CompletedPhase 1

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

France9 participantsStarted 2006-11

Plain-language 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).

Who can participate

Age range15 Years

SexALL

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Inclusion criteria

✓. Confirmed diagnosis of LGMD 2C including:
✓. Lower age limit of 15 years
✓. Males and females may be equally enrolled
✓. Adequate carpi radialis muscle bulk for muscle biopsy as assessed by examination. Subjects should be able to communicate with the investigation staff. They should be able to understand, to comply with and to perform all needed evaluations during the trial period, including muscle strength tests. Forearm muscle strength should be of at least 3+ as assessed through the British Medical Research Council (MRC) Manual Muscle Testing (MMT) scale.
✓. Subjects should be able and willing to return for follow up
✓. Subjects should be able and willing to give signed informed consent. For minor subjects, a signed informed consent will be given by legally authorized representative
✓. Eligible subjects belonging to a multiplex family should not be enrolled in the same cohort.

Exclusion criteria

✕. Severity of disease and presence of ill-prognosis complications:
✕. Underlying conditions, diseases or active viral infections likely to increase risk of complications or to interfere with the investigational treatment:

What they're measuring

Number of patients with adverse events or general or local signs as a measure of clinical safety

Timeframe: 6 months

Trial details

NCT IDNCT01344798

SponsorGenethon

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2010-06

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