Status:
COMPLETED
Pentoxifylline in Duchenne Muscular Dystrophy
Lead Sponsor:
Cooperative International Neuromuscular Research Group
Conditions:
Muscular Dystrophy, Duchenne
Eligibility:
MALE
4-7 years
Phase:
PHASE1
PHASE2
Brief Summary
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 all...
Detailed Description
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 r...
Eligibility Criteria
Inclusion
- Male
- Age 4 to 7 years
- Ambulant independently. Subjects may use a wheelchair occasionally, but only for long distances
- Diagnosis of DMD confirmed by at least one of the following:
- Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD OR
- Gene deletion 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.
- Glucocorticosteroid - naïve (i.e. has not been treated with prednisone or Deflazacort within 1 year before onset of the study)
- Has not participated in other therapeutic research protocol within the last 6 months.
- Evidence of muscle weakness by MRC score or clinical functional evaluation
- Ability to provide reproducible repeat QMT bicep score of either the right or left arm within 15% of first assessment score.
Exclusion
- Symptomatic DMD carrier
- Use of any medication, nutritional supplement or herb for treatment of DMD within the last 3 months.
- Symptomatic cardiomyopathy or ventricular arrhythmias
- History of significant concomitant illness, impairment of blood clotting ability (as evidenced by increased PT/PTT or bleeding time over the upper limit of normal (ULN)), recent cerebral or retinal hemorrhage, bleeding diathesis, gastric ulcer, hypotension or significant impairment of renal or hepatic function (defined as serum creatinine and GGT respectively, greater than 1.5 times normal upper limit for age and gender).
Key Trial Info
Start Date :
March 1 2002
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2007
Estimated Enrollment :
17 Patients enrolled
Trial Details
Trial ID
NCT00102453
Start Date
March 1 2002
End Date
May 1 2007
Last Update
October 27 2011
Active Locations (5)
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1
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
2
Mayo Clinic
Rochester, Minnesota, United States, 55905
3
Washington University at St. Louis
St Louis, Missouri, United States, 63110
4
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213