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This study uses a type of gene therapy to address methylmalonic acidemia (MMA), a rare genetic defect that causes problems with digestion of fats and proteins resulting in a buildup of methylmalonic acid in tissues and blood. While certain therapies can address symptoms, there presently is no cure for MMA. Through this trial, researchers will assess effects of a single intravenous infusion of hLB-001 in pediatric patients with MMA characterized by mutation of the methylmalonyl-CoA mutase gene (MMUT).
This study is enrolling candidates, ages 6 months to 12 years of age, who have MMA characterized by the MMUT mutation.
Boys and Girls: 6 months to 12 years of age
Following a 16-day screening/run-in period for testing, participants will receive the study drug via intravenous infusion at the hospital. Participants will remain in the hospital for at least 2 days for testing and observation before being discharged. They will be followed by the research team for about 12 months and will be invited to participate in a longer-term follow-up study that tracks them for up to 15 years.
Visits: Requires hospitalization for the infusion and observation
Duration: 12 months
Study Description at National Institutes of Health
Genetic and Genomic Medicine Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Elizabeth McCracken, MS, CGC
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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