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.
DMD is the most common and devastating type of muscular dystrophy (incidence 1 in 3500 live born males worldwide). DMD is characterized by a complete loss of dystrophin, leading to progressive muscle weakness and wasting. No cure is currently available despite our present understanding of the disorder and the discovery and characterization of the causative gene and its protein product dystrophin in 1987. Corticosteroids (prednisone, deflazacort) may delay disease progression and until now it is the only treatment that proved to be beneficial for patients with DMD. Other alternative supplements like creatine and glutamine also delay diseased progression.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
64
Participants will be randomized to receive either pentoxifylline or placebo in addition to their stable steroid therapy. Active drug and placebo preparations will be supplied as gel capsules of identical size, appearance and taste. Active drug capsules will contain one 400 mg time-release pentoxifylline tablet and inert filler. Placebo capsules will contain inert filler. Based on weight at screening, \<30 mg will receive 1 400 capsule/day; 30-49 kg will receive two 400 capsules/day; 50 kg or greater will receive three 400 mg capsules/day.
Children's National Medical Center
Washington D.C., District of Columbia, United States
Mayo Clinic
Rochester, Minnesota, United States
Washington University, St. Louis
St Louis, Missouri, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Tennessee
Memphis, Tennessee, United States
Hospital Frances
Buenos Aires, Argentina
Children's Hospital
Melbourne, Victoria, Australia
Alberta Children's Hospital
Calgary, Alberta, Canada
University of Alberta
Edmonton, Alberta, Canada
Hadassah Hospital, Mt. Scopus
Jerusalem, Israel
...and 1 more locations
Quantitative muscle strength will be measured using a CINRG Quantitative Muscle System (CQMS). The highest value of two consecutive maximal efforts will be recorded. The primary strength endpoint will be total CQMS score.
Time frame: January 2008
Strength of arm, leg and grip QMT scores Measured Screening and Months 1, 3, 6, 9 & 12
Time frame: January 2008
Manual Muscle Testing (MMT) score measured at screening and months 1, 3, 6, 9 & 12 using the Medical Research Council (MRC) scoring system.
Time frame: January 2008
Functional evaluations measured at screening and months 1, 3, 6, 9 & 12
Time frame: January 2008
Time function assessments, including time rising from the floor, time to climb four standard stairs, and time to walk 10 meters. They will be measured at screening and months 1, 3, 6, 9 & 12.
Time frame: January 2008
pulmonary function test (PFA's) measured at screening and months 1, 3, 6, 9 & 12
Time frame: January 2008
Pediatric Quality of Life (PQOL) measured at screening and months 1, 3, 6, 9 & 12
Time frame: January 2008
Goniometry measured at screening and months 1, 3, 6, 9 & 12
Time frame: January 2008
TNF-alpha and TGF-beta measured at screening and months 1, 3, 6, 9 & 12
Time frame: February 2008
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