A Phase 1/2 interventional study of Pentoxifylline in Muscular Dystrophy, Duchenne, sponsored by Cooperative International Neuromuscular Research Group. Completed at 5 sites in United States. Open to male participants aged 4 Years to 7 Years. Per ClinicalTrials.gov, last updated 2011-10-27.
Sponsored by Cooperative International Neuromuscular Research Group · Phase 1/2, Interventional, and Treatment
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.
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.
548 studies on the registry are indexed under Muscular Dystrophies; 89 are open to participants now.
This study's enrollment of 17 is below the median of 24 across 344 interventional studies indexed under Muscular Dystrophies.
Browse Muscular Dystrophies studies →Cooperative International Neuromuscular Research Group is the lead sponsor of 17 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Diagnosis of DMD confirmed by at least one of the following:
Exclusion Criteria:
All enrolled participants were give pentoxifylline in this pilot protocol.
Drug: Pentoxifylline
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.
Also known as: Supplier: Frank's Pharmacy, Ocala, Fl. 34474., Product: Pentoxifylline BP, CAS number: 5/6/6493, Formula weight: 278.35, Chemical formula: C13H18N4O3
QMT measurements
Quantitative muscle testing (QMT) is a technique utilized to assess muscle strength. Measurements of force are collected using a load cell while performing a maximum voluntary isometric contraction. This set-up is able to measure changes in strength of 0.25 lb which provides accurate and sensitive measurement of muscular strength. QMT is performed by a CINRG physical therapist.
Time frame: Each study visit
Change in manual muscle test (MMT) at 12 months
Manual muscle testing (MMT), which is graded according to the modified Medical Research Council (MRC) scale, is a test of a participant's muscle strength, or ability of the muscle to move a part of the body against resistance. A CINRG physical therapist will perform MMT testing with each participant.
Time frame: Each study visit
This study is completed, as verified in Oct 2011. You cannot join it, but the record below documents what was studied.
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Cooperative International Neuromuscular Research Group