- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00102453
Pentoxifylline in Duchenne Muscular Dystrophy
An Open-Label Pilot Study of Pentoxifylline in Steroid-naive Duchenne Muscular Dystrophy
Study Overview
Status
Conditions
Intervention / Treatment
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.
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
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District of Columbia
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Washington, District of Columbia, United States, 20010
- Children's National Medical Center
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Minnesota
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Rochester, Minnesota, United States, 55905
- Mayo Clinic
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Missouri
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St. Louis, Missouri, United States, 63110
- Washington University at St. Louis
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Pennsylvania
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Pittsburgh, Pennsylvania, United States, 15213
- Children's Hospital of Pittsburgh
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Texas
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Dallas, Texas, United States
- Texas Scottish Rite Hospital
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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).
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Solution
All enrolled participants were give pentoxifylline in this pilot protocol.
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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 Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
QMT measurements
Time Frame: Each study visit
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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.
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Each study visit
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in manual muscle test (MMT) at 12 months
Time Frame: Each study visit
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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.
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Each study visit
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Collaborators and Investigators
Investigators
- Study Chair: Diana Escolar, MD, Children's National Research Institute
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Nervous System Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Musculoskeletal Diseases
- Muscular Diseases
- Neuromuscular Diseases
- Muscular Disorders, Atrophic
- Muscular Dystrophies
- Muscular Dystrophy, Duchenne
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Vasodilator Agents
- Enzyme Inhibitors
- Platelet Aggregation Inhibitors
- Protective Agents
- Antioxidants
- Phosphodiesterase Inhibitors
- Free Radical Scavengers
- Radiation-Protective Agents
- Pentoxifylline
Other Study ID Numbers
- CNMC0302
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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