Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)
Completed · Phase 1 · Has a placebo group
Conditions studied: Muscular Dystrophies
In brief
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.
Key facts
- Study ID
- NCT00494195
- Run by
- Nationwide Children's Hospital
- People needed
- 6
- Starts
- 2008-03-01
- Expected to finish
- 2011-08-01
- Last updated by the study team
- 2013-02-05
Who can join
Age: 5 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Six LGMD2D subjects ages 5 and older based on the clinical degree of involvement (impaired muscle function/weakness, sufficient muscle preservation)
- Preservation of EDB muscle or another muscle if judged more favorable because of adequate muscle mass for gene transfer
- Males and females of any ethnic group
- Established mutations of an -SG gene on both alleles
- Ability to cooperate for testing
- Sexually active patients must be willing to practice a reliable method of contraception during the study
You may not qualify if…
- Active viral infection (symptoms listed in section 9.0 of the protocol)
- LGMD2D subjects without weakness or functional loss
- Cardiomyopathy based on clinical exam and ECHO with ejection fraction less than 40%
- HIV infected
- Hepatitis A, B, or C infected
- Autoimmune diseases and immunosuppressive drugs (other than pulse methylprednisolone at time of gene transfer)
- Persistent leucopenia or leucocytosis (WBC less than or equal to 3.5 K/cu mm or at least 20.0 K/ cu mm) or neutrophils less than 1.5 K/ cu mm
- Concomitant illness or requirement for chronic drug treatment that in the opinion of the Principal Investigator creates unnecessary risks for gene transfer
- Pregnancy
- Abnormal laboratory values considered clinically significant
- Alcoholism (CAGE questionnaire), and laboratory tests such as GGT and MCV
Where it is running
- The Research Institute at Nationwide Children's Hospital — Columbus, Ohio, United States
Full record on ClinicalTrials.gov
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