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

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.

Official Title

A Double-Blinded Randomized Placebo Controlled Study of Daily Pentoxifylline as a Rescue Treatment in DMD

Conditions

Duchenne Muscular Dystrophy

Study Type

Interventional

Study Design

Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study

Further Details

Primary Outcome Measures:

• The comparison of muscle strength at month 1, 3, 6, 9 & 12.

Secondary Outcome Measures:

• Strength of arm, leg and grip QMT scores
• MMT score
• functional evaluations
• time function tests
• pulmonary function test(PFA’s)
• PQOL
• Goniometry
• TNF-alpha and TGF-beta

(all measured at Months 1, 3, 6, 9 & 12)

Study Start

September 2005; Study completion: December 2007

Eligibility & Criteria

• Ages Eligible for Study: 7 Years and above
• Genders Eligible for Study: Male

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:
  • Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD
  • Gene deletions test positive (missing one or more exons) in the central rod domain (exons 25-60) of dystrophin, where reading frame can be predicted as ‘out-of-frame’, and clinical picture consistent with typical DMD
  • Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, or other mutation resulting in a stop codon mutation) that can be definitely associated with DMD, with a typical clinical picture of DMD
  • Positive family history of DMD, confirmed by one of the criteria listed above in a sibling or maternal uncle, and clinical picture typical of DMD.
• 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.

Total Enrolment

64

Contact Details

• Children’s Hospital, Melbourne, Victoria, 3052, Australia

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