Alagille Syndrome clinical trials at UCSF
3 in progress, 1 open to eligible people
Alagille syndrome is a genetic disorder that affects the liver, heart, and other organs. UCSF is studying the long-term safety and effectiveness of a drug called odevixibat for people with this condition. This drug aims to help manage symptoms over time.
Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
open to all eligible people
The purpose of this study is to assess the long-term safety and effectiveness of odevixibat in participants with Alagille syndrome (ALGS). The participants of this study will have ALGS a rare genetic disorder that can affect multiple organ systems of the body including the liver, heart, skeleton, eyes and kidneys. Common symptoms, which often develop during the first three months of life, include blockage of the flow of bile from the liver (cholestasis), yellowing of the skin and mucous membranes (jaundice), poor weight gain and growth and severe itching (pruritis). The drug used for the study is odevixibat and was authorized for the treatment of cholestatic pruritus in infants with ALGS over 12 months of age by the United States Food and Drug Administration on 13 June 2023.
San Francisco, California and other locations
Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)
Sorry, currently not accepting new patients, but might later
Cholestasis is a condition in which bile is not properly transported from the liver to the small intestine. Cholestasis can be caused by an array of childhood diseases, including the genetic diseases Alagille syndrome (ALGS), alpha-1 antitrypsin (a-1AT) deficiency, bile acid synthesis and metabolism defects, and progressive familial intrahepatic cholestasis (PFIC) or benign recurrent intrahepatic cholestasis(BRIC). This study will investigate the natural history and progression of the four previously mentioned cholestatic liver diseases to provide a better understanding of the causes and effects of the diseases.
San Francisco, California and other locations
Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
Sorry, not accepting new patients
An open-label, single-arm, multi-centre program providing treatment access to ALGS patients with cholestatic pruritus in the US. Access can be requested through Clinigen Healthcare Ltd via email MirumALGS@clinigengroup.com or telephone +1-877-768-4303.
San Francisco, California and other locations
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