Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy
Muscular Dystrophy, Duchenne
About this trial
This is an interventional treatment trial for Muscular Dystrophy, Duchenne focused on measuring Duchenne, Genetic, Muscular Dystrophy, DMD
Eligibility Criteria
Inclusion Criteria: Male Age 7 years to 100 years Ability to ambulate for 10 meters. Assistive devices are allowed. Diagnosis of DMD confirmed by at least one the following: On stable dose of prednisone, prednisolone or deflazacort for at least 12 months prior to screening. Participants who are on stable dose of any combination of the following compounds (creatine, glutamine, coenzyme Q10, vitamin E, C or D, JUVEN, arginine, calcium) must have taken these medications for at least 2 months prior to screening. Subjects are not required to take these medications to participate in the study. All other herbs, supplements or green tea (other than those noted above) have been discontinued 3 months prior to screening. Ability to provide reproducible QMT bicep score with no more than 15% variation between scores during screening. Normal blood clotting ability evidenced by a platelet function assessment (PFA). Exclusion Criteria: Currently enrolled in another treatment clinical trial. History of significant concomitant illness or significant impairment of renal or hepatic function. History of impairment of blood clotting ability (as evidenced by increased PT/PTT or PFA over the upper limit of normal (ULN)). Recent cerebral or retinal hemorrhage. History of bleeding diathesis or gastric ulcer.
Sites / Locations
- Children's National Medical Center
- Mayo Clinic
- Washington University, St. Louis
- Children's Hospital of Pittsburgh
- University of Tennessee
- Hospital Frances
- Children's Hospital
- Alberta Children's Hospital
- University of Alberta
- Hadassah Hospital, Mt. Scopus
- IRCCS C Mondino Foundation
Arms of the Study
Arm 1
Arm 2
Active Comparator
No Intervention
1
2
Pentoxifylline
Placebo