Clinical Trial: Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Phase I Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C
Brief Summary: The purpose of this trial is to study the evaluation of clinical safety and feasibility of gene therapy in patients with limb girdle muscular dystrophy type 2C (gamma-sarcoglycanopathy).
Detailed Summary:
Sponsor: Genethon
Current Primary Outcome: Number of patients with adverse events or general or local signs as a measure of clinical safety [ Time Frame: 6 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Number of patients with modified biological values (blood count, standard biochemistry, viral serology) [ Time Frame: 6 months ]
Assessment of biological tolerance:
- blood count
- standard biochemistry
- CPK viral serology (hepatitis B & C)
- number of patients with changed or increased humoral immunity to AAV [ Time Frame: 6 months ]assessment of anti-AAV antibodies titers
- Number of patients with changed/increased humoral immunity to transgene [ Time Frame: 6 months ]assessment of anti-gamma-sarcoglycan antibodies titers
- Number of patients with changed/increased cellular immunity to AAV [ Time Frame: 6 months ]assessment cellular immunity against AAV (ELispot assay)
- Number of patients with changed/increased cellular immunity to transgene [ Time Frame: 6 months ]assessment cellular immunity against gamma-sarcoglycan (ELispot assay)
- number of patients with positively stained muscular fibers to gamma-sarcoglycan protein [ Time Frame: 30 days ]Muscular biopsy immunohistaining for the detection of gamma-sarcoglycan
- Number of patients with modified/decreased muscular force [ Time Frame: 6 months ]functional testing of treated muscle through a specially designed ergometer
Original Secondary Outcome: Same as current
Information By: Genethon
Dates:
Date Received: March 17, 2011
Date Started: November 2006
Date Completion:
Last Updated: April 28, 2011
Last Verified: April 2011