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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.
Duration: 4 months
CoA Therapeutics, Inc.
Study Description at National Institutes of Health
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Michele Graham, RN
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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