Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy
NCT ID: NCT00243789
Last Updated: 2011-10-27
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1/PHASE2
64 participants
INTERVENTIONAL
2005-09-30
2008-01-31
Brief Summary
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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.
Detailed Description
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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.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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1
Pentoxifylline
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
Placebo
No interventions assigned to this group
Interventions
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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.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
7 Years
MALE
No
Sponsors
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Cooperative International Neuromuscular Research Group
NETWORK
Responsible Party
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CINRG
Principal Investigators
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Diana Escolar, MD
Role: STUDY_CHAIR
Children's National Medical Center, Center for Genetic Medicine
Locations
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Children's National Medical Center
Washington D.C., District of Columbia, United States
Mayo Clinic
Rochester, Minnesota, United States
Washington University, St. Louis
St Louis, Missouri, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Tennessee
Memphis, Tennessee, United States
Hospital Frances
Buenos Aires, , Argentina
Children's Hospital
Melbourne, Victoria, Australia
Alberta Children's Hospital
Calgary, Alberta, Canada
University of Alberta
Edmonton, Alberta, Canada
Hadassah Hospital, Mt. Scopus
Jerusalem, , Israel
IRCCS C Mondino Foundation
Pavia, Italy, Italy
Countries
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Related Links
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Related Info
Other Identifiers
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CNMC0705
Identifier Type: -
Identifier Source: org_study_id