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This international multi-center study compares the effects of glycerol phenylbutyrate and sodium phenylbutyrate to manage urea cycle disorders (UCDs). Rare genetic conditions, UCDs cause accumulation of high levels of ammonia in the blood, which can lead to brain damage and other serious problems. Glycerol phenylbutyrate, known by the trade name Ravicti® and sodium phenylbutyrate, known as Buphenyl®, work similarly by helping to remove unwanted ammonia from the body. Both Raviciti, a liquid, and Buphenyl, a tablet or powder, can be taken orally or by feeding tube.
Candidates must have a suspected or confirmed UCD diagnosis without having been treated over a long period of time with either of the study drugs.
Males and Females: All ages
By random selection, participants will be assigned to a treatment arm that starts with either Raviciti or Buphenyl to be taken 3 times a day for 4 weeks. After that, participants who started on Buphenyl will transition to Raviciti for 8 more weeks and those who started on Raviciti will continue on the drug. During the dosing periods, participants will return to the clinic for examinations, blood tests and other evaluations. A 12-week safety extension period will provide additional examinations and monitoring.
Duration: About 25 weeks
Study Description at National Institutes of Health
Urea Cycle Disorders
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Jessica Lindenberger, MSN, CRNP, FNP-C
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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