ArticleNeurology. Genetics2022
Efficacy and Safety of Leriglitazone in Patients With Friedreich Ataxia: A Phase 2 Double-Blind, Randomized Controlled Trial (FRAMES).
Registry-linked trialOpen access · goldAbstract read
In one paragraph
Article in Neurology. Genetics, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03917225 (A Double-Blind, Placebo-controlled Study on the Effects of MIN-102 on Biochemical, Imaging, Neurophysiological, and Clinical Markers in Patients With Friedreich's Ataxia), which is not on this map. Cited by 20 papers.
0numbers the graph read from it
0cells of the map it votes in
20citing papers in PubMed
2.6field-weighted citation impact, top 10% 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 Double-Blind, Placebo-controlled Study on the Effects of MIN-102 on Biochemical, Imaging, Neurophysiological, and Clinical Markers in Patients With Friedreich's Ataxia
Who cites it
20 citing papers in PubMed, 28 citations in OpenAlex.
- Small-Molecule NANT Therapeutics Targeting the Brain-Immune Axis in Alzheimer's Disease: Mechanisms, Clinical Progress, and Translational Challenges.Molecules (Basel, Switzerland) · 2026Review
- Evolution of Friedreich's Ataxia Management Across Established and Emerging Therapies-Systematic Review and Meta-Analysis.Journal of clinical medicine · 2026Review
- Value of MRI Outcomes for Preventive and Early-Stage Trials in Spinocerebellar Ataxias 1 and 3.Annals of clinical and translational neurology · 2026Article
- MRI end-points for clinical trials in ataxias: recommendations from the Ataxia Global Initiative MRI Biomarkers Working Group.Nature reviews. Neurology · 2026Review
- Recent developments in Friedreich's ataxia: a state-of-the-art review.Brain communications · 2026Review
- Peripheral frataxin levels govern long-term clinical progression in Friedreich ataxia.BMJ neurology open · 2026Article
- Autosomal Recessive Cerebellar Ataxias: Translating Genes to Therapies.Annals of neurology · 2025Review
- SCA1 in Brazil: Local Cohort Profile and Scientific Contributions.Cerebellum (London, England) · 2025Review
- A global perspective on research advances and future challenges in Friedreich ataxia.Nature reviews. Neurology · 2025Review
- Friedreich Ataxia: An (Almost) 30-Year History After Gene Discovery.Neurology. Genetics · 2025Article
- New and Emerging Drug and Gene Therapies for Friedreich Ataxia.CNS drugs · 2024Review
- Sensitivity of Advanced Magnetic Resonance Imaging to Progression over Six Months in Early Spinocerebellar Ataxia.Movement disorders : official journal of the Movement Disorder Society · 2024Article
- Skeletal Muscle Involvement in Friedreich Ataxia.International journal of molecular sciences · 2024Review
- PPARγ activation ameliorates cognitive impairment and chronic microglial activation in the aftermath of r-mTBI.Journal of neuroinflammation · 2024Article
- Prospective motion correction for cervical spinal cord MRS.Magnetic resonance in medicine · 2024Article
- Longitudinal changes of SARA scale in Friedreich ataxia: Strong influence of baseline score and age at onset.Annals of clinical and translational neurology · 2023Article
- Mitochondrial modulation with leriglitazone as a potential treatment for Rett syndrome.Journal of translational medicine · 2023Article
- Inherited Disorders of Coenzyme A Biosynthesis: Models, Mechanisms, and Treatments.International journal of molecular sciences · 2023Review
- Development of PPARγ Agonists for the Treatment of Neuroinflammatory and Neurodegenerative Diseases: Leriglitazone as a Promising Candidate.International journal of molecular sciences · 2023Review
- PPAR Gamma Agonist Leriglitazone Recovers Alterations Due to Pank2-Deficiency in hiPS-Derived Astrocytes.Pharmaceutics · 2023Article
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 1 institution in 1 country.
Massimo PandolfoUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Kathrin ReetzUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Alejandra DarlingUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Francisco Javier Rodriguez de RiveraUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Pierre-Gilles HenryUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
James JoersUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Christophe LengletUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.ORCID https://orcid.org/0000-0003-4646-3185
Isaac AdanyeguhUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Dinesh DeelchandUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Fanny MochelUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Françoise PoussetUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Sílvia PascualUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Delphine Van den EedeUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Itziar Martin-UgarteUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Anna Vilà-BrauUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Adriana MantillaUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
María PascualUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Marc MartinellUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Uwe MeyaUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Alexandra DurrUniversité Libre de Bruxelles (M. Pandolfo), Brussels, Belgium; Department of Neurology (K.R.), RWTH Aachen University, Germany; JARA-BRAIN Institute Molecular Neuroscience and Neuroimaging (K.R.), Forschungszentrum Jülich GmbH, Germany; Hospital Sant Joan de Déu (A. Darling), Barcelona, Spain; La Paz University Hospital (F.J.R.R.), Madrid, Spain; Department of Radiology (P.-G.H., J.J., C.L., I.A., D.D.), Center for Magnetic Resonance Research, University of Minnesota, Minneapolis; Institut du Cerveau (ICM) (F.M.), Sorbonne Université, Paris, France; AP-HP (F.M.), Groupe Hospitalier Pitié-Salpêtrière, Département de Génétique, Paris, France; Department of Cardiology (F.P.), Sorbonne Université, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France; Institute of Cardiometabolism and Nutrition (ICAN) (F.P.), Pitié-Salpêtrière Hospital, Paris, France; Allies in Cardiovascular Trials Initiatives and Organized Networks (ACTION) Group (F.P.), URC Lariboisière University Hospital, Paris, France; Minoryx Therapeutics SL (S.P., I.M.-U., A.V.-B., A.M., M. Pascual, M.M., U.M.), Barcelona, Spain; Minoryx Therapeutics BE (D.V.E.), Charleroi, Belgium; and Sorbonne Université (A. Durr), Paris Brain Institute, AP-HP, INSERM, CNRS, France.
Jülich Aachen Research Alliance · DE
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background and Objectives: Friedreich ataxia (FRDA) is an autosomal recessive ataxia with no approved treatments. Leriglitazone is a selective peroxisome proliferator-activated receptor γ agonist that crosses the blood-brain barrier and, in preclinical models, improved mitochondrial function and energy production. We assessed effects of leriglitazone in patients with FRDA in a proof-of-concept study. Methods: In this double-blind, randomized controlled trial, eligible participants (age 12-60 years) had genetically confirmed FRDA, a Scale for the Assessment and Rating of Ataxia (SARA) total score <25, and a SARA item 1 score of 2-6, inclusive. Key exclusion criteria were age at FRDA onset ≥25 years and history of cardiac dysfunction. Participants were randomly assigned (2:1) to receive a daily, oral, individualized dose of leriglitazone or placebo for 48 weeks. The primary endpoint was the change from baseline to week 48 in spinal cord area (C2-C3) (measured by MRI). Secondary endpoints included the change from baseline to week 48 in iron accumulation in the dentate nucleus (quantitative susceptibility mapping) and total Results: Overall, 39 patients were enrolled (mean age 24 years; 43.6% women; mean time since symptom onset 10.5 years): 26 patients received leriglitazone (20 completed) and 13 received placebo (12 completed). There was no difference between groups in spinal cord area from baseline to week 48 (least-squares [LS] mean change [standard error (SE)]: leriglitazone, -0.39 [0.55] mm Discussion: The primary endpoint of change in spinal cord area was not met. Secondary endpoints provide evidence supporting proof of concept for leriglitazone mode of action and, with acceptable safety data, support larger studies in patients with FRDA. Trial Registration Information: ClinicalTrials.gov: NCT03917225; EudraCT: 2018-004405-64; submitted April 17, 2019; first patient enrolled April 2, 2019. clinicaltrials.gov/ct2/show/NCT03917225?term=NCT03917225&draw=2&rank=1. Classification of Evidence: This study provides Class I evidence that individualized dosing of leriglitazone, compared with placebo, is not associated with changes in spinal cord area in patients with FRDA.
Identifiers
What OpenQuestion holds
Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390
Registered trials
NCT03917225ClinicalTrials.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.