Duchenne Muscular Dystrophy Clinical Trial
Official title:
A Randomized Study of Daily vs. High-dose Weekly Prednisone Therapy in Duchenne Muscular Dystrophy
This study will help to determine whether a high-dose weekly course of prednisone therapy is safer than and at least as effective as daily dose therapy for people with Duchenne muscular dystrophy (DMD). Boys who are enrolled in this study should not have taken carnitine, other amino acids, creatine, glutamine, Coenzyme Q10 or any herbal medicines within the last three months. There will be a two-visit screening to take place in one week to ensure a reproducible manual muscle test. The subject will then be randomized and put into either the daily or weekly regimen. The duration of the study is twelve 28-day treatment cycles (approximately 12 months) with follow-up visits at month one, three and then every three months.
Status | Completed |
Enrollment | 64 |
Est. completion date | February 2008 |
Est. primary completion date | December 2007 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 4 Years to 10 Years |
Eligibility |
Inclusion Criteria: - 4 to 10 years of age - Ambulant - Confirmed DMD Diagnosis - Steroid naive - Evidence of muscle weakness by MRC score or clinical functional evaluation - Ability to provide reproducible QMT bicep score Exclusion Criteria: - History of significant concomitant illness or significant impairment of renal or hepatic function, or other contraindication to steroid therapy - Symptomatic DMD carrier - Positive PPD - Lack of prior exposure to chickenpox or immunization - Use of carnitine, glutamine, Coenzyme Q10, other amino acids or any herbal medications within the last 3 months - History of symptomatic cardiomyopathy - Prior attainment of quota for the age group in which the patient belongs |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), 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,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Quantitative muscle strength will be measured using the CINRG Quantitative Measurement System (CQMS) | February 2008 | No | |
Primary | Primary strength endpoints will be quantitative myometry (QMT) scores of the upper and lower extremities, consisting of paired flexor/extensor groups. | February 2008 | No | |
Secondary | Secondary strength endpoints will include individual QMT scores of elbow and knee flexors and extensors and hand grip, manual muscle testing scores, which will be measured using the Medical Research Council's (MRC) muscle strength scoring method. | February 2008 | No | |
Secondary | Side-effect profiles will assessed by monitoring side-effects, including differences in growth (height and weight), calculated weight/height ratio, bone density, cataract formation, blood glucose, blood pressure and behavioral changes. | February 2008 | Yes |
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