High-dose pharmaceutical grade biotin (MD1003) in amyotrophic lateral sclerosis: A pilot study.
ALS
Amyotrophic lateral sclerosis
Biotin
MD1003
MND
Motor neuron disease
Motor neurone disease
phase 2
Journal
EClinicalMedicine
ISSN: 2589-5370
Titre abrégé: EClinicalMedicine
Pays: England
ID NLM: 101733727
Informations de publication
Date de publication:
Feb 2020
Feb 2020
Historique:
received:
30
06
2019
revised:
20
12
2019
accepted:
24
12
2019
entrez:
7
3
2020
pubmed:
7
3
2020
medline:
7
3
2020
Statut:
epublish
Résumé
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, MD1003 treatment was safe and well tolerated. It was not possible to establish MD1003 efficacy in this relatively small study. Given the favourable safety profile of MD1003 and an imbalance between treatment groups favouring placebo, additional, larger studies in ALS are warranted. MedDay Pharmaceuticals.
Sections du résumé
BACKGROUND
BACKGROUND
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.
METHODS
METHODS
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.
FINDINGS
RESULTS
Between June and December 2016, 30 patients were enrolled (MD1003,
INTERPRETATION
CONCLUSIONS
MD1003 treatment was safe and well tolerated. It was not possible to establish MD1003 efficacy in this relatively small study. Given the favourable safety profile of MD1003 and an imbalance between treatment groups favouring placebo, additional, larger studies in ALS are warranted.
FUNDING
BACKGROUND
MedDay Pharmaceuticals.
Identifiants
pubmed: 32140672
doi: 10.1016/j.eclinm.2019.100254
pii: S2589-5370(19)30263-9
pii: 100254
pmc: PMC7046518
doi:
Banques de données
ClinicalTrials.gov
['NCT03114215']
Types de publication
Journal Article
Langues
eng
Pagination
100254Informations de copyright
© 2019 Published by Elsevier Ltd.
Déclaration de conflit d'intérêts
WC received personal fees from Actelion, Effik, Merck, MedDay Pharmaceuticals, Novartis Pharma, Roche, and Sanofi. AB and FS are employees of MedDay Pharmaceuticals. RJM, NP and SA have nothing to disclose. SS was funded by MedDay Pharmaceuticals.
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