Pentoxifylline in Duchenne Muscular Dystrophy
Condition: Muscular Dystrophy, Duchenne
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Study type: Interventional
Phase: Phase 1/Phase 2

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 allow us to develop a rigorous statistical plan in the future randomized study.
      The specific estimation technique to be applied will use a linear random effects model to
      estimate average strength change during the 3-month lead-in period and then during the
      twelve-month treatment period, taking into account the quantitative muscle testing (QMT)
      measures for each subject. Accounting for the correlation between repeated measures from each
      subject by using a random effects model will yield an unbiased estimate of variability for
      the population average change in strength. We will use an analysis of pre- and post-treatment
      data to inform a best estimate of treatment effect. For example, the difference in QMT trends
      pre- and post-treatment would provide a straightforward measure of efficacy.
    

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 results in a fragile muscle membrane that permits an abnormal permeability to
      electrolytes, especially Ca ++. The increase in intracellular calcium triggers a pathological
      cascade of events that ultimately results in muscle necrosis and fibrosis, which impedes
      normal muscle regeneration. The increased knowledge of the pathophysiology of DMD opens the
      opportunity for pharmacological treatment, with the purpose of altering the disease process
      and or reverting the muscle degeneration.

      This research study requires having Duchenne muscular dystrophy (DMD) and the subject to be
      between 4 and 7 years old. We expect 5 children to take part in this study at Children's
      Hospital and 10 other children to participate at other hospitals worldwide.

      There will be two (2) screening visits to help decide whether you will be able to participate
      in the study. At the second screening visit, there will be a blood test (about 13 tablespoons
      of blood), and an EKG. Once the study doctors decide eligibility to be in the study, the
      subject will then come back once a month for three months to have his strength tested. After
      three months, the subject will begin to take the pentoxifylline and have an MRI (you will
      have a test called an MRI to look inside the muscles of your legs). This will continue for 12
      months.
    

Intervention name(s): Pentoxifylline
Intervention type(s): Drug
Intervention description(s): Pentoxifylline dosing: 20mg/Kg/day in a 20 mg/mL solution. Maximum dose of 1200mg/day. Dosing split into two equal parts taken morning and night with food.
Other intervention name(s): Supplier: Frank's Pharmacy, Ocala, Fl. 34474.; Product: Pentoxifylline BP; CAS number: 5/6/6493; Formula weight: 278.35; Chemical formula: C13H18N4O3
Intervention MeSH terms: Pentoxifylline
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Duchenne; Genetics