Study of Daily Pentoxifylline as a Rescue Treatment 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: NCT00243789
Recruitment Status : Completed
First Posted : October 25, 2005
Last Update Posted : October 27, 2011
Information provided by:
Cooperative International Neuromuscular Research Group

Brief Summary:

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.

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

Detailed Description:

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  (Clinical Trial)
Actual Enrollment : 64 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Double-Blinded Randomized Placebo Controlled Study of Daily Pentoxifylline as a Rescue Treatment in DMD
Study Start Date : September 2005
Actual Primary Completion Date : December 2007
Actual Study Completion Date : January 2008

Arm Intervention/treatment
Active Comparator: 1
Drug: Pentoxifylline

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.

Other Name: Trental

No Intervention: 2

Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. Strength of arm, leg and grip QMT scores Measured Screening and Months 1, 3, 6, 9 & 12 [ Time Frame: January 2008 ]
  2. 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 ]
  3. Functional evaluations measured at screening and months 1, 3, 6, 9 & 12 [ Time Frame: January 2008 ]
  4. 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 ]
  5. pulmonary function test (PFA's) measured at screening and months 1, 3, 6, 9 & 12 [ Time Frame: January 2008 ]
  6. Pediatric Quality of Life (PQOL) measured at screening and months 1, 3, 6, 9 & 12 [ Time Frame: January 2008 ]
  7. Goniometry measured at screening and months 1, 3, 6, 9 & 12 [ Time Frame: January 2008 ]
  8. TNF-alpha and TGF-beta measured at screening and months 1, 3, 6, 9 & 12 [ Time Frame: February 2008 ]

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Ages Eligible for Study:   7 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male
  • Age 7 years to 100 years
  • Ability to ambulate for 10 meters. Assistive devices are allowed.
  • Diagnosis of DMD confirmed by at least one the following:
  • On stable dose of prednisone, prednisolone or deflazacort for at least 12 months prior to screening.
  • Participants who are on stable dose of any combination of the following compounds (creatine, glutamine, coenzyme Q10, vitamin E, C or D, JUVEN, arginine, calcium) must have taken these medications for at least 2 months prior to screening. Subjects are not required to take these medications to participate in the study.
  • All other herbs, supplements or green tea (other than those noted above) have been discontinued 3 months prior to screening.
  • Ability to provide reproducible QMT bicep score with no more than 15% variation between scores during screening.
  • Normal blood clotting ability evidenced by a platelet function assessment (PFA).

Exclusion Criteria:

  • Currently enrolled in another treatment clinical trial.
  • History of significant concomitant illness or significant impairment of renal or hepatic function.
  • History of impairment of blood clotting ability (as evidenced by increased PT/PTT or PFA over the upper limit of normal (ULN)).
  • Recent cerebral or retinal hemorrhage.
  • History of bleeding diathesis or gastric ulcer.

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): NCT00243789

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, St. Louis
St. Louis, Missouri, United States, 63110
United States, Pennsylvania
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
United States, Tennessee
University of Tennessee
Memphis, Tennessee, United States, 38104
Hospital Frances
Buenos Aires, Argentina, 1434
Australia, Victoria
Children's Hospital
Melbourne, Victoria, Australia, 3052
Canada, Alberta
Alberta Children's Hospital
Calgary, Alberta, Canada, T2T 5C7
University of Alberta
Edmonton, Alberta, Canada, T6G 2J3
Hadassah Hospital, Mt. Scopus
Jerusalem, Israel, 91240
IRCCS C Mondino Foundation
Pavia, Italy, 27100
Sponsors and Collaborators
Cooperative International Neuromuscular Research Group
Study Chair: Diana Escolar, MD Children's National Medical Center, Center for Genetic Medicine

Additional Information:
Responsible Party: Study Chair, CINRG Identifier: NCT00243789     History of Changes
Other Study ID Numbers: CNMC0705
First Posted: October 25, 2005    Key Record Dates
Last Update Posted: October 27, 2011
Last Verified: October 2011

Keywords provided by Cooperative International Neuromuscular Research Group:
Muscular Dystrophy

Additional relevant MeSH terms:
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