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Thank you for your interest in this study. Alpha-1 antitrypsin deficiency (ATD) is a genetic condition that can cause liver disease in children and adults as well as serious lung disease in adults. ATD occurs when there is a lack of protein in the blood called alpha-1 antitrypsin (AT). Produced by the liver, the main function of AT is to protect the lungs from inflammation caused by infection and inhaled irritants.
ATD affects 1 in 2,000 births. The resultant liver scarring is the most common genetic cause for which children undergo liver transplantation, presently the only therapeutic option. However, laboratory work has demonstrated that a drug called carbamazepine may help, and may have the potential to reverse fibrosis. Carbamazepine (also known by the brand name Tegretol) is presently used to treat seizure disorders, and it is well understood by the medical community. This has helped enable Children’s Hospital of Pittsburgh of UPMC to initiate this study, which is supported by the National Institutes of Health and Novartis.
Our study is currently enrolling male and female candidates ages 14 and up who have been diagnosed with liver disease due to ATD. For more complete information about the study and to learn how to enroll your patients, please see this Letter to Physicians (PDF).
Below are links to additional information about ATD to help with patient education and enrollment in this study.
If you wish to refer a patient to the study, please:
You may also post copies of the Alpha-1 Study Informational Flier (PDF) within your practice.
Several published works about this investigation are listed below. Review a complete list of published works about this investigation.
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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