Trial reportCNS drugs2018
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.
Trial report in CNS drugs, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02220244 (Effect of MD1003 in Chronic Visual Loss Related to Optic Neuritis in Multiple Sclerosis), which is not on this map. Cited by 17 papers, 1 of them a synthesis 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.
Effect of MD1003 in Chronic Visual Loss Related to Optic Neuritis in Multiple Sclerosis: a Pivotal Randomized Double Masked Placebo Controlled Study
Who cites it
17 citing papers in PubMed, 1 synthesis or guideline pooled it, 38 citations in OpenAlex.
- Dietary interventions for multiple sclerosis-related outcomes.The Cochrane database of systematic reviews · 2020Pooled it
- Natural Molecules for Brain Health and Resilience.International journal of molecular sciences · 2026Review
- Alpha-Lipoic Acid and Biotin in Neurodegenerative Diseases: Convergent Mechanistic Insights from Preclinical Models to Clinical Perspectives.Neurology international · 2026Review
- Region-specific brain structural modulation and amyloid-β pathology associated with dietary biotin: insights into dementia neuropathology.EBioMedicine · 2026Article
- Review
- Dose-Dependent Effect of a New Biotin Compound in Hippocampal Remyelination in Rats.Molecular neurobiology · 2025Article
- Nutritional interventional studies in patients with multiple sclerosis: a scoping review of the current clinical evidence.Journal of neurology · 2024Article
- Managing multiple sclerosis in individuals aged 55 and above: a comprehensive review.Frontiers in immunology · 2024Review
- Visual outcome measures in clinical trials of remyelinating drugs.BMJ neurology open · 2024Review
- Updates and advances in multiple sclerosis neurotherapeutics.Neurodegenerative disease management · 2023Review
- Symptomatic and restorative therapies in neuromyelitis optica spectrum disorders.Journal of neurology · 2022Review
- Review
- Radiomic analysis of the optic nerve at the first episode of acute optic neuritis: an indicator of optic nerve pathology and a predictor of visual recovery?La Radiologia medica · 2021Article
- Oligodendrocyte Dysfunction in Amyotrophic Lateral Sclerosis: Mechanisms and Therapeutic Perspectives.Cells · 2021Review
- Biomarkers of treatment response in patients with progressive multiple sclerosis treated with high-dose pharmaceutical-grade biotin (MD1003).Brain and behavior · 2021Article
- Promoting remyelination in multiple sclerosis.Journal of neurology · 2021Review
- Regulation of Oligodendrocyte Functions: Targeting Lipid Metabolism and Extracellular Matrix for Myelin Repair.Journal of clinical medicine · 2020Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
20 authors at 18 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundChronic 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.
objectiveThe aim of this study was to evaluate whether MD1003 improves vision compared with placebo in MS patients with chronic visual loss.
methodsThe 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.
resultsNinety-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.
conclusionsMD1003 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 registrationEudraCT identifier 2013-002112-27. ClinicalTrials.gov Identifier: NCT02220244
fundingMedDay Pharmaceuticals.
Indexed as
Identifiers
What Socratic holds
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 Socratic graph.