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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: | NCT00102453 |
In this study, the primary aim will be to estimate the magnitude and variability of strength change over time that may be expected for subjects on the study treatment. This estimate of effect will allow us to develop a rigorous statistical plan in the future randomized study. The specific estimation technique to be applied will use a linear random effects model to estimate average strength change during the 3-month lead-in period and then during the twelve-month treatment period, taking into account the quantitative muscle testing (QMT) measures for each subject. Accounting for the correlation between repeated measures from each subject by using a random effects model will yield an unbiased estimate of variability for the population average change in strength. We will use an analysis of pre- and post-treatment data to inform a best estimate of treatment effect. For example, the difference in QMT trends pre- and post-treatment would provide a straightforward measure of efficacy.
Condition | Intervention | Phase |
---|---|---|
Muscular Dystrophy, Duchenne |
Drug: Pentoxifylline |
Phase I Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | An Open-Label Pilot Study of Pentoxifylline in Steroid-Naive Duchenne Muscular Dystrophy |
Estimated Enrollment: | 15 |
Study Start Date: | March 2002 |
Estimated Study Completion Date: | July 2006 |
Duchenne muscular dystrophy (DMD) is a progressive disease of skeletal muscle caused by the absence of dystrophin due to a genetic mutation in the x-linked dystrophin gene. The absence of dystrophin results in a fragile muscle membrane that permits an abnormal permeability to electrolytes, especially Ca ++. The increase in intracellular calcium triggers a pathological cascade of events that ultimately results in muscle necrosis and fibrosis, which impedes normal muscle regeneration. The increased knowledge of the pathophysiology of DMD opens the opportunity for pharmacological treatment, with the purpose of altering the disease process and or reverting the muscle degeneration.
This research study requires having Duchenne muscular dystrophy (DMD) and the subject to be between 4 and 7 years old. We expect 5 children to take part in this study at Children's Hospital and 10 other children to participate at other hospitals worldwide.
There will be two (2) screening visits to help decide whether you will be able to participate in the study. At the second screening visit, there will be a blood test (about 13 tablespoons of blood), and an EKG. Once the study doctors decide eligibility to be in the study, the subject will then come back once a month for three months to have his strength tested. After three months, the subject will begin to take the pentoxifylline and have an MRI (you will have a test called an MRI to look inside the muscles of your legs). This will continue for 12 months.
Ages Eligible for Study: | 4 Years to 7 Years |
Genders Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Diagnosis of DMD confirmed by at least one of the following:
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 | |
United States, Missouri | |
Washington University at St. Louis | |
St. Louis, Missouri, United States | |
United States, Pennsylvania | |
Children's Hospital of Pittsburgh | |
Pittsburgh, Pennsylvania, United States | |
United States, Texas | |
Texas Scottish Rite Hospital | |
Dallas, Texas, United States |
Study Chair: | Diana Escolar, MD | Childrens Research Institute |
Study ID Numbers: | CNMC0302 |
Study First Received: | January 29, 2005 |
Last Updated: | June 9, 2008 |
ClinicalTrials.gov Identifier: | NCT00102453 |
Health Authority: | United States: Institutional Review Board |
Duchenne Genetics |
Muscular dystrophy, Duchenne and Becker type Pentoxifylline 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 |
Radiation-Protective Agents Vasodilator Agents Antioxidants Molecular Mechanisms of Pharmacological Action Nervous System Diseases Hematologic Agents Physiological Effects of Drugs Enzyme Inhibitors |
Cardiovascular Agents Protective Agents Pharmacologic Actions Phosphodiesterase Inhibitors Therapeutic Uses Free Radical Scavengers Platelet Aggregation Inhibitors |