Muscular Dystrophy, Duchenne Clinical Trial
Official title:
A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10-18 Year Old Patients With Duchenne Muscular Dystrophy
The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD. Furthermore, the safety and tolerability of idebenone was assessed.
This study was a Phase III, multicenter, randomized, double-blind, placebo-controlled efficacy and safety study. DMD patients (ambulatory and non-ambulatory) at age 10-18 years were enrolled at sites in Europe and North America. Study subjects were randomized in a 1:1 ratio to receive either idebenone (900 mg/day) or placebo 3 times a day with meals for 52 weeks. The primary endpoint was the difference between Catena®/Raxone® and placebo in the change from Baseline to week 52 in Peak Expiratory Flow (PEF as percent predicted, PEF%p, a measure of respiratory muscle strength) as measured by hospital-based spirometry. PEF was also measured by the patient at home using the hand-held ASMA-1 device (secondary endpoint). Other respiratory endpoints included Forced Expiratory Volume in 1 second (as percent predicted, FEV1%p, an additional measure of respiratory muscle strength) and Forced Vital Capacity (as percent predicted, FVC%p, a measure of restrictive lung disease predictive of morbidity and mortality in DMD). ;
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Status | Clinical Trial | Phase | |
---|---|---|---|
Terminated |
NCT01865084 -
A Study of Tadalafil for Duchenne Muscular Dystrophy
|
Phase 3 | |
Completed |
NCT00243789 -
Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy
|
Phase 1/Phase 2 | |
Completed |
NCT00033189 -
An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy
|
Phase 2 | |
Completed |
NCT03703882 -
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
|
Phase 3 | |
Enrolling by invitation |
NCT04626674 -
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
|
Phase 1 | |
Completed |
NCT02286947 -
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
|
Phase 2 | |
Completed |
NCT03406780 -
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
|
Phase 2 | |
Completed |
NCT01826487 -
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
|
Phase 3 | |
Completed |
NCT02710591 -
Rimeporide in Patients With Duchenne Muscular Dystrophy
|
Phase 1 | |
Completed |
NCT01826422 -
Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients
|
N/A | |
Completed |
NCT00102453 -
Pentoxifylline in Duchenne Muscular Dystrophy
|
Phase 1/Phase 2 | |
Terminated |
NCT02090959 -
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
|
Phase 3 | |
Recruiting |
NCT05833633 -
Study of Genotype and Phenotype Characterization in Duchenne Muscular Dystrophy With Small Mutations
|
||
Completed |
NCT05209087 -
Effects of Parental Influence on Physical Activity Level and Participation in Children With Duchenne Muscular Dystrophy
|
||
Completed |
NCT03789734 -
Safety Study of BLS-M22 in Healthy Volunteers
|
Phase 1 | |
Recruiting |
NCT05126758 -
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
|
Phase 3 | |
Completed |
NCT00016653 -
Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy
|
Phase 2/Phase 3 | |
Completed |
NCT03127241 -
User-centred Assistive System for Arm Functions in Neuromuscular Subjects
|
N/A | |
Completed |
NCT03490214 -
Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography
|
N/A | |
Completed |
NCT03179631 -
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
|
Phase 3 |