Read the Latest
Children's Hospital is part of the UPMC family.
Be safe anytime, anywhere.
To find a pediatrician or pediatric specialist, please call 412-692-7337 or search our directory.
A resource for our network of referring physicians.
For more information about research, please call our main office at 412-692-6438.
Ranked #9 Nationally by U.S. News & World Report.
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
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Mark Tumblin, Clinical Research Coordinator
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.
With myCHP, you can request appointments, review test results, and more.
For questions about a hospital bill call:
To pay your bill online, please visit UPMC's online bill payment system.
Interested in giving to Children's Hospital? Support the hospital by making a donation online, joining our Heroes in Healing monthly donor program, or visiting our site to learn about the other ways you can give back.