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Pentoxifylline in Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00102453
Recruitment Status : Completed
First Posted : January 31, 2005
Last Update Posted : October 27, 2011
Information provided by:
Cooperative International Neuromuscular Research Group

Brief 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.

Condition or disease Intervention/treatment Phase
Muscular Dystrophy, Duchenne Drug: Pentoxifylline Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 17 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Pilot Study of Pentoxifylline in Steroid-naive Duchenne Muscular Dystrophy
Study Start Date : March 2002
Actual Primary Completion Date : July 2006
Actual Study Completion Date : May 2007

Arm Intervention/treatment
Experimental: Solution
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.
Other Names:
  • Supplier: Frank's Pharmacy, Ocala, Fl. 34474.
  • Product: Pentoxifylline BP
  • CAS number: 5/6/6493
  • Formula weight: 278.35
  • Chemical formula: C13H18N4O3

Primary Outcome Measures :
  1. QMT measurements [ Time Frame: Each study visit ]
    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.

Secondary Outcome Measures :
  1. Change in manual muscle test (MMT) at 12 months [ Time Frame: Each study visit ]
    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.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   4 Years to 7 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Male
  2. Age 4 to 7 years
  3. Ambulant independently. Subjects may use a wheelchair occasionally, but only for long distances
  4. 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.
  5. 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.
  6. Glucocorticosteroid - naïve (i.e. has not been treated with prednisone or Deflazacort within 1 year before onset of the study)
  7. Has not participated in other therapeutic research protocol within the last 6 months.
  8. Evidence of muscle weakness by MRC score or clinical functional evaluation
  9. Ability to provide reproducible repeat QMT bicep score of either the right or left arm within 15% of first assessment score.

Exclusion Criteria:

  1. Symptomatic DMD carrier
  2. Use of any medication, nutritional supplement or herb for treatment of DMD within the last 3 months.
  3. Symptomatic cardiomyopathy or ventricular arrhythmias
  4. 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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00102453

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United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, Missouri
Washington University at St. Louis
St. Louis, Missouri, United States, 63110
United States, Pennsylvania
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
United States, Texas
Texas Scottish Rite Hospital
Dallas, Texas, United States
Sponsors and Collaborators
Cooperative International Neuromuscular Research Group
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Study Chair: Diana Escolar, MD Children's National Research Institute

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Responsible Party: CINRG Medical Director, CINRG Identifier: NCT00102453    
Other Study ID Numbers: CNMC0302
First Posted: January 31, 2005    Key Record Dates
Last Update Posted: October 27, 2011
Last Verified: October 2011
Keywords provided by Cooperative International Neuromuscular Research Group:
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Phosphodiesterase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Platelet Aggregation Inhibitors
Radiation-Protective Agents
Protective Agents
Physiological Effects of Drugs
Vasodilator Agents
Free Radical Scavengers