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Through this study, researchers are investigating whether the study drug, Carbaglu, also known as N-carbamylglutamate or NCG, reduces blood ammonia levels and helps to protect the brain in cases of the genetic diseases: propionic acidemia; methylmalonic acidemia; carbamylphosphate synthetase I deficiency; or ornithine transcarbamylase deficiency. When high levels of ammonia accumulate in the blood, a condition called hyperammonemia, brain damage and other serious problems can occur. Carbaglu is already approved for treatment of children and adults with N-acetylglutamate synthase deficiency, another disease with high blood ammonia levels.
Subject to exclusion criteria, the study is accepting patients of all ages and of both genders, who have been hospitalized due to a high blood ammonia level related to propionic acidemia; methylmalonic acidemia; carbamylphosphate synthetase I deficiency; or ornithine transcarbamylase deficiency.
Males and Females: All ages
Enrolled patients will continue to receive the standard medical treatment for their disease. Additionally, while in the study, during each hospitalization for high blood ammonia, the patient receive twice-daily doses of either Carbaglu or a placebo, based on random selection, for up to 7 days while in the hospital. While in the hospital, participants will receive tests and visits by members of the study team, and within three days after discharge will be contacted by the study team as part of follow up care.
Visits: Not applicable; Occurs during hospitalization for hyperammonemia
Duration: Up to 4 years
National Institutes of Health
Study Description at National Institutes of Health
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Sandra Braden, RN, BSN
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
Children’s Hospital of Pittsburgh of UPMC
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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