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

Muscular Dystrophy, Duchenne Clinical Trial

Official title:

A Double-Blinded Randomized Placebo Controlled Study of Daily Pentoxifylline as a Rescue Treatment in DMD

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00243789
• Other study ID #: CNMC0705
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: October 21, 2005
• Last updated: October 26, 2011
• Start date: September 2005
• Est. completion date: January 2008

Study information

• Verified date: October 2011
• Source: Cooperative International Neuromuscular Research Group
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 64
• Est. completion date: January 2008
• Est. primary completion date: December 2007
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 7 Years and older

Eligibility

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.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

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.

Locations

Country	Name	City	State
Argentina	Hospital Frances	Buenos Aires
Australia	Children's Hospital	Melbourne	Victoria
Canada	Alberta Children's Hospital	Calgary	Alberta
Canada	University of Alberta	Edmonton	Alberta
Israel	Hadassah Hospital, Mt. Scopus	Jerusalem
Italy	IRCCS C Mondino Foundation	Pavia
United States	University of Tennessee	Memphis	Tennessee
United States	Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Mayo Clinic	Rochester	Minnesota
United States	Washington University, St. Louis	St. Louis	Missouri
United States	Children's National Medical Center	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Cooperative International Neuromuscular Research Group

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.		January 2008	No
Secondary	Strength of arm, leg and grip QMT scores Measured Screening and Months 1, 3, 6, 9 & 12		January 2008	No
Secondary	Manual Muscle Testing (MMT) score measured at screening and months 1, 3, 6, 9 & 12 using the Medical Research Council (MRC) scoring system.		January 2008	No
Secondary	Functional evaluations measured at screening and months 1, 3, 6, 9 & 12		January 2008	No
Secondary	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.		January 2008	No
Secondary	pulmonary function test (PFA's) measured at screening and months 1, 3, 6, 9 & 12		January 2008	No
Secondary	Pediatric Quality of Life (PQOL) measured at screening and months 1, 3, 6, 9 & 12		January 2008	No
Secondary	Goniometry measured at screening and months 1, 3, 6, 9 & 12		January 2008	No
Secondary	TNF-alpha and TGF-beta measured at screening and months 1, 3, 6, 9 & 12		February 2008	No

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