| Active Not Recruiting | A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dyst Duchenne Muscular Dystrophy | Phase 4 | 2024-11-10 |
| Completed | Evaluation of Vamorolone CYP3A4 Induction on Midazolam (a Sensitive CYP 3A4 Substrate) Pharmacokinetics Drug Interaction | Phase 1 | 2024-08-13 |
| Completed | Evaluation of Vamorolone Mineralocorticoid Receptor Antagonism in Healthy Subjects Pharmacodynamic | Phase 1 | 2024-06-05 |
| Completed | A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (D Duchenne Muscular Dystrophy | Phase 2 | 2022-03-21 |
| Completed | Clinical Study to Investigate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of POL6014 in Patien Cystic Fibrosis | Phase 1 / Phase 2 | 2018-11-08 |
| Terminated | Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) Duchenne Muscular Dystrophy | Phase 3 | 2018-07-04 |
| Terminated | A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucoc Duchenne Muscular Dystrophy (DMD) | Phase 3 | 2016-09-01 |
| Completed | Post Authorisation Safety Study With Raxone in LHON Patients Leber's Hereditary Optic Neuropathy (LHON) | — | 2016-09-01 |
| Completed | A Study to Assess the Potential for Pre-systemic Inhibition of CYP3A by Idebenone Using Midazolam as a Substra Drug-Drug Interaction | Phase 1 | 2016-09-01 |
| Completed | Study to Assess the Efficacy and Safety of Raxone in LHON Patients Leber's Hereditary Optic Neuropathy (LHON) | Phase 4 | 2016-05-01 |
| Completed | Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LH Leber's Hereditary Optic Neuropathy (LHON) | — | 2016-05-01 |
| Completed | Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Le Congenital Muscular Dystrophy | Phase 1 | 2014-12-01 |
| Completed | Leber Hereditary Optic Neuropathy (LHON) Historical Case Record Survey Leber Hereditary Optic Neuropathy (LHON) | — | 2013-08-01 |
| Completed | RHODOS Follow-up Single-visit Study Leber's Hereditary Optic Neuropathy | — | 2011-09-01 |
| Completed | Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROT Friedreich's Ataxia | Phase 3 | 2011-04-01 |
| Completed | Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD) Muscular Dystrophy, Duchenne, Ambulatory Care | Phase 3 | 2009-07-01 |
| Completed | Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension) Duchenne Muscular Dystrophy | Phase 2 | 2008-09-01 |
| Completed | Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients Friedreich's Ataxia | Phase 3 | 2008-07-01 |
| Completed | Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Friedreich's Ataxia | Phase 3 | 2007-12-01 |
| Completed | Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neu Leber's Hereditary Optic Neuropathy | Phase 2 | 2007-11-01 |
| Completed | Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension) Freidreich's Ataxia | Phase 3 | 2007-06-01 |
| Completed | A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patie Friedreich's Ataxia | Phase 3 | 2006-04-01 |
| Completed | Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dyst Duchenne Muscular Dystrophy (DMD) | Phase 2 | 2005-10-01 |
| No Longer Available | Expanded Access Program for Idebenone in Patients With Leber's Hereditary Optic Neuropathy Who Completed the L Leber's Hereditary Optic Neuropathy | — | — |
| Withdrawn | Study With Idebenone in Patients With Chronic Vision Loss Due to Leber's Hereditary Optic Neuropathy (LHON) Leber's Hereditary Optic Neuropathy | Phase 3 | — |
| Available | Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy | — | — |
| No Longer Available | Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD) Duchenne Muscular Dystrophy | — | — |