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

Primary Purpose

Muscular Dystrophy, Duchenne

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
Pentoxifylline
Sponsored by
Cooperative International Neuromuscular Research Group
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Muscular Dystrophy, Duchenne focused on measuring Duchenne, Genetic, Muscular Dystrophy, DMD

Eligibility Criteria

7 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Children's National Medical Center
  • Mayo Clinic
  • Washington University, St. Louis
  • Children's Hospital of Pittsburgh
  • University of Tennessee
  • Hospital Frances
  • Children's Hospital
  • Alberta Children's Hospital
  • University of Alberta
  • Hadassah Hospital, Mt. Scopus
  • IRCCS C Mondino Foundation

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

No Intervention

Arm Label

1

2

Arm Description

Pentoxifylline

Placebo

Outcomes

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.

Secondary Outcome Measures

Strength of arm, leg and grip QMT scores Measured Screening and Months 1, 3, 6, 9 & 12
Manual Muscle Testing (MMT) score measured at screening and months 1, 3, 6, 9 & 12 using the Medical Research Council (MRC) scoring system.
Functional evaluations measured at screening and months 1, 3, 6, 9 & 12
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.
pulmonary function test (PFA's) measured at screening and months 1, 3, 6, 9 & 12
Pediatric Quality of Life (PQOL) measured at screening and months 1, 3, 6, 9 & 12
Goniometry measured at screening and months 1, 3, 6, 9 & 12
TNF-alpha and TGF-beta measured at screening and months 1, 3, 6, 9 & 12

Full Information

First Posted
October 21, 2005
Last Updated
October 26, 2011
Sponsor
Cooperative International Neuromuscular Research Group
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1. Study Identification

Unique Protocol Identification Number
NCT00243789
Brief Title
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

2. Study Status

Record Verification Date
October 2011
Overall Recruitment Status
Completed
Study Start Date
September 2005 (undefined)
Primary Completion Date
December 2007 (Actual)
Study Completion Date
January 2008 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Cooperative International Neuromuscular Research Group

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Dystrophy, Duchenne
Keywords
Duchenne, Genetic, Muscular Dystrophy, DMD

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
64 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Active Comparator
Arm Description
Pentoxifylline
Arm Title
2
Arm Type
No Intervention
Arm Description
Placebo
Intervention Type
Drug
Intervention Name(s)
Pentoxifylline
Other Intervention Name(s)
Trental
Intervention Description
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.
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
Strength of arm, leg and grip QMT scores Measured Screening and Months 1, 3, 6, 9 & 12
Time Frame
January 2008
Title
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
Title
Functional evaluations measured at screening and months 1, 3, 6, 9 & 12
Time Frame
January 2008
Title
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
Title
pulmonary function test (PFA's) measured at screening and months 1, 3, 6, 9 & 12
Time Frame
January 2008
Title
Pediatric Quality of Life (PQOL) measured at screening and months 1, 3, 6, 9 & 12
Time Frame
January 2008
Title
Goniometry measured at screening and months 1, 3, 6, 9 & 12
Time Frame
January 2008
Title
TNF-alpha and TGF-beta measured at screening and months 1, 3, 6, 9 & 12
Time Frame
February 2008

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Diana Escolar, MD
Organizational Affiliation
Children's National Medical Center, Center for Genetic Medicine
Official's Role
Study Chair
Facility Information:
Facility Name
Children's National Medical Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Washington University, St. Louis
City
St. Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
University of Tennessee
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38104
Country
United States
Facility Name
Hospital Frances
City
Buenos Aires
ZIP/Postal Code
1434
Country
Argentina
Facility Name
Children's Hospital
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
Alberta Children's Hospital
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2T 5C7
Country
Canada
Facility Name
University of Alberta
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 2J3
Country
Canada
Facility Name
Hadassah Hospital, Mt. Scopus
City
Jerusalem
ZIP/Postal Code
91240
Country
Israel
Facility Name
IRCCS C Mondino Foundation
City
Pavia
ZIP/Postal Code
27100
Country
Italy

12. IPD Sharing Statement

Links:
URL
http://www.cinrgresearch.org
Description
Related Info

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

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