Children's Hospital is part of the UPMC family.
Our Sites
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 #6 Nationally by U.S. News & World Report.
The goal of this study is to see how well people with methylmalonic acidemia (MMA) and propionic acidemia (PA) accept treatment with the investigational drug BBP-671. The study will evaluate the effects of BBP- 671 on certain biomarkers and will determine if BBP-671 is safe and tolerable. Through this study, researchers hope to further understand BBP-671 and these rare genetic diseases that impair the proper digestion of fats and proteins, leading to a buildup of toxic acidic substances in the blood and altered energy metabolism.
Subject to certain exclusion criteria, this study is accepting patients, ages 18 through 55 years, who have been diagnosed with methylmalonic acidemia or propionic acidemia. Males and Females: Ages 18 through 55 years
Following screening and a run-in period that includes physicals and blood testing, participants will be randomly divided into groups that will either receive the study drug or a placebo. The study drug will be taken twice daily for a 6-week period, with clinic visits scheduled at specific intervals for physical examinations, laboratory tests including blood collection and electrocardiograms. Visits: 12 Duration: 4 months
CoA Therapeutics, Inc.
Study Description at National Institutes of Health Methylmalonic Acidemia Genetic and Genomic Medicine Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact: Michele Graham, RN 412-692-3476
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.