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Sponsored by: |
Cooperative International Neuromuscular Research Group |
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Information provided by: | Cooperative International Neuromuscular Research Group |
ClinicalTrials.gov Identifier: | NCT00110669 |
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.
Condition | Intervention | Phase |
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Duchenne Muscular Dystrophy |
Drug: Prednisone |
Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator), Placebo Control, Parallel Assignment, Safety/Efficacy Study |
Official Title: | A Randomized Study of Daily vs. High-Dose Weekly Prednisone Therapy in Duchenne Muscular Dystrophy |
Enrollment: | 64 |
Study Start Date: | January 2004 |
Study Completion Date: | February 2008 |
Primary Completion Date: | December 2007 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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High Dose Prednisone: Active Comparator
Subjects who are randomized to the high-dose prednisone arm of the study will receive the following starting dose: •Prednisone at 10.0 mg/kg/wk (divided into two doses given on Saturday and Sunday) |
Drug: Prednisone
Prednisone and dummy preparations for this study will be obtained from Frank's Pharmacy in Ocala, FL and will be supplied as a tablet containing 2.5mg, 5mg, 10mg, 20mg or 50mg Prednisone. Inactive "dummy" pills of similar look/taste will be supplied to maintain blinding.
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Daily Prednisone: Active Comparator
Subjects who are randomized to the daily prednisone arm of the study will receive the following starting dose: •Prednisone at 0.75 mg/kg/d |
Drug: Prednisone
Prednisone and dummy preparations for this study will be obtained from Frank's Pharmacy in Ocala, FL and will be supplied as a tablet containing 2.5mg, 5mg, 10mg, 20mg or 50mg Prednisone. Inactive "dummy" pills of similar look/taste will be supplied to maintain blinding.
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Duchenne muscular dystrophy (DMD) is the most common lethal inherited disorder worldwide. Despite the exponential increase in our understanding of the disorder since the discovery and characterization of the causative gene and its product dystrophin in 1987, current therapeutic management remains largely supportive. Awaiting a final genetic cure to be available in the future, further investments in developing better drug therapies for DMD remain important. The effect of a high dose prednisone regimen will be evaluated in comparison to a daily dose regimen in a multi-center, randomized, double-blind placebo-controlled 4-arm study. Ambulant children aged 4-10 years with an established DMD diagnosis will be studied. Patients will undergo 2 screening evaluations within 1 week. Patients will be randomized into treatment groups on the second screening visit, followed by a 12-month treatment period. During the treatment period, patients will be evaluated at monthly intervals. The primary endpoints are percentage change in average muscle strength score and QMT performance for specific muscle groups. Secondary endpoints include timed function tests, functional grades for arms and legs, and pulmonary function tests.
Ages Eligible for Study: | 4 Years to 10 Years |
Genders Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, California | |
UC Davis | |
Sacramento, California, United States | |
United States, District of Columbia | |
Children's National Medical Center | |
Washington, District of Columbia, United States, 20010 | |
United States, Pennsylvania | |
Children's Hospital of Pittsburgh | |
Pittsburgh, Pennsylvania, United States | |
United States, Tennessee | |
University of Tennessee | |
Memphis, Tennessee, United States | |
India | |
Sundaram Medical Foundation | |
Chennai, India |
Study Chair: | Diana Escolar, MD | Childrens Research Institute |
Responsible Party: | CINRG ( Study Chair ) |
Study ID Numbers: | CNMC0601 |
Study First Received: | May 12, 2005 |
Last Updated: | June 9, 2008 |
ClinicalTrials.gov Identifier: | NCT00110669 |
Health Authority: | United States: Institutional Review Board |
Muscular dystrophy, Duchenne and Beckers Beckers Muscular dystrophy |
Prednisone Benzocaine Muscular dystrophy, Duchenne and Becker type Muscular Dystrophies Muscular Diseases Becker's muscular dystrophy Muscular Disorders, Atrophic Musculoskeletal Diseases |
Neuromuscular Diseases Genetic Diseases, Inborn Muscular Dystrophy, Duchenne Genetic Diseases, X-Linked Duchenne muscular dystrophy Atrophy Muscular dystrophy |
Anti-Inflammatory Agents Antineoplastic Agents, Hormonal Antineoplastic Agents Therapeutic Uses Physiological Effects of Drugs |
Nervous System Diseases Hormones, Hormone Substitutes, and Hormone Antagonists Hormones Glucocorticoids Pharmacologic Actions |