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Sponsors and Collaborators: |
Cooperative International Neuromuscular Research Group Department of Defense |
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Information provided by: | Cooperative International Neuromuscular Research Group |
ClinicalTrials.gov Identifier: | NCT00308113 |
This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD. Participants who are enrolled in this study should not have taken any corticosteroids within the last six months. This is a 13-month, prospective, randomized study comparing a daily prednisone arm (0.75mg/kg/day), a CoQ10 arm (serum of greater than 2.5 ug/mL) and a combination arm (prednisone and CoQ10) with an enhanced standard of care arm in wheelchair confined males age 10 to 18 years with an established DMD diagnosis.
Condition | Intervention | Phase |
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Duchenne Muscular Dystrophy |
Drug: Prednisone Dietary Supplement: Coenzyme Q10 |
Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Open Label, Active Control, Parallel Assignment, Safety/Efficacy Study |
Official Title: | PITT0503: Clinical Trial of Coenzyme Q10 and Prednisone in Duchenne Muscular Dystrophy |
Estimated Enrollment: | 120 |
Study Start Date: | March 2006 |
Estimated Study Completion Date: | December 2010 |
Estimated Primary Completion Date: | December 2010 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Active Comparator
CoenzymeQ10 taken once a day each morning by mouth.
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Dietary Supplement: Coenzyme Q10
serum levels of greater or equal to 2.5 micrograms/mL.
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2: Active Comparator
Prednisone taken once a day each morning by mouth
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Drug: Prednisone
Prednisone 0/75 mg/kg/day.
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3: Active Comparator
CoenzymeQ10 and prednisone each taken once a day in the morning by mouth.
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Drug: Prednisone
Prednisone 0/75 mg/kg/day.
Dietary Supplement: Coenzyme Q10
serum levels of greater or equal to 2.5 micrograms/mL.
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4: No Intervention
Enhanced standard of care.
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Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy affecting 1:3500 male births worldwide. Despite an 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. Improvement in the treatment of DMD will depend upon the development of better therapies. Affected boys become symptomatic at 3 to 5 years of age with proximal leg weakness that impairs mobility, ability to get up from a squat, and precludes a normal ability to run. By 8 years of age, some affected boys begin to lose the ability to walk and resort to a wheelchair for mobility. This shift from the ambulant to non-ambulant phase occurs in all boys with a diagnosis of DMD by age 12 years. In this study, participants will be randomized into groups after being screened to determine eligibility. Participants will then be followed for a 12-month investigation period.
Ages Eligible for Study: | 10 Years to 18 Years |
Genders Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, District of Columbia | |
Children's National Medical Center | |
Washington, District of Columbia, United States, 20010 | |
United States, Pennsylvania | |
University of Pittsburgh | |
Pittsburgh, Pennsylvania, United States, 15213 | |
Australia, Victoria | |
Royal Children's Hospital | |
Melbourne, Victoria, Australia, 3052 | |
Puerto Rico | |
University of Puerto Rico, Medical Sciences Campus | |
San Juan, Puerto Rico, 00936 |
Study Chair: | Paula R Clemens, M.D. | University of Pittsburgh |
Responsible Party: | Cooperative International Neuromuscular Research Group ( Study Chair, Dr. Paula Clemens ) |
Study ID Numbers: | PITT0503 |
Study First Received: | March 27, 2006 |
Last Updated: | June 9, 2008 |
ClinicalTrials.gov Identifier: | NCT00308113 |
Health Authority: | United States: Food and Drug Administration |
Muscular dystrophy Duchenne CoQ10 prednisone |
Prednisone Muscular dystrophy, Duchenne and Becker type Coenzyme Q10 Muscular Dystrophies Muscular Diseases Becker's muscular dystrophy Muscular Disorders, Atrophic Musculoskeletal Diseases |
Neuromuscular Diseases Genetic Diseases, Inborn Muscular Dystrophy, Duchenne Ubiquinone Genetic Diseases, X-Linked Duchenne muscular dystrophy Atrophy Muscular dystrophy |
Anti-Inflammatory Agents Antineoplastic Agents, Hormonal Antineoplastic Agents Growth Substances Nervous System Diseases Physiological Effects of Drugs Hormones, Hormone Substitutes, and Hormone Antagonists |
Glucocorticoids Hormones Pharmacologic Actions Therapeutic Uses Vitamins Micronutrients |