Pentoxifylline in Duchenne Muscular Dystrophy
Muscular Dystrophy, Duchenne
About this trial
This is an interventional treatment trial for Muscular Dystrophy, Duchenne focused on measuring Duchenne, Genetics
Eligibility Criteria
Inclusion Criteria: 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 Criteria: 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).
Sites / Locations
- Children's National Medical Center
- Mayo Clinic
- Washington University at St. Louis
- Children's Hospital of Pittsburgh
- Texas Scottish Rite Hospital
Arms of the Study
Arm 1
Experimental
Solution
All enrolled participants were give pentoxifylline in this pilot protocol.