Trial reportMultiple sclerosis (Houndmills, Basingstoke, England)2016
MD1003 (high-dose biotin) for the treatment of progressive multiple sclerosis: A randomised, double-blind, placebo-controlled study.
Trial report in Multiple sclerosis (Houndmills, Basingstoke, England), 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03427086 (Safety and Tolerability of High Dose Biotin in Patients With Amyotrophic Lateral Sclerosis), which is not on this map. Cited by 107 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Safety and Tolerability of High Dose Biotin in Patients With Amyotrophic Lateral Sclerosis
Who cites it
107 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Evaluating the Effects of Dietary Interventions on Disease Progression and Symptoms of Adults with Multiple Sclerosis: An Umbrella Review.Advances in nutrition (Bethesda, Md.) · 2020Pooled it
- Dietary interventions for multiple sclerosis-related outcomes.The Cochrane database of systematic reviews · 2020Pooled it
- Idebenone does not inhibit disability progression in primary progressive MS.Multiple sclerosis and related disorders · 2020Trial
- Trial
- Developing Interim Outcome Measures for Multi-Arm Multistage Clinical Trials in Multiple Sclerosis.Neurology · 2026Article
- The Potential Neuroprotective Role of Biotin in Neurorehabilitation: A Review.Current nutrition reports · 2026Review
- Alpha-Lipoic Acid and Biotin in Neurodegenerative Diseases: Convergent Mechanistic Insights from Preclinical Models to Clinical Perspectives.Neurology international · 2026Review
- Review
- Linking movement-related beta oscillations to cortical excitability, structural damage, and fatigue in multiple sclerosis.Brain communications · 2026Article
- Concordance between European medicine agency good clinical practice inspections and medical literature: a meta-research survey.BMC medicine · 2025Article
- Review
- Primary Progressive Multiple Sclerosis: New Therapeutic Approaches.Neuropsychopharmacology reports · 2025Review
- Dose-Dependent Effect of a New Biotin Compound in Hippocampal Remyelination in Rats.Molecular neurobiology · 2025Article
- High-Dose Biotin Supplementation Exacerbates Chronic Migraine: A Case Report.International medical case reports journal · 2025Article
- From prevention to management: Exploring the impact of diet on multiple sclerosis.Translational neuroscience · 2025Review
- Biotin Homeostasis and Human Disorders: Recent Findings and Perspectives.International journal of molecular sciences · 2024Review
- Diagnostic, Theranostic and Prognostic Value of Thyroglobulin in Thyroid Cancer.Journal of clinical medicine · 2024Review
- Managing multiple sclerosis in individuals aged 55 and above: a comprehensive review.Frontiers in immunology · 2024Review
- Practical supplements for prevention and management of migraine attacks: a narrative review.Frontiers in nutrition · 2024Review
- The clinical significance of low dose biotin supplements (<300μg/day) in the treatment of patients with hypothyroidism: crucial or overestimated?Thyroid research · 2023Article
47 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
18 authors.
Funding
Abstract
backgroundTreatment with MD1003 (high-dose biotin) showed promising results in progressive multiple sclerosis (MS) in a pilot open-label study.
objectiveTo confirm the efficacy and safety of MD1003 in progressive MS in a double-blind, placebo-controlled study.
methodsPatients (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.
resultsA 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.
conclusionMD1003 achieves sustained reversal of MS-related disability in a subset of patients with progressive MS and is well tolerated.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.