Trial reportNeurology(R) neuroimmunology & neuroinflammation2022
Efficacy and Safety of Masitinib in Progressive Forms of Multiple Sclerosis: A Randomized, Phase 3, Clinical Trial.
2 registry-linked trialsOpen access · goldAbstract readClinical Trial, Phase IIIRandomized Controlled Trial
In one paragraph
Trial report in Neurology(R) neuroimmunology & neuroinflammation, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 40 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
40citing papers in PubMed, 2 pooled it
9.0field-weighted citation impact, top 1% of its field
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.
A 96 Week, Prospective, Multicentre, Randomized, Double-blind, Placebo-controlled, 2 Parallel-groups, Phase 3 Study to Compare Efficacy and Safety of Masitinib 4.5 mg/kg/Day Versus Placebo in the Treatment of Patients With Primary Progressive or Relapse-free Secondary Progressive Multiple Sclerosis
A 96-Week, Prospective, Multicenter, Randomised, Double-Blind, Placebo-Controlled, Phase 3 Study to Compare Efficacy and Safety of Masitinib Dose Titration to 4.5 mg/kg/Day Versus Placebo in the Treatment of Patients With Primary Progressive or Secondary Progressive Multiple Sclerosis Without Relapse
Who cites it
40 citing papers in PubMed, 2 syntheses or guidelines pooled it, 64 citations in OpenAlex.
- Safety of masitinib in patients with neurodegenerative diseases: a meta-analysis of randomized controlled trials.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024Pooled it
- Safety and efficacy of tyrosine kinase inhibitors for the treatment of multiple sclerosis: A systematic review and meta-analysis from randomized controlled trials.Frontiers in neurology · 2022Pooled it
- Review
- Effect of intrathecal injection of mesenchymal stem cell-neural progenitors on cerebrospinal fluid biomarkers in progressive multiple sclerosis.Stem cells translational medicine · 2026Article
- Small-Molecule NANT Therapeutics Targeting the Brain-Immune Axis in Alzheimer's Disease: Mechanisms, Clinical Progress, and Translational Challenges.Molecules (Basel, Switzerland) · 2026Review
- Glial cells in chronic inflammation: diversity, dysfunction and therapeutic targeting.Nature reviews. Immunology · 2026Review
- Astrocyte Functional Heterogeneity in Multiple Sclerosis.Journal of clinical neurology (Seoul, Korea) · 2026Review
- Neuroendocrine-associated epigenetic factors in cellular senescence: mechanisms and therapeutic implications.Biogerontology · 2026Review
- Translational molecular imaging and drug development in multiple sclerosis.Theranostics · 2026Review
- Revolutionizing Therapeutic Approaches Against Pathophysiology of Alzheimer's Disease: A Therapeutic Review.Current aging science · 2026Review
- The Potential of β-Synuclein-Specific Regulatory T Cell Therapy as a Treatment for Progressive Multiple Sclerosis.International journal of molecular sciences · 2025Review
- Repair mechanisms of the central nervous system: From axon sprouting to remyelination.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2025Review
- Progressive multiple sclerosis: Evaluating current therapies and exploring future treatment strategies.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2025Review
- Update on novel multiple sclerosis treatments: from dismal defeat to scintillating success.Current opinion in neurology · 2025Review
- Microglia/Macrophages in Autoimmune Demyelinating Encephalomyelitis (Multiple Sclerosis/Neuromyelitis Optica).International journal of molecular sciences · 2025Review
- Modulation of Peripheral Mast Cell and Brain Microglia Axis via Kinase Inhibition.Metabolites · 2025Article
- Alzheimer's Disease and Vascular Dementia, Connecting and Differentiating Features.Current Alzheimer research · 2025Review
- ALS: A Silent Slayer of Motor Neurons. Traditional Chinese Herbal Medicine as an Effective Therapy.Current pharmaceutical design · 2025Review
- Current challenges in secondary progressive multiple sclerosis: diagnosis, activity detection and treatment.Frontiers in immunology · 2025Review
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 1 institution in 4 countries.
Patrick VermerschFrom the Univ. Lille (P.V.), UMR Inserm U1172, CHU Lille, FHU Precise, France; Neurology Department (L.B.-R.), Hospital Arnau de Vilanova de Lleida, Spain; Mellen Center for Multiple Sclerosis (R.J.F.), Neurological Institute, Cleveland Clinic, OH; Experimental and Clinical Research Center and NeuroCure Clinical Research Center (F.P.), Max Delbrueck Center for Molecular Medicine and Charité Universitaetsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Germany; Neurology Department (L.R.-T.), Dr Josep Trueta University Hospital, Girona; Neurodegeneration and Neuroinflammation Research Group (L.R.-T.), IDIBGI, Salt; Medical Science Department (L.R.-T.), University of Girona, Spain; Neurology Department (M.S.), Jena University Hospital, Germany; AB Science (A.M., C.M., O.H.), Paris, France; Imagine Institute (O.H.), INSERM UMR 1163, Laboratory of Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutic Implication, Hôpital Necker, Paris, France; and MA LEK AM Maciejowscy SC Centrum Terapii SM (M.M.), Katowice, Poland. patrick.vermersch@univ-lille.fr.
Luis Brieva-RuizFrom the Univ. Lille (P.V.), UMR Inserm U1172, CHU Lille, FHU Precise, France; Neurology Department (L.B.-R.), Hospital Arnau de Vilanova de Lleida, Spain; Mellen Center for Multiple Sclerosis (R.J.F.), Neurological Institute, Cleveland Clinic, OH; Experimental and Clinical Research Center and NeuroCure Clinical Research Center (F.P.), Max Delbrueck Center for Molecular Medicine and Charité Universitaetsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Germany; Neurology Department (L.R.-T.), Dr Josep Trueta University Hospital, Girona; Neurodegeneration and Neuroinflammation Research Group (L.R.-T.), IDIBGI, Salt; Medical Science Department (L.R.-T.), University of Girona, Spain; Neurology Department (M.S.), Jena University Hospital, Germany; AB Science (A.M., C.M., O.H.), Paris, France; Imagine Institute (O.H.), INSERM UMR 1163, Laboratory of Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutic Implication, Hôpital Necker, Paris, France; and MA LEK AM Maciejowscy SC Centrum Terapii SM (M.M.), Katowice, Poland.
Robert J FoxFrom the Univ. Lille (P.V.), UMR Inserm U1172, CHU Lille, FHU Precise, France; Neurology Department (L.B.-R.), Hospital Arnau de Vilanova de Lleida, Spain; Mellen Center for Multiple Sclerosis (R.J.F.), Neurological Institute, Cleveland Clinic, OH; Experimental and Clinical Research Center and NeuroCure Clinical Research Center (F.P.), Max Delbrueck Center for Molecular Medicine and Charité Universitaetsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Germany; Neurology Department (L.R.-T.), Dr Josep Trueta University Hospital, Girona; Neurodegeneration and Neuroinflammation Research Group (L.R.-T.), IDIBGI, Salt; Medical Science Department (L.R.-T.), University of Girona, Spain; Neurology Department (M.S.), Jena University Hospital, Germany; AB Science (A.M., C.M., O.H.), Paris, France; Imagine Institute (O.H.), INSERM UMR 1163, Laboratory of Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutic Implication, Hôpital Necker, Paris, France; and MA LEK AM Maciejowscy SC Centrum Terapii SM (M.M.), Katowice, Poland.ORCID 0000-0002-4263-3717
Friedemann PaulFrom the Univ. Lille (P.V.), UMR Inserm U1172, CHU Lille, FHU Precise, France; Neurology Department (L.B.-R.), Hospital Arnau de Vilanova de Lleida, Spain; Mellen Center for Multiple Sclerosis (R.J.F.), Neurological Institute, Cleveland Clinic, OH; Experimental and Clinical Research Center and NeuroCure Clinical Research Center (F.P.), Max Delbrueck Center for Molecular Medicine and Charité Universitaetsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Germany; Neurology Department (L.R.-T.), Dr Josep Trueta University Hospital, Girona; Neurodegeneration and Neuroinflammation Research Group (L.R.-T.), IDIBGI, Salt; Medical Science Department (L.R.-T.), University of Girona, Spain; Neurology Department (M.S.), Jena University Hospital, Germany; AB Science (A.M., C.M., O.H.), Paris, France; Imagine Institute (O.H.), INSERM UMR 1163, Laboratory of Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutic Implication, Hôpital Necker, Paris, France; and MA LEK AM Maciejowscy SC Centrum Terapii SM (M.M.), Katowice, Poland.
Lluis Ramio-TorrentaFrom the Univ. Lille (P.V.), UMR Inserm U1172, CHU Lille, FHU Precise, France; Neurology Department (L.B.-R.), Hospital Arnau de Vilanova de Lleida, Spain; Mellen Center for Multiple Sclerosis (R.J.F.), Neurological Institute, Cleveland Clinic, OH; Experimental and Clinical Research Center and NeuroCure Clinical Research Center (F.P.), Max Delbrueck Center for Molecular Medicine and Charité Universitaetsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Germany; Neurology Department (L.R.-T.), Dr Josep Trueta University Hospital, Girona; Neurodegeneration and Neuroinflammation Research Group (L.R.-T.), IDIBGI, Salt; Medical Science Department (L.R.-T.), University of Girona, Spain; Neurology Department (M.S.), Jena University Hospital, Germany; AB Science (A.M., C.M., O.H.), Paris, France; Imagine Institute (O.H.), INSERM UMR 1163, Laboratory of Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutic Implication, Hôpital Necker, Paris, France; and MA LEK AM Maciejowscy SC Centrum Terapii SM (M.M.), Katowice, Poland.
Matthias SchwabFrom the Univ. Lille (P.V.), UMR Inserm U1172, CHU Lille, FHU Precise, France; Neurology Department (L.B.-R.), Hospital Arnau de Vilanova de Lleida, Spain; Mellen Center for Multiple Sclerosis (R.J.F.), Neurological Institute, Cleveland Clinic, OH; Experimental and Clinical Research Center and NeuroCure Clinical Research Center (F.P.), Max Delbrueck Center for Molecular Medicine and Charité Universitaetsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Germany; Neurology Department (L.R.-T.), Dr Josep Trueta University Hospital, Girona; Neurodegeneration and Neuroinflammation Research Group (L.R.-T.), IDIBGI, Salt; Medical Science Department (L.R.-T.), University of Girona, Spain; Neurology Department (M.S.), Jena University Hospital, Germany; AB Science (A.M., C.M., O.H.), Paris, France; Imagine Institute (O.H.), INSERM UMR 1163, Laboratory of Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutic Implication, Hôpital Necker, Paris, France; and MA LEK AM Maciejowscy SC Centrum Terapii SM (M.M.), Katowice, Poland.
Alain MoussyFrom the Univ. Lille (P.V.), UMR Inserm U1172, CHU Lille, FHU Precise, France; Neurology Department (L.B.-R.), Hospital Arnau de Vilanova de Lleida, Spain; Mellen Center for Multiple Sclerosis (R.J.F.), Neurological Institute, Cleveland Clinic, OH; Experimental and Clinical Research Center and NeuroCure Clinical Research Center (F.P.), Max Delbrueck Center for Molecular Medicine and Charité Universitaetsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Germany; Neurology Department (L.R.-T.), Dr Josep Trueta University Hospital, Girona; Neurodegeneration and Neuroinflammation Research Group (L.R.-T.), IDIBGI, Salt; Medical Science Department (L.R.-T.), University of Girona, Spain; Neurology Department (M.S.), Jena University Hospital, Germany; AB Science (A.M., C.M., O.H.), Paris, France; Imagine Institute (O.H.), INSERM UMR 1163, Laboratory of Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutic Implication, Hôpital Necker, Paris, France; and MA LEK AM Maciejowscy SC Centrum Terapii SM (M.M.), Katowice, Poland.
Colin MansfieldFrom the Univ. Lille (P.V.), UMR Inserm U1172, CHU Lille, FHU Precise, France; Neurology Department (L.B.-R.), Hospital Arnau de Vilanova de Lleida, Spain; Mellen Center for Multiple Sclerosis (R.J.F.), Neurological Institute, Cleveland Clinic, OH; Experimental and Clinical Research Center and NeuroCure Clinical Research Center (F.P.), Max Delbrueck Center for Molecular Medicine and Charité Universitaetsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Germany; Neurology Department (L.R.-T.), Dr Josep Trueta University Hospital, Girona; Neurodegeneration and Neuroinflammation Research Group (L.R.-T.), IDIBGI, Salt; Medical Science Department (L.R.-T.), University of Girona, Spain; Neurology Department (M.S.), Jena University Hospital, Germany; AB Science (A.M., C.M., O.H.), Paris, France; Imagine Institute (O.H.), INSERM UMR 1163, Laboratory of Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutic Implication, Hôpital Necker, Paris, France; and MA LEK AM Maciejowscy SC Centrum Terapii SM (M.M.), Katowice, Poland.
Olivier HermineFrom the Univ. Lille (P.V.), UMR Inserm U1172, CHU Lille, FHU Precise, France; Neurology Department (L.B.-R.), Hospital Arnau de Vilanova de Lleida, Spain; Mellen Center for Multiple Sclerosis (R.J.F.), Neurological Institute, Cleveland Clinic, OH; Experimental and Clinical Research Center and NeuroCure Clinical Research Center (F.P.), Max Delbrueck Center for Molecular Medicine and Charité Universitaetsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Germany; Neurology Department (L.R.-T.), Dr Josep Trueta University Hospital, Girona; Neurodegeneration and Neuroinflammation Research Group (L.R.-T.), IDIBGI, Salt; Medical Science Department (L.R.-T.), University of Girona, Spain; Neurology Department (M.S.), Jena University Hospital, Germany; AB Science (A.M., C.M., O.H.), Paris, France; Imagine Institute (O.H.), INSERM UMR 1163, Laboratory of Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutic Implication, Hôpital Necker, Paris, France; and MA LEK AM Maciejowscy SC Centrum Terapii SM (M.M.), Katowice, Poland.ORCID 0000-0003-2574-3874
Maciej MaciejowskiFrom the Univ. Lille (P.V.), UMR Inserm U1172, CHU Lille, FHU Precise, France; Neurology Department (L.B.-R.), Hospital Arnau de Vilanova de Lleida, Spain; Mellen Center for Multiple Sclerosis (R.J.F.), Neurological Institute, Cleveland Clinic, OH; Experimental and Clinical Research Center and NeuroCure Clinical Research Center (F.P.), Max Delbrueck Center for Molecular Medicine and Charité Universitaetsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Germany; Neurology Department (L.R.-T.), Dr Josep Trueta University Hospital, Girona; Neurodegeneration and Neuroinflammation Research Group (L.R.-T.), IDIBGI, Salt; Medical Science Department (L.R.-T.), University of Girona, Spain; Neurology Department (M.S.), Jena University Hospital, Germany; AB Science (A.M., C.M., O.H.), Paris, France; Imagine Institute (O.H.), INSERM UMR 1163, Laboratory of Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutic Implication, Hôpital Necker, Paris, France; and MA LEK AM Maciejowscy SC Centrum Terapii SM (M.M.), Katowice, Poland.
AB07002 Study Group
Inserm · FR
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
objectivesMasitinib is a selective tyrosine kinase inhibitor, targeting innate immune cells (mast cells and microglia) that are involved in the pathophysiology of progressive multiple sclerosis (MS). Study AB07002 assessed oral masitinib in patients with progressive MS who were progressing but not clinically active.
methodsThis randomized, double-blind, 2 parallel-group, placebo-controlled trial assessing 2 dose levels of masitinib vs equivalent placebo was conducted at 116 hospital clinics and specialized MS centers in 20 countries. Randomization (2:1) with minimization was performed centrally using an automated system. Patients, physicians, and outcome assessors remained masked to treatment group allocation. Patients with primary progressive MS (PPMS) or nonactive secondary progressive MS (nSPMS) without relapse for ≥2 years, aged 18-75 years, with baseline Expanded Disability Status Scale (EDSS) 2.0-6.0, and regardless of time from onset were treated for 96 weeks. The primary end point was overall EDSS change from baseline using repeated measures (generalized estimating equation, timeframe W12-W96, measured every 12 weeks), with positive values indicating increased clinical deterioration. Efficacy and safety were assessed in all randomly assigned and treated patients.
resultsA total of 611 patients were randomized; 301 in the masitinib 4.5 mg/kg/d parallel group and 310 in the uptitrated masitinib 6.0 mg/kg/d parallel group. Masitinib (4.5 mg/kg/d) (n = 199) showed significant benefit over placebo (n = 101) according to the primary end point, 0.001 vs 0.098, respectively, with a between-group difference of -0.097 (97% CI -0.192 to -0.002); DISCUSSION: Masitinib (4.5 mg/kg/d) can benefit people with PPMS and nSPMS. A confirmatory phase 3 study will be initiated to substantiate these data. TRIAL REGISTRATION INFORMATION: The first participant was randomized to study AB07002 on August 25, 2011. The trial was registered with the European Clinical Trials Database (#EudraCT 2010-021219-17) on July 1, 2011 (clinicaltrialsregister.eu/ctr-search/trial/2010-021219-17/ES) and with ClinicalTrials.gov (#NCT01433497) on September 14, 2011 (clinicaltrials.gov/ct2/show/NCT01433497). CLASSIFICATION OF EVIDENCE: This study provides Class II evidence that masitinib 4.5 mg/kg/d decreased progression of disability, measured by the EDSS, in adults with PPMS or patients with nSPMS (with no exacerbations in the last 2 years).
Indexed as
Disease ProgressionAdolescentAdultBenzamidesDouble-Blind MethodFemaleHumansMaleMiddle AgedMultiple Sclerosis, Chronic ProgressiveOutcome Assessment, Health CarePiperidinesProtein Kinase InhibitorsPyridinesThiazolesYoung AdultBenzamidesmasitinibPiperidinesProtein Kinase InhibitorsPyridinesThiazoles
Identifiers
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LicenceCC BY-NC-ND
Read underepoch 390
Registered trials
NCT01433497ClinicalTrials.gov
NCT05441488ClinicalTrials.gov
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.