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The goal of this multi-center investigation is to see if the drug HST5040 will help in the treatment of methylmalonic acidemia (MMA) and propionic acidemia (PA), while also studying its safety and side effects. MMA and PA are rare genetic diseases, which 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 people with MMA and PA ages 2 and older.
Males and Females: Ages 2 years and older
In this 3-part study, the drug is taken orally or via gastronomy tube twice daily. In the first part of the study, participants are monitored without treatment for 4 weeks before starting the drug therapy. Thereafter, the dosage level will be increased every 4 weeks until an optimal dose is achieved. In the second part of the study, participants will be randomly divided to either continue with their optimal dose of the study drug or take a placebo for roughly the first half of the 7-month study period. Then they will switch to the placebo or study drug, respectively, for the second half. In the final part of the study, which may extend for 5 years, participants will continue to receive their optimal dose, as determined by results from the first two parts of the study.
Visits: Up to 30, some of which may be home health visits
Duration: Up to 6 years
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:
Lead Clinical Research Coordinator
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
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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