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This study tests an investigational drug called mRNA-3927 for the potential treatment of propionic acidemia (PA). Researchers will study effects and potential side effects of the drug. Caused by a rare genetic defect, PA results in problems with an enzyme responsible for breaking down protein and fats, making it difficult to turn food into energy. Conventional treatments, including liver transplant, can help symptoms of PA, but do not fix the cause of the disease.
This study is enrolling male and female candidates age 1 and older who have been diagnosed with PA and who have not had an organ transplant.
Boys and Girls: 1 year and older
The study drug is given by intravenous infusion scheduled several weeks apart for a total of 12 infusions over 9 months. Participants are required to stay in the hospital for observation 2 days and nights before the first infusion and for several days after. Hospitalization will be required for the subsequent 2 doses, and doses 4-12 are done on an outpatient basis. Patients will be seen 11 more times in the 2-year follow-up period after the treatment period ends.
Visits: About 40
Duration: About 3 years
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
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