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Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)

NCT00494195 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2013-02-05

No results posted yet for this study

Summary

Limb girdle muscular dystrophy type 2D (LGMD2D) is a genetic disease that affects skeletal muscle. Insufficient levels of the protein alpha-sarcoglycan result in muscle weakness that worsens over time. The purpose of this study is to evaluate the safety and effectiveness of gene therapy in treating children and adults with LGMD2D.

Conditions

  • Muscular Dystrophies

Interventions

GENETIC

rAAV1.tMCK.human-alpha-sarcoglycan- First cohort

The first cohort of subjects with LGMD2D (alpha-sarcoglycan deficiency) and proven mutations will undergo gene transfer with a minimum of three subjects enrolled into this cohort and will receive a total of 1.5 ml volume of study agent in two to six separate injections into the selected muscle (extensor digitorum brevis) or other muscle if more appropriate upon considering the individual patient. The dose will be 3.25 X 10 to the 11 vg in 1.5 ml. In each cohort, only one extremity will receive vector with transgene while the opposite extremity will be injected with placebo.

GENETIC

Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort

The second cohort will receive the same dosis of 3.25 X 10 to the 11 vg in 1.5 ml delivered to muscle according to the same paradigm. In each cohort, only one extremity will receive vector with transgene while the opposite extremity will be injected with placebo.

Sponsors & Collaborators

  • National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

    collaborator NIH

  • Muscular Dystrophy Association

    collaborator OTHER

  • Nationwide Children's Hospital

    lead OTHER

Principal Investigators

  • Jerry R. Mendell, MD · The Research Institute at Nationwide Children's Hospital

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-03-31
Primary Completion
2011-06-30
Completion
2011-08-31

Countries

  • United States

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 NCT00494195 on ClinicalTrials.gov