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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00243789
• Date of registration: 21/10/2005
• Prospective Registration: No
• Primary sponsor: Cooperative International Neuromuscular Research Group
• Public title: Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy
• Scientific title: A Double-Blinded Randomized Placebo Controlled Study of Daily Pentoxifylline as a Rescue Treatment in DMD
• Date of first enrolment: September 2005
• Target sample size: 64
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00243789
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
• Phase: Phase 1/Phase 2

Countries of recruitment

Argentina, Australia, Canada, Israel, Italy, United States

Contacts

• Name: Diana Escolar, MD
• Affiliation: Children's National Medical Center, Center for Genetic Medicine

Key inclusion & exclusion criteria

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.



Age minimum: 7 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Muscular Dystrophy, Duchenne

Intervention(s)

Drug: Pentoxifylline

Primary Outcome(s)

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(s)

Functional evaluations measured at 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]

pulmonary function test (PFA's) 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]

Strength of arm, leg and grip QMT scores Measured 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]

Pediatric Quality of Life (PQOL) 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]

Secondary ID(s)

CNMC0705

Source(s) of Monetary Support

Please refer to primary and secondary sponsors