Pentoxifylline in Duchenne Muscular Dystrophy

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Brief Title

Pentoxifylline in Duchenne Muscular Dystrophy

Official Title

An Open-Label Pilot Study of Pentoxifylline in Steroid-naive Duchenne Muscular Dystrophy

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.
    

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 Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

QMT measurements

Secondary Outcome

 Change in manual muscle test (MMT) at 12 months

Condition

Muscular Dystrophy, Duchenne

Intervention

Pentoxifylline

Study Arms / Comparison Groups

 Solution
Description:  All enrolled participants were give pentoxifylline in this pilot protocol.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

17

Start Date

March 2002

Completion Date

May 2007

Primary Completion Date

July 2006

Eligibility Criteria

        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).
      

Gender

Male

Ages

4 Years - 7 Years

Accepts Healthy Volunteers

No

Contacts

Diana Escolar, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00102453

Organization ID

CNMC0302



Study Sponsor

Cooperative International Neuromuscular Research Group


Study Sponsor

Diana Escolar, MD, Study Chair, Children's National Research Institute


Verification Date

October 2011