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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 February 2015 |
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Main ID: |
NCT00428935 |
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Date of registration:
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26/01/2007 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy
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Scientific title:
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Phase 1 Clinical Trial of rAAV2.5-CMV-mini-Dystrophin Gene Vector in Duchenne Muscular Dystrophy |
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Date of first enrolment:
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March 2006 |
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Target sample size:
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6 |
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Recruitment status: |
Completed |
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URL:
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http://clinicaltrials.gov/show/NCT00428935 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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Phase:
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Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Jerry R. Mendell, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Nationwide Children's Hospital |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Known null mutation of the Dystrophin gene
- Male age of 5 years or older
- If taking corticosteroids, must have dose unchanged for the past 3 months
- Serum creatine kinase elevation greater than 10x normal value (established by
Children's Hospital)
- Progressive, symmetrical proximal muscle weakness of arms and legs
Exclusion Criteria:
- Unable to cooperate for muscle strength testing
- Joint contractures that prohibit muscle strength testing
- Concomitant illness
- Individuals predisposed to excessive vagal responses (bradyarrhythmia or hypotension)
- Controlled substance abuse
Age minimum:
5 Years
Age maximum:
15 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Duchenne Muscular Dystrophy
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Intervention(s)
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Biological: rAAV2.5-CMV-minidystrophin (d3990)
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Primary Outcome(s)
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Number of Participants with Adverse Events as a Measure of Safety and Tolerability
[Time Frame: followed for 2 years post injection]
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Secondary Outcome(s)
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mini-dystrophin gene expression at the site of gene transfer
[Time Frame: 90 days post injection]
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Maximal Volume Isometric Contraction Testing as a measure of muscle strength
[Time Frame: out to 2 years post injection]
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Secondary ID(s)
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CCRI IRB05-00118
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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