Toulouse]
5 Service de neurologie 1 [CHU Pitié-Salpétrière]
6 Service de Neurologie [Hospices civils de Lyon - Hôpital Pierre Wertheimer]
7 Centre d’Investigation Clinique Plurithématique (CIC - P) - CIC Strasbourg
8 Service de neurologie [CHRU Nancy]
9 Fondation Ophtalmologique Adolphe de Rothschild - Service de neurologie [Paris]
10 Service de Neurologie [CHU Clermont-Ferrand]
11 Service de Neurologie [CHU Caen]
12 U1064 Inserm - CRTI - Centre de Recherche en Transplantation et Immunologie
13 Service de neurologie [Nantes]
14 CHU Dijon - Centre Hospitalier Universitaire de Dijon - Hôpital François Mitterrand
15 Département de neurologie [Montpellier]
16 Service de neurologie [Bordeaux]
17 MedDay Pharmaceuticals
18 TIMONE - Hôpital de la Timone [CHU - APHM]
19 CRMBM - Centre de résonance magnétique biologique et médicale
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, placebocontrolled 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 : secondary progressive multiple sclerosis primary progressive multiple sclerosis Multiple sclerosis clinical trial disability progression high-dose biotin MD1003
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Submitted on : Wednesday, June 12, 2019 - 11:24:54 AM
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INSERM | HCL | UNIV-RENNES1 | CNRS | COMUE-NORMANDIE | UNIV-MONTPELLIER | UNIV-NANTES | SITE-ALSACE | UNIV-RENNES | CRMBM | CRTI | UNIV-AMU | UNICAEN | SORBONNE-UNIVERSITE | SU-TI

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Ayman Tourbah, Christine Lebrun Frenay, Gilles Edan, Michel Clanet, Caroline Papeix, et al.. MD1003 (high-dose biotin) for the treatment of progressive multiple sclerosis: A randomised, double-blind, placebo-controlled study. Multiple Sclerosis Journal, SAGE Publications, 2016, 22 (13), pp.1719--1731. ⟨10.1177/1352458516667568Multiple⟩. ⟨inserm-02153440⟩

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