drugset
drugset / Trial / NCT01344798

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

NCT01344798 ↗

Phase 1 Completed 9 enrolled Genethon
Non-randomizedSingle-groupOpen-labelTreatment

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).

Timeline

Start
2006-11
Primary completion
2010-06
Completion
2010-06

Drugs

EvaluationDrugModalityDoseRoute
Subject AAV1-gamma-sarcoglycan vector Gene therapy (AAV / viral vector) 3e+09 vg Intramuscular
Subject AAV1-gamma-sarcoglycan vector Gene therapy (AAV / viral vector) 1.5e+10 vg Intramuscular
Subject AAV1-gamma-sarcoglycan vector Gene therapy (AAV / viral vector) 4.5e+10 vg Intramuscular

Indications

autosomal recessive limb-girdle muscular dystrophy type 2C