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

NCT00243789 | Completed Phase 1 DMC

• 1 other identifier: CNMC0705
• Study Type: interventional
• Target: 64
• Locations: 6 countries
• Sites: 11

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.

Conditions

Muscular Dystrophy, Duchenne

Keywords

Duchenne; Genetic; Muscular Dystrophy; DMD

Outcome Measures

Primary Outcomes (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.

  January 2008

Secondary Outcomes (8)

• Strength of arm, leg and grip QMT scores Measured Screening and Months 1, 3, 6, 9 & 12

  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.

  January 2008

• Functional evaluations measured at screening and months 1, 3, 6, 9 & 12

  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.

  January 2008

• pulmonary function test (PFA's) measured at screening and months 1, 3, 6, 9 & 12

  January 2008

Study Arms (2)

1

ACTIVE COMPARATOR

Pentoxifylline

Drug: Pentoxifylline

2

NO INTERVENTION

Placebo

Interventions

Pentoxifylline DRUG

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.

Also known as: Trental

1

Eligibility Criteria

• Age: 7 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Cooperative International Neuromuscular Research Group: lead

Study Sites (11)

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Washington University, St. Louis

St Louis, Missouri, 63110, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

University of Tennessee

Memphis, Tennessee, 38104, United States

Location

Hospital Frances

Buenos Aires, 1434, Argentina

Location

Children's Hospital

Melbourne, Victoria, 3052, Australia

Location

Alberta Children's Hospital

Calgary, Alberta, T2T 5C7, Canada

Location

University of Alberta

Edmonton, Alberta, T6G 2J3, Canada

Location

Hadassah Hospital, Mt. Scopus

Jerusalem, 91240, Israel

Location

IRCCS C Mondino Foundation

Pavia, Italy, 27100, Italy

Location

Related Links

• Related Info

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies

Interventions

Pentoxifylline

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Theobromine; Xanthines; Purinones; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Diana Escolar, MD

  Children's National Medical Center, Center for Genetic Medicine

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: NETWORK

Study Record Dates

• First Submitted: October 21, 2005
• First Posted: October 25, 2005
• Study Start: September 1, 2005
• Primary Completion: December 1, 2007
• Study Completion: January 1, 2008
• Last Updated: October 27, 2011

Record last verified: 2011-10

Locations

AR (1); IT (1); IL (1); US (5); AU (1); CA (2)