Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00243789




Registration number
NCT00243789
Ethics application status
Date submitted
21/10/2005
Date registered
25/10/2005
Date last updated
27/10/2011

Titles & IDs
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
Secondary ID [1] 0 0
CNMC0705
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Muscular Dystrophy, Duchenne 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Pentoxifylline

Active comparator: 1 - Pentoxifylline

No intervention: 2 - Placebo


Treatment: Drugs: 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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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.
Timepoint [1] 0 0
January 2008
Secondary outcome [1] 0 0
Strength of arm, leg and grip QMT scores Measured Screening and Months 1, 3, 6, 9 & 12
Timepoint [1] 0 0
January 2008
Secondary outcome [2] 0 0
Manual Muscle Testing (MMT) score measured at screening and months 1, 3, 6, 9 & 12 using the Medical Research Council (MRC) scoring system.
Timepoint [2] 0 0
January 2008
Secondary outcome [3] 0 0
Functional evaluations measured at screening and months 1, 3, 6, 9 & 12
Timepoint [3] 0 0
January 2008
Secondary outcome [4] 0 0
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.
Timepoint [4] 0 0
January 2008
Secondary outcome [5] 0 0
pulmonary function test (PFA's) measured at screening and months 1, 3, 6, 9 & 12
Timepoint [5] 0 0
January 2008
Secondary outcome [6] 0 0
Pediatric Quality of Life (PQOL) measured at screening and months 1, 3, 6, 9 & 12
Timepoint [6] 0 0
January 2008
Secondary outcome [7] 0 0
Goniometry measured at screening and months 1, 3, 6, 9 & 12
Timepoint [7] 0 0
January 2008
Secondary outcome [8] 0 0
TNF-alpha and TGF-beta measured at screening and months 1, 3, 6, 9 & 12
Timepoint [8] 0 0
February 2008

Eligibility
Key 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).
Minimum age
7 Years
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/09/2005
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/01/2008
Sample size
Target
Accrual to date
Final
64
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Children's Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
3052 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
District of Columbia
Country [2] 0 0
United States of America
State/province [2] 0 0
Minnesota
Country [3] 0 0
United States of America
State/province [3] 0 0
Missouri
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania
Country [5] 0 0
United States of America
State/province [5] 0 0
Tennessee
Country [6] 0 0
Argentina
State/province [6] 0 0
Buenos Aires
Country [7] 0 0
Canada
State/province [7] 0 0
Alberta
Country [8] 0 0
Israel
State/province [8] 0 0
Jerusalem
Country [9] 0 0
Italy
State/province [9] 0 0
Pavia

Funding & Sponsors
Primary sponsor type
Other
Name
Cooperative International Neuromuscular Research Group
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Diana Escolar, MD
Address 0 0
Children's National Medical Center, Center for Genetic Medicine
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT00243789