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

NCT01344798 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2011-04-29

No results posted yet for this study

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

BIOLOGICAL

AAV1-gamma-sarcoglycan vector injection

single intramuscular injection into carpi radialis muscle under open procedure

Sponsors & Collaborators

  • Genethon

    lead OTHER

Principal Investigators

  • Serge Herson, Prof · Assistance Publique - Hôpitaux de Paris

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2006-11-30
Primary Completion
2010-06-30
Completion
2010-06-30

Countries

  • France

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01344798 on ClinicalTrials.gov