Orphan Neuromuscular Disease Targets
Total Trials
32
As Lead Sponsor
27
As Collaborator
5
Total Enrollment
2,823
NCT00654784
Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy
Phase: Phase 2
Role: Lead Sponsor
Start: Oct 31, 2005
Completion: Aug 31, 2007
NCT00905268
A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients
Phase: Phase 3
Start: Apr 30, 2006
Completion: Jan 31, 2010
NCT00993967
Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)
Start: Jun 30, 2007
Completion: Jun 30, 2012
NCT00559871
Safety and Efficacy Study of Fipamezole in Treatment of Motor Dysfunctions in Parkinson's Disease
Role: Collaborator
Start: Oct 31, 2007
Completion: May 31, 2009
NCT00747487
Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy
Start: Nov 30, 2007
Completion: Feb 28, 2010
NCT00537680
Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia
Start: Dec 31, 2007
Completion: Apr 30, 2009
NCT00697073
Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients
Start: Jul 31, 2008
Completion: May 31, 2010
NCT00758225
Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
Start: Sep 30, 2008
Completion: Jan 31, 2011
NCT00758849
Fipamezole in Neurogenic Orthostatic Hypotension
NCT00887562
Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes
Start: May 31, 2009
Completion: Jul 31, 2012
NCT01027884
Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
Start: Jul 31, 2009
Completion: Apr 30, 2014
NCT01303406
Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)
Start: Apr 30, 2011
NCT01421381
RHODOS Follow-up Single-visit Study
Phase: N/A
Start: Sep 30, 2011
Completion: Dec 31, 2011
NCT01892943
Leber Hereditary Optic Neuropathy (LHON) Historical Case Record Survey
Start: Aug 31, 2013
Completion: Feb 28, 2014
NCT01805024
Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)
Phase: Phase 1
Start: Dec 31, 2014
Completion: Jan 29, 2018
NCT02774005
Study to Assess the Efficacy and Safety of Raxone in LHON Patients
Phase: Phase 4
Start: May 31, 2016
Completion: Mar 29, 2021
NCT02796274
Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)
Completion: Jul 31, 2018
NCT02771379
Post Authorisation Safety Study With Raxone in LHON Patients
Start: Sep 30, 2016
Completion: Apr 16, 2021
NCT02814019
A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
Completion: Dec 1, 2020
NCT02887443
A Study to Assess the Potential for Pre-systemic Inhibition of CYP3A by Idebenone Using Midazolam as a Substrate
Completion: Nov 30, 2016
NCT03603288
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
Start: Jul 4, 2018
Completion: Nov 25, 2020
NCT03680365
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
Start: Sep 20, 2018
Completion: Mar 15, 2019
NCT03748199
Clinical Study to Investigate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of POL6014 in Patients With CF
Phase: Phase 1/2
Start: Nov 8, 2018
Completion: Dec 30, 2020
NCT05185622
A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
Start: Mar 21, 2022
Completion: Jul 16, 2024
NCT05166109
A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)
Start: Jul 7, 2022
Completion: Aug 18, 2025
NCT06649409
Evaluation of Vamorolone Mineralocorticoid Receptor Antagonism in Healthy Subjects
Start: Jun 5, 2024
Completion: Jul 6, 2024
NCT06689527
Evaluation of Vamorolone CYP3A4 Induction on Midazolam (a Sensitive CYP 3A4 Substrate) Pharmacokinetics
Start: Aug 13, 2024
Completion: Oct 16, 2024
NCT06713135
A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy
Start: Nov 10, 2024
Completion: Sep 30, 2028
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