Muscular Dystrophy, Duchenne Clinical Trial
NCT number | NCT00033813 |
Other study ID # | KUL0401 |
Secondary ID | |
Status | Completed |
Phase | Phase 2 |
First received | April 10, 2002 |
Last updated | March 7, 2014 |
Start date | January 2002 |
This study will help to determine the safety and efficacy of the mast cell stabilizer Oxatomide as a treatment for Duchenne muscular dystrophy (DMD). Boys with DMD who are enrolled in this study will should not have taken steroids to treat DMD for at least twelve months, and should not have taken any nutritional supplements for at least three months. Subjects will complete a two screening visits within a one-week period, and if enrolled will then have their strength tested monthly for three months before beginning therapy with Oxatomide. Once Oxatomide therapy is started, participants will have their strength tested monthly for six months. Following the six month treatment period, participants will be given the option to remain on Oxatomide until the study is completed.
Status | Completed |
Enrollment | 15 |
Est. completion date | |
Est. primary completion date | December 2006 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 5 Years to 10 Years |
Eligibility |
Subject Inclusion Criteria 1. 5 to 10 years of age 2. ambulatory 3. diagnosis of DMD confirmed by at least one of the following: - Positive x-linked family history of DMD in older male relatives (onset by 5 years, wheelchair bound by 12 years), or; - Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, or; - Gene deletion test positive (missing one or more exons) in the central rod domain (exons 25-60) of dystrophin, where reading frame can be predicted as 'out of frame', and clinical picture consistent with typical DMD. 4. glucocorticosteroid-naive (i.e. has not been treated with prednisone or deflazacort within the past year) 5. Evidence of muscle weakness by MRC score or clinical functional evaluation 6. QMT biceps score variability no greater than 10% between screening visits Subject Exclusion Criteria 1. Failure to achieve one or more of the inclusion criteria listed above 2. Inability to suitably cooperate with strength assessments 3. Symptomatic DMD carrier 4. Use of oxatomide (or other anti-histamine drugs) within the last 6 months for DMD or any other disease 5. Use of creatine monohydrate or glutamine within the last 6 months 6. Use of carnitine, Coenzyme Q10, other amino acids or any herbal medications within the last 3 months 7. History of symptomatic cardiomyopathy 8. History of impairment of hepatic function 9. History of significant concomitant illness or significant impairment of renal function. |
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | Children's National Medical Center | Washington | District of Columbia |
Lead Sponsor | Collaborator |
---|---|
Cooperative International Neuromuscular Research Group |
United States,
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 |