11 completed, 2 active not recruiting, 1 not yet recruiting, 1 recruiting. 16 Phase 3, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 16 | Completed 11, Active not recruiting 2, Not yet recruiting 1, Recruiting 1, Terminated 1 |
| Phase 2 | 3 | Completed 2, Terminated 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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia Vatiquinone - Friedreich Ataxia | Phase 3 | Active not recruiting | PTC Therapeutics | 130 | 2022-12-08 | 2025-10-30 |
| 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 |
| A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia RT001, Placebo - Friedreich's Ataxia | Phase 3 | Completed | Biojiva LLC | 65 | 2019-10-30 | 2022-05-27 |
| Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia interferon γ-1b - Friedreich's Ataxia | Phase 3 | Completed | Amgen | 38 | 2016-06-28 | 2024-12-10 |
| Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study Interferon γ-1b - Friedreich's Ataxia | Phase 3 | Completed | Amgen | 86 | 2015-12-25 | 2024-12-19 |
| Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Interferon γ-1b, Placebo - Friedreich's Ataxia | Phase 3 | Completed | Amgen | 92 | 2015-06-01 | 2024-12-19 |
| Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI) Idebenone, Placebo - Friedreich's Ataxia | Phase 3 | Completed | Santhera Pharmaceuticals | 29 | 2011-04-01 | 2016-03-09 |
| Efficacy of Riluzole in Hereditary Cerebellar Ataxia riluzole, Placebo comparator - Cerebellar Ataxia | Phase 2 / Phase 3 | Completed | S. Andrea Hospital | 60 | 2010-04-01 | 2015-04-21 |
| Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia varenicline, placebo - Friedreich's Ataxia | Phase 2 / Phase 3 | Terminated | University of South Florida | 28 | 2009-05-01 | 2012-07-24 |
| Effect of Pioglitazone Administered to Patients With Friedreich's Ataxia: Proof of Concept pioglitazone, Placebo - Friedreich's Ataxia | Phase 3 | Completed | Assistance Publique - Hôpitaux de Paris | 40 | 2008-12-01 | 2013-09-04 |
| Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients Idebenone - Friedreich's Ataxia | Phase 3 | Completed | Santhera Pharmaceuticals | 68 | 2008-07-01 | 2023-07-11 |
| Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Idebenone, Placebo - Friedreich's Ataxia | Phase 3 | Completed | Santhera Pharmaceuticals | 70 | 2007-12-01 | 2023-07-11 |
| A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients idebenone, Placebo - Friedreich's Ataxia | Phase 3 | Completed | Santhera Pharmaceuticals | 232 | 2006-04-01 | 2016-06-27 |
Cerebellar Ataxia (1) · Spinocerebellar Ataxias (1)
Brain Diseases (16) · Central Nervous System Diseases (16) · Cerebellar Diseases (16) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Genetic Diseases, Inborn (16) · Heredodegenerative Disorders, Nervous System (16) · Metabolic Diseases (16) · Mitochondrial Diseases (16) · Nervous System Diseases (16) · Neurodegenerative Diseases (16) · Nutritional and Metabolic Diseases (16) · Spinal Cord Diseases (16) · Spinocerebellar Degenerations (16) · Ataxia (1) · Dyskinesias (1)
idebenone (4) · alpha-tocotrienol quinone (3) · interferon gamma-1b (3) · Pioglitazone (1) · RT001 (1) · Riluzole (1) · Varenicline (1) · omaveloxolone (1)
Santhera Pharmaceuticals (4) · Amgen (3) · PTC Therapeutics (3) · Assistance Publique - Hôpitaux de Paris (1) · Biogen (1) · Biojiva LLC (1) · Larimar Therapeutics, Inc. (1) · S. Andrea Hospital (1) · University of South Florida (1)
United States (12) · France (6) · Germany (5) · Brazil (4) · Canada (4) · Italy (4) · Spain (4) · Australia (3) · Austria (3) · Netherlands (3) · United Kingdom (3) · Belgium (2) · New Zealand (2) · Denmark (1) · India (1)
David Lynch, MD (2) · Susan Perlman, MD (2) · Isabelle Husson (1) · Nick Wood, Professor (1) · Paola Giunti, M.D (1) · Peter Milner, MD (1) · Silvia Romano, MD, PhD (1) · Theresa Zesiewicz, M.D. (1)
PMID 21779958, PMID 20697044, PMID 21392622, PMID 26321318, PMID 31495483
2025 (2)