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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
LogicBio Therapeutics
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 412-692-5662
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
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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