Title :

MD1003 (High-Dose Pharmaceutical-Grade Biotin) for the Treatment of Chronic Visual Loss Related to Optic Neuritis in Multiple Sclerosis: A Randomized, Double-Blind, Placebo-Controlled Study.

Author(s) :

Tourbah, Ayman [Auteur]
AP-HP. Université Paris Saclay
Gout, Olivier [Auteur]
Fondation Ophtalmologique Adolphe de Rothschild [Paris]
Vighetto, Alain [Auteur]
Centre de recherche en neurosciences de Lyon - Lyon Neuroscience Research Center [CRNL]
Deburghgraeve, Veronique [Auteur]
Centre Hospitalier Universitaire de Rennes [CHU Rennes] = Rennes University Hospital [Pontchaillou]
Pelletier, Jean [Auteur]
Aix Marseille Université [AMU]
Papeix, Caroline [Auteur]
CHU Pitié-Salpêtrière [AP-HP]
Lebrun-Frenay, Christine [Auteur]
Université Côte d'Azur [UniCA]
Labauge, Pierre [Auteur]
CHU Montpellier = Montpellier University Hospital
Brassat, David [Auteur]
Université de Toulouse [UT]
Toosy, Ahmed [Auteur]
Laplaud, David-Axel [Auteur]
Centre de Recherche en Transplantation et Immunologie [U1064 Inserm - CRTI]
Outteryck, Olivier [Auteur]
Lille Inflammation Research International Center (LIRIC) - U995
Moreau, Thibault [Auteur]
Service de Neurologie générale, vasculaire et dégénérative (CHU de Dijon)
Debouverie, Marc [Auteur]
Service de neurologie [CHRU Nancy]
Clavelou, Pierre [Auteur]
Service Neurologie [CHU Clermont-Ferrand]
Heinzlef, Olivier [Auteur]
CHI Poissy-Saint-Germain
De Seze, Jerome [Auteur]
Service de Neurologie [Strasbourg]
Defer, Gilles [Auteur]
Service de Neurologie [CHU Caen]
Sedel, Frederic [Auteur]
MedDay Pharmaceuticals
Arndt, Carl [Auteur]
Université de Reims Champagne-Ardenne [URCA]

Journal title :

CNS Drugs

Abbreviated title :

CNS Drugs

Volume number :

32

Pages :

661-672

Publication date :

2018-07

HAL domain(s) :

Sciences du Vivant [q-bio]

English abstract : [en]

BACKGROUND: Chronic visual loss is a disabling feature in patients with multiple sclerosis (MS). It was recently shown that MD1003 (high-dose pharmaceutical-grade biotin or hdPB) may improve disability in patients with ...
Show more >BACKGROUND: Chronic visual loss is a disabling feature in patients with multiple sclerosis (MS). It was recently shown that MD1003 (high-dose pharmaceutical-grade biotin or hdPB) may improve disability in patients with progressive MS. OBJECTIVE: The aim of this study was to evaluate whether MD1003 improves vision compared with placebo in MS patients with chronic visual loss. METHODS: The MS-ON was a 6-month, randomized, double-blind, placebo-controlled study with a 6-month open-label extension phase. Adult patients with MS-related chronic visual loss of at least one eye [visual acuity (VA) below 0.5 decimal chart] were randomized 2:1 to oral MD1003 300 mg/day or placebo. The selected eye had to show worsening of VA within the past 3 years following either acute optic neuritis (AON) or slowly progressive optic neuropathy (PON). The primary endpoint was the mean change from baseline to month 6 in VA measured in logarithm of the minimum angle of resolution (logMAR) at 100% contrast of the selected eye. Visually evoked potentials, visual field, retinal nerve fiber layer (RNFL) thickness, and health outcomes were also assessed. RESULTS: Ninety-three patients received MD1003 (n = 65) or placebo (n = 28). The study did not meet its primary endpoint, as the mean change in the primary endpoint was nonsignificantly larger (p = 0.66) with MD1003 (- 0.061 logMAR, + 3.1 letters) than with placebo (- 0.036 logMAR, + 1.8 letters). Pre-planned subgroup analyses showed that 100% contrast VA improved by a mean of + 2.8 letters (- 0.058 logMAR) with MD1003 and worsened by - 1.5 letters (+ 0.029 logMAR) with placebo (p = 0.45) in the subgroup of patients with PON. MD1003-treated patients also had nonsignificant improvement in logMAR at 5% contrast and in RNFL thickness and health outcome scores when compared with placebo-treated patients. There was no superiority of MD1003 vs placebo in patients with AON. The safety profile of MD1003 was similar to that of placebo. CONCLUSIONS: MD1003 did not significantly improve VA compared with placebo in patients with MS experiencing chronic visual loss. An interesting trend favoring MD1003 was observed in the subgroup of patients with PON. Treatment was overall well tolerated. TRIAL REGISTRATION: EudraCT identifier 2013-002112-27. ClinicalTrials.gov Identifier: NCT02220244 FUNDING: MedDay Pharmaceuticals.Show less >

Language :

Anglais

Audience :

Internationale

Popular science :

Non

Administrative institution(s) :

Inserm
Université de Lille
CHU Lille

Collections :

• Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286

Research team(s) :

Immunity, inflammation and fibrsis in auto and allo-reactivity

Submission date :

2019-03-01T14:08:08Z
2024-03-15T08:51:03Z
2024-03-15T08:57:01Z