Clinical trial NCT00243789: Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy
Official title: A Double-Blinded Randomized Placebo Controlled Study of Daily Pentoxifylline as a Rescue Treatment in DMD
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Muscular Dystrophy, Duchenne
Keywords: Duchenne
Genetic
Muscular Dystrophy
DMD
Lead sponsor: Cooperative International Neuromuscular Research Group
Sponsor class: NETWORK
First posted: 2005-10-25
Enrollment: 64
Sex: MALE
Minimum age (years): 7
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Eligibility 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.

Primary outcomes:
measure: 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.
timeFrame: January 2008
Secondary outcomes:
measure: Strength of arm, leg and grip QMT scores Measured Screening and Months 1, 3, 6, 9 & 12
timeFrame: January 2008
measure: Manual Muscle Testing (MMT) score measured at screening and months 1, 3, 6, 9 & 12 using the Medical Research Council (MRC) scoring system.
timeFrame: January 2008
measure: Functional evaluations measured at screening and months 1, 3, 6, 9 & 12
timeFrame: January 2008
measure: 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.
timeFrame: January 2008
measure: pulmonary function test (PFA's) measured at screening and months 1, 3, 6, 9 & 12
timeFrame: January 2008
measure: Pediatric Quality of Life (PQOL) measured at screening and months 1, 3, 6, 9 & 12
timeFrame: January 2008
measure: Goniometry measured at screening and months 1, 3, 6, 9 & 12
timeFrame: January 2008
measure: TNF-alpha and TGF-beta measured at screening and months 1, 3, 6, 9 & 12
timeFrame: February 2008
Other outcomes:
Locations:
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: St. Louis
country: United States
facility: Washington University, St. Louis
state: Missouri
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh
state: Pennsylvania
city: Memphis
country: United States
facility: University of Tennessee
state: Tennessee
city: Buenos Aires
country: Argentina
facility: Hospital Frances
city: Melbourne
country: Australia
facility: Children's Hospital
state: Victoria
city: Calgary
country: Canada
facility: Alberta Children's Hospital
state: Alberta
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Jerusalem
country: Israel
facility: Hadassah Hospital, Mt. Scopus
city: Pavia
country: Italy
facility: IRCCS C Mondino Foundation
Study officials:
affiliation: Children's National Medical Center, Center for Genetic Medicine
name: Diana Escolar, MD
role: STUDY_CHAIR
References: