Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2008-01-01
• Target enrollment: 0
• Participant gender: Male

Summary

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Cooperative International Neuromuscular Research Group
• Treatments: Pentoxifylline

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.