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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: | NCT00243789 |
The purpose of this study is to see if male children with Duchenne muscular dystrophy (DMD) have changes in strength when given the drug Pentoxifylline as a rescue treatment. A total of 64 subjects are expected to participate through all other centers of the Cooperative International Neuromuscular Research Group (CINRG) worldwide.
The primary purpose of this study is to see whether the addition of pentoxifylline to a steroid regimen is effective in treating deteriorating muscle strength by comparing the muscle strength of PTX treated subjects and placebo treated subjects.
Condition | Intervention | Phase |
---|---|---|
Muscular Dystrophy, Duchenne |
Drug: Pentoxifylline |
Phase I Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator), Placebo Control, Parallel Assignment, Safety/Efficacy Study |
Official Title: | A Double-Blinded Randomized Placebo Controlled Study of Daily Pentoxifylline as a Rescue Treatment in DMD |
Enrollment: | 64 |
Study Start Date: | September 2005 |
Study Completion Date: | January 2008 |
Primary Completion Date: | December 2007 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Active Comparator
Pentoxifylline
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Drug: Pentoxifylline
Participants will be randomized to receive either pentoxifylline or placebo in addition to their stable steroid therapy. Active drug and placebo preparations will be supplied as gel capsules of identical size, appearance and taste. Active drug capsules will contain one 400 mg time-release pentoxifylline tablet and inert filler. Placebo capsules will contain inert filler. Based on weight at screening, <30 mg will receive 1 400 capsule/day; 30-49 kg will receive two 400 capsules/day; 50 kg or greater will receive three 400 mg capsules/day. |
2: No Intervention
Placebo
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DMD is the most common and devastating type of muscular dystrophy (incidence 1 in 3500 live born males worldwide). DMD is characterized by a complete loss of dystrophin, leading to progressive muscle weakness and wasting.
No cure is currently available despite our present understanding of the disorder and the discovery and characterization of the causative gene and its protein product dystrophin in 1987. Corticosteroids (prednisone, deflazacort) may delay disease progression and until now it is the only treatment that proved to be beneficial for patients with DMD. Other alternative supplements like creatine and glutamine also delay diseased progression.
Ages Eligible for Study: | 7 Years and older |
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, Minnesota | |
Mayo Clinic | |
Rochester, Minnesota, United States, 55905 | |
United States, Missouri | |
Washington University, St. Louis | |
St. Louis, Missouri, United States, 63110 | |
United States, Pennsylvania | |
Children's Hospital of Pittsburgh | |
Pittsburgh, Pennsylvania, United States, 15213 | |
United States, Tennessee | |
University of Tennessee | |
Memphis, Tennessee, United States, 38104 | |
Argentina | |
Hospital Frances | |
Buenos Aires, Argentina, 1434 | |
Australia, Victoria | |
Children's Hospital | |
Melbourne, Victoria, Australia, 3052 | |
Canada, Alberta | |
Alberta Children's Hospital | |
Calgary, Alberta, Canada, T2T 5C7 | |
University of Alberta | |
Edmonton, Alberta, Canada, T6G 2J3 | |
Israel | |
Hadassah Hospital, Mt. Scopus | |
Jerusalem, Israel, 91240 | |
Italy | |
IRCCS C Mondino Foundation | |
Pavia, Italy, 27100 |
Study Chair: | Diana Escolar, MD | Children's National Medical Center, Center for Genetic Medicine |
Responsible Party: | CINRG ( Study Chair ) |
Study ID Numbers: | CNMC0705 |
Study First Received: | October 21, 2005 |
Last Updated: | January 4, 2008 |
ClinicalTrials.gov Identifier: | NCT00243789 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Duchenne Genetic Muscular Dystrophy DMD |
Becker's Muscular Dystrophy Vasodilator Agents Radiation-Protective Agents Antioxidants Cardiovascular Agents Pentoxifylline Muscular Dystrophy, Duchenne and Becker Type Muscular Dystrophies Phosphodiesterase Inhibitors Muscular Diseases |
Muscular Disorders, Atrophic Musculoskeletal Diseases Neuromuscular Diseases Genetic Diseases, Inborn Muscular Dystrophy, Duchenne Duchenne Muscular Dystrophy Genetic Diseases, X-Linked Platelet Aggregation Inhibitors Atrophy Muscular Dystrophy |
Vasodilator Agents Radiation-Protective Agents Antioxidants Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs Hematologic Agents Nervous System Diseases Enzyme Inhibitors Cardiovascular Agents Protective Agents Pharmacologic Actions Pentoxifylline |
Muscular Dystrophies Muscular Diseases Phosphodiesterase Inhibitors Muscular Disorders, Atrophic Musculoskeletal Diseases Neuromuscular Diseases Genetic Diseases, Inborn Therapeutic Uses Muscular Dystrophy, Duchenne Free Radical Scavengers Genetic Diseases, X-Linked Platelet Aggregation Inhibitors |