CLINICAL TRIAL/STUDY

DISEASE CLASSIFICATION(S): Duchenne muscular dystrophy (DMD)

NAME OF CLINICAL TRIAL/STUDY:

Pentoxifylline -- An open-label study of pentoxifylline (CNMC0302) in DMD in Children Who Have Not Taken Steroids

PURPOSE AND RATIONALE:

Pentoxifylline is a drug that has Food and Drug Administration approval for the treatment of obstruction of the arteries. It decreases the viscosity (“stickiness”) of the blood and improves blood flow. This drug is also an anti-inflammatory drug different from steroids, and has an anti-fibrotic effect (decreases the formation of scar tissue)

In DMD this drug has the potential of decreasing muscle breakdown due to its anti-inflammatory actions and to decrease the abnormal scar tissue that forms in muscle and prevents muscle regeneration. These effects have been seen in the mouse model of DMD (MDX mouse) and in experiments on DMD muscle tissue in the laboratory. The decrease on muscle breakdown and decrease on fibrosis might result in improvement of muscle strength. <please fill in rationale for use in DMD; does it have to do with calcium, or ?>

STUDY DETAILS:

This study is for 4 to 7 years old boys with DMD that have not taken steroids before. The total duration of the trial is 15 months. Patient eligibility will be assessed during 2 screening visits, where clinical history, muscle strength testing, EKG and blood work will be done. 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 (lead –in period). After three months, participants will begin to take pentoxifylline in a liquid form. This form has NOT been approved by the FDA and is only investigational. The liquid form is being used because it allows the investigators to administer the exact dose per kg of weight for each boy, to prevent under dosing and overdosing (with its associated toxicity). This study will also require a magnetic resonance imaging scan (MRI) of the leg muscles at baseline and at the end of the study.

In this study, the primary aim will be to assess the safety of administration of PTX to children with DMD. A secondary aim is to estimate strength change over time. This estimate will allow the investigators to develop a rigorous statistical plan for future studies.

OPENING/CLOSING DATES: Currently enrolling, expecting to finish enrollment end 2/2005; closing 2/2005

TARGET NUMBER OF PARTICIPANTS:

15

RECRUITMENT STATUS: open

ELIGIBILITY REQUIREMENTS:

Participants must

• have Duchenne muscular dystrophy (DMD)
• be between 4 and 7 years old
• never have taken steroids, such as prednisone or deflazacort

MAIN SPONSORING LOCATION/CONTACT:

Angela Zimmerman
Research Coordinator, Cooperative International
     Neuromuscular Research Group
Center for Genetic Medicine
Children's Research Institute
111 Michigan Avenue, NW
Washington, DC  20010

Phone: 202-884-4110
E-mail: AZimmerman@cnmcresearch.org

ADDITIONAL LOCATION(S)/CONTACT(S):

Minnesota

Rochester

Mayo Clinic
Wendy Korn-Peterson
507-284-3351
kornpeterson.wendy@mayo.edu
Nancy Kuntz, MD,  Sub-Investigator

Recruiting

Missouri

St. Louis

Washington University
Charlie Wulf
314-362-6981
harperc@neuro.wustl.edu
Ann Connolly, MD,  Sub-Investigator

Recruiting

Pennsylvania

Pittsburgh

Children's Hospital
Lauren Morgenroth
412-383-7207
morgenrothl@upmc.edu
Paula Clemens, MD,  Sub-Investigator

Recruiting

Texas

Dallas

Texas Scottish Rite Hospital
Betsy Teitell
214-559-7694
Betsy.Teitell@tsrh.org
Susan Iannaccone, MD,  Sub-Investigator

Recruiting

FUNDING (Agencies, Pharmaceutical Companies, etc.):

Foundation to Eradicate Duchenne (FED)

IRB and FDA APPROVAL:

yes

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