NCT01344798

Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C

Phase I Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Genethon · Academic / Other
• Sex: All
• Age: 15 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Limb Girdle Muscular Dystrophy Type 2C
• Gamma-sarcoglycanopathy

Interventions

Type	Name	Description
BIOLOGICAL	AAV1-gamma-sarcoglycan vector injection	single intramuscular injection into carpi radialis muscle under open procedure

Timeline

• Start date: 2006-11-01
• Primary completion: 2010-06-01
• Completion: 2010-06-01
• First posted: 2011-04-29
• Last updated: 2011-04-29

Locations

1 site across 1 country: France