Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
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|ClinicalTrials.gov Identifier: NCT02354781|
Recruitment Status : Completed
First Posted : February 3, 2015
Last Update Posted : December 2, 2017
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Biological: rAAV1.CMV.huFollistin344||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||3 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I/II Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy|
|Study Start Date :||January 2015|
|Actual Primary Completion Date :||November 2017|
|Actual Study Completion Date :||November 2017|
The vector will be delivered to both limbs via multiple, direct intramuscular injections of rAAV1.CMV.huFollistin344; the number of injections per muscle will depend on the size of the patient. A total dose of 2.4E12 vg/kg (1.2E12vg/kg/limb) will be delivered to the lower limbs of 6 DMD subjects
Six DMD patients will receive rAAV1.CMV.huFollistatin344 to both limbs by multiple injections to gluteal muscles, quadriceps and tibialis anterior muscles.
- Organ system toxicity based on blood chemistries and immune studies [ Time Frame: 2 years ]The blood chemistries and immune studies to monitor organs system abnormalities and T cell response measured by ELISpots.
- Muscle Function [ Time Frame: 2 years ]The distance walked on the 6MWT is the major functional outcome to be evaluated.
- Size of muscle fibers with lack of toxicity of follistatin following gene transfer [ Time Frame: 2 years ]• Muscle biopsies on quadriceps muscles (a muscle biopsy on one leg at baseline screening visit and the post gene transfer biopsy on the opposite leg at day 180.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02354781
|United States, Ohio|
|Nationwide Children's Hospital|
|Columbus, Ohio, United States, 43205|
|Principal Investigator:||Jerry R Mendell, MD||Director, Center for Gene Therapy, Nationwide Children's Hospital|