66 completed, 19 recruiting, 8 active not recruiting, 6 not yet recruiting. 36 Phase 2, 22 Phase 1, 16 Phase 3, 10 Not applicable, 2 Early Phase 1.
Follow Friedreich Ataxia: new trials by email Literature Review of Friedreich Ataxia trials Research Report on Friedreich Ataxia
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| GV101 for Friedreich's Ataxia (FA) Treatment GV101, Placebo - Friedreich's Ataxia | Phase 2 | Not yet recruiting | Graviton Bioscience Corporation | 48 | 2026-11-01 | 2026-10-15 |
| A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia Nomlabofusp, Placebo - Friedreich Ataxia, Friedreich's Ataxia | Phase 3 | Recruiting | Larimar Therapeutics, Inc. | 150 | 2026-10-26 | 2026-10-09 |
| A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies Omaveloxolone - Friedreich Ataxia | Recruiting | Biogen | 10 | 2026-10-26 | 2026-07-10 | |
| A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults ATI-105, Placebo - Friedreich Ataxia, Cardiomyopathies | Phase 1 | Not yet recruiting | Adjuvia Therapeutics, Inc. | 60 | 2026-10-26 | 2026-10-09 |
| A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World Friedreich's Ataxia | Not yet recruiting | Biogen | 40 | 2026-10-15 | 2026-10-21 | |
| Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA) Vatiquinone - Friedreich's Ataxia | Phase 3 | Not yet recruiting | PTC Therapeutics | 120 | 2026-08-15 | 2026-08-14 |
| Digital Outcome Assessment Using AI Active Gaming and Motion Capture in Friedreich Ataxia Laboratory validation session (VSimulator), Home-based active gaming assessment - Friedreich Ataxia | Not yet recruiting | University of Exeter | 40 | 2026-07-30 | 2026-07-28 | |
| Cardiac Output and Fatigue in Friedreich's Ataxia Aerobic Exercise - Friedreich's Ataxia | Not applicable | Recruiting | Scott Barbuto | 30 | 2026-07-15 | 2026-09-22 |
| Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy LX2006, Usual Care - Friedreich Ataxia, Cardiomyopathy, Secondary | Phase 2 | Recruiting | Lexeo Therapeutics | 26 | 2026-06-25 | 2026-09-02 |
| Friedreich Ataxia Nerve Ultrasund Nerve ultrasound - routine exam - Friedreich Ataxia | Recruiting | Centre Hospitalier Universitaire de Nice | 14 | 2025-12-01 | 2026-04-02 | |
| A Study of SGT-212 Gene Therapy in Friedreich's Ataxia SGT-212 - Friedreich's Ataxia (FA) | Phase 1 | Recruiting | Solid Biosciences Inc. | 10 | 2025-10-22 | 2026-09-29 |
| Clinical Course Of Disease In Participants With FA-CM Friedreich Ataxia, Cardiomyopathy | Recruiting | Lexeo Therapeutics | 65 | 2025-09-30 | 2026-10-19 | |
| The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. Assistive gait devices combined with physiotherapy - Inclusion Body Myositis, Myotonic Dystrophy 1 | Not applicable | Enrolling by invitation | LMU Klinikum | 30 | 2025-09-01 | 2025-07-18 |
| Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome. HD-EEG recordings, combined with cognitive and motor assessment - Friedreich's Ataxia, Motor Disorders | Not applicable | Recruiting | IRCCS Eugenio Medea | 30 | 2025-07-07 | 2025-07-31 |
| A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia Omaveloxolone, Placebo - Friedreich Ataxia | Phase 3 | Active not recruiting | Biogen | 255 | 2025-06-09 | 2026-10-02 |
| A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia DT-216P2 - Friedreich Ataxia | Phase 1 / Phase 2 | Recruiting | Design Therapeutics, Inc. | 20 | 2025-06-01 | 2025-05-18 |
| Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich's Ataxia SKYCLARYS (omaveloxolone) - Friedreich Ataxia | Recruiting | Centre Hospitalier Universitaire de Nice | 40 | 2025-06-01 | 2026-04-29 | |
| Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia Geneticlly confirmed disease causing FXN mutatuion, Healthy Control - Friedreich Ataxia, Rare Diseases | Withdrawn | Friedreich's Ataxia Research Alliance | 2025-05-01 | 2025-06-08 | ||
| Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia Balance training over stabilometric platform, Balance training by means of conventional rehabilitation - Friedreich Ataxia | Not applicable | Recruiting | IRCCS Eugenio Medea | 24 | 2025-02-03 | 2025-05-02 |
| A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants DT-216P2, Saline - Friedreich Ataxia | Phase 1 | Not yet recruiting | Design Therapeutics, Inc. | 36 | 2025-02-01 | 2025-02-21 |
Showing 20 of 110. Sign in to see all of them.
Ataxia (12) · Cardiomyopathies (10) · Rare Diseases (6) · Mitochondrial Diseases (4) · Spinocerebellar Ataxias (4) · Genetic Diseases, Inborn (3) · Lambert-Eaton Myasthenic Syndrome (3) · Machado-Joseph Disease (3) · Neuromuscular Diseases (3) · Cerebellar Ataxia (2) · Glycogen Storage Disease Type II (2) · Guillain-Barre Syndrome (2) · Heart Diseases (2) · Hereditary Sensory and Motor Neuropathy (2)
Brain Diseases (110) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (110) · Heredodegenerative Disorders, Nervous System (110) · Metabolic Diseases (110) · Nutritional and Metabolic Diseases (110) · Spinal Cord Diseases (110) · Central Nervous System Diseases (109) · Cerebellar Diseases (109) · Nervous System Diseases (109) · Neurodegenerative Diseases (108) · Spinocerebellar Degenerations (108) · Genetic Diseases, Inborn (107) · Mitochondrial Diseases (106) · Pathological Conditions, Signs and Symptoms (25) · Signs and Symptoms (16)
alpha-tocotrienol quinone (7) · idebenone (7) · omaveloxolone (6) · Blood Specimen Collection (4) · interferon gamma-1b (4) · Deferiprone (2) · Epoetin Alfa (2) · Exercise Test (2) · Interferon-gamma (2) · RT001 (2) · Resveratrol (2) · Acetylcarnitine (1) · Bupropion (1) · Calcitriol (1) · Catechin (1)
Larimar Therapeutics, Inc. (8) · Biogen (6) · Children's Hospital of Philadelphia (6) · PTC Therapeutics (6) · Design Therapeutics, Inc. (4) · IRCCS Eugenio Medea (4) · Santhera Pharmaceuticals (4) · University of South Florida (4) · Amgen (3) · Friedreich's Ataxia Research Alliance (3) · Institut National de la Santé Et de la Recherche Médicale, France (3) · Lexeo Therapeutics (3) · National Institute of Neurological Disorders and Stroke (NINDS) (3) · University of Florida (3) · ApoPharma (2)
United States (70) · France (22) · Italy (21) · Germany (17) · Australia (14) · Spain (12) · Austria (10) · Canada (10) · Belgium (8) · United Kingdom (8) · Brazil (7) · Netherlands (5) · Czechia (4) · New Zealand (4) · India (3)
David Lynch, MD, PhD (4) · David Lynch, MD (3) · Magdy Shenouda, M.D. (3) · Arnold Munnich, M.D. (2) · David Lynch, MD PhD (2) · Massimo Pandolfo, M.D. (2) · Ronald G Crystal, MD (2) · Susan Perlman, MD (2) · Theresa A Zesiewicz, MD (2) · Alberto Benussi, MD (1) · Alessandro Filla, MD (1)
PMID 40739200, PMID 17562928, PMID 21779958, PMID 42307907, PMID 16267188, PMID 16849828, PMID 16900450, PMID 17826341, PMID 20090835, PMID 20599042, PMID 20697044, PMID 21392622, PMID 22003934, PMID 22633670, PMID 22744651, PMID 26321318, PMID 27839651, PMID 28168524, PMID 28950889, PMID 29279305, PMID 30097477, PMID 30135258, PMID 30286776, PMID 30656180, PMID 31495483, PMID 32677106, PMID 32746884, PMID 33068037, PMID 33305808, PMID 33324899, PMID 33770527, PMID 34713932, PMID 35817567, PMID 36410013, PMID 36524101, PMID 36693691, PMID 37171121, PMID 37527887, PMID 38140802, PMID 39201893, PMID 39738982, PMID 40119735, PMID 40207026, PMID 40614535, PMID 40767617, PMID 41014100
2025 (3), 2008 (1), 2019 (1), R61HL151355 (1), UL1TR000064 (1)