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Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy

This study has been completed.
Information provided by:
Cooperative International Neuromuscular Research Group Identifier:
First received: October 21, 2005
Last updated: October 26, 2011
Last verified: October 2011

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 Intervention Phase
Muscular Dystrophy, Duchenne Drug: Pentoxifylline Phase 1 Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:

Further study details as provided by Cooperative International Neuromuscular Research Group:

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

Enrollment: 64
Study Start Date: September 2005
Study Completion Date: January 2008
Primary Completion Date: December 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.


Ages Eligible for Study:   7 Years and older   (Child, Adult, Senior)
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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: 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
  More Information

Additional Information:
Responsible Party: Study Chair, CINRG Identifier: NCT00243789     History of Changes
Other Study ID Numbers: CNMC0705
Study First Received: October 21, 2005
Last Updated: October 26, 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
Antioxidants processed this record on September 21, 2017