29 completed. 29 Phase 2, 6 Phase 1, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 29 | Completed 29 |
| Phase 1 | 6 | Completed 6 |
| Phase 3 | 2 | Completed 2 |
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 |
|---|---|---|---|---|---|---|
| A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia Vatiquinone - Friedreich Ataxia | Phase 2 | Completed | PTC Therapeutics | 5 | 2022-10-12 | 2025-08-22 |
| A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia CTI-1601, Placebo - Friedreich Ataxia | Phase 2 | Completed | Larimar Therapeutics, Inc. | 28 | 2022-09-21 | 2024-11-29 |
| Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA) Artesunate Oral Product - Friedreich Ataxia | Phase 1 / Phase 2 | Completed | Institut National de la Santé Et de la Recherche Médicale, France | 20 | 2022-05-06 | 2026-05-20 |
| FRDA Investigator Initiated Study (IIS) With Elamipretide Elamipretide - Friedreich Ataxia | Phase 1 / Phase 2 | Completed | Children's Hospital of Philadelphia | 20 | 2022-03-03 | 2025-12-12 |
| NAD+ Precursor Supplementation in Friedreich's Ataxia MIB-626 - Friedreich Ataxia | Phase 2 | Completed | Metro International Biotech, LLC | 7 | 2021-05-17 | 2023-07-17 |
| A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia Vatiquinone, Placebo - Friedreich Ataxia | Phase 2 / Phase 3 | Completed | PTC Therapeutics | 146 | 2020-12-17 | 2026-04-13 |
| Safety and Efficacy of Etravirine in Friedreich Ataxia Patients Etravirine Tablets - Friedreich Ataxia | Phase 2 | Completed | IRCCS Eugenio Medea | 30 | 2020-09-17 | 2024-12-04 |
| Micronised Resveratrol as a Treatment for Friedreich Ataxia Resveratrol - Friedreich Ataxia | Phase 2 | Completed | Murdoch Childrens Research Institute | 25 | 2019-05-23 | 2024-05-22 |
| A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients MIN-102, Placebo - Friedreich Ataxia | Phase 2 | Completed | Minoryx Therapeutics, S.L. | 39 | 2019-03-26 | 2022-10-13 |
| Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia TAK-831, TAK-831 Placebo - Friedreich Ataxia | Phase 2 | Completed | Neurocrine Biosciences | 67 | 2017-11-08 | 2021-06-14 |
| (+) Epicatechin to Treat Friedreich's Ataxia (+)-Epicatechin - Friedreich's Ataxia | Phase 2 | Completed | Ralitza Gavrilova | 10 | 2016-09-01 | 2019-12-05 |
| Open Trail of γIFN for Friedreich Ataxia gamma interferon - Friedreich Ataxia | Phase 2 | Completed | IRCCS Eugenio Medea | 12 | 2016-06-26 | 2020-02-26 |
| A First in Human Study of RT001 in Patients With Friedreich's Ataxia Low dose cohort, High dose cohort - Friedreich's Ataxia | Phase 1 / Phase 2 | Completed | Biojiva LLC | 19 | 2015-08-01 | 2020-11-27 |
| A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg - Friedreich Ataxia | Phase 2 | Completed | Biogen | 172 | 2015-01-31 | 2026-09-03 |
| EPI-743 in Friedreich's Ataxia Point Mutations EPI-743 - Friedreich's Ataxia | Phase 2 | Completed | University of South Florida | 4 | 2013-10-01 | 2016-06-29 |
| Interferon Gamma-1b in Friedreich Ataxia (FRDA) Interferon Gamma-1b - Friedreich Ataxia | Phase 2 | Completed | Children's Hospital of Philadelphia | 12 | 2013-08-01 | 2021-04-13 |
| A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels in FRDA Patients gamma interferon - Friedreich Ataxia | Phase 2 | Completed | Azienda Policlinico Umberto I | 10 | 2013-05-01 | 2017-04-21 |
| Efficacy Study of Epoetin Alfa in Friedreich Ataxia Epoetin alfa, Placebo - Friedreich Ataxia | Phase 2 | Completed | Federico II University | 56 | 2013-01-01 | 2015-08-11 |
| Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia Placebo, EPI-743 400 mg, EPI-743 200 mg - Friedreich's Ataxia | Phase 2 | Completed | PTC Therapeutics | 60 | 2012-12-31 | 2020-08-31 |
| A Study of Resveratrol as Treatment for Friedreich Ataxia Resveratrol - Friedreich Ataxia | Phase 1 / Phase 2 | Completed | Murdoch Childrens Research Institute | 27 | 2011-04-01 | 2014-01-22 |
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Ataxia (3) · Cardiomyopathies (1) · Cerebellar Ataxia (1) · Mitochondrial Diseases (1) · Spinocerebellar Ataxias (1)
Brain Diseases (29) · Central Nervous System Diseases (29) · Cerebellar Diseases (29) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (29) · Genetic Diseases, Inborn (29) · Heredodegenerative Disorders, Nervous System (29) · Metabolic Diseases (29) · Nervous System Diseases (29) · Neurodegenerative Diseases (29) · Nutritional and Metabolic Diseases (29) · Spinal Cord Diseases (29) · Spinocerebellar Degenerations (29) · Mitochondrial Diseases (28) · Dyskinesias (4) · Neurologic Manifestations (4)
alpha-tocotrienol quinone (4) · Deferiprone (2) · Epoetin Alfa (2) · Interferon-gamma (2) · Resveratrol (2) · Catechin (1) · Cohort Studies (1) · Ginkgo biloba extract (1) · ORF 50 transactivator (1) · RT001 (1) · Riluzole (1) · arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide (1) · etravirine (1) · idebenone (1) · interferon gamma-1b (1)
PTC Therapeutics (3) · ApoPharma (2) · Children's Hospital of Philadelphia (2) · Federico II University (2) · IRCCS Eugenio Medea (2) · Murdoch Childrens Research Institute (2) · Assistance Publique - Hôpitaux de Paris (1) · Azienda Policlinico Umberto I (1) · Biogen (1) · Biojiva LLC (1) · H. Lundbeck A/S (1) · Institut National de la Santé Et de la Recherche Médicale, France (1) · Ipsen (1) · Larimar Therapeutics, Inc. (1) · Metro International Biotech, LLC (1)
United States (14) · Italy (10) · France (7) · Australia (5) · Spain (4) · Belgium (3) · Germany (3) · Austria (2) · Canada (2) · Brazil (1) · New Zealand (1) · United Kingdom (1)
David Lynch, MD, PhD (3) · Arnold Munnich, M.D. (2) · Massimo Pandolfo, M.D. (2) · Alessandro Filla, MD (1) · Alexandra Durr (1) · Andrea Martinuzzi, MD (1) · Arnold Munnich, MD (1) · Carlo Casali, MD (1) · Curtis Scribner, MD (1) · Franco Taroni (1) · Franco Taroni, M.D. (1)
PMID 17826341, PMID 20090835, PMID 20599042, PMID 22633670, PMID 22744651, PMID 26321318, PMID 30656180, PMID 32677106, PMID 33068037, PMID 36524101, PMID 39201893, PMID 40614535
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