Clinical Assessment Study in Crigler-Najjar Syndrome (LUSTRO)
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Ages Eligible for Study:
1 Year and older (Child, Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
The study will enroll Crigler-Najjar syndrome subjects aged equal or greater than 1 year of age requiring daily phototherapy.
Key Inclusion Criteria:
Subject has a diagnosis of Crigler-Najjar syndrome resulting from a confirmed mutation in the UGT1A1 gene
Subject is aged equal or greater than 1 year of age
Subject receives daily phototherapy for a minimum of 6 hours within a 24-hour period (daily illumination time)
Subject is currently participating in an interventional study or has received gene or cell therapy
Subject has received a whole liver, partial liver, or hepatocyte transplant; or subject has a liver transplant scheduled within the planned participation period of this study
Subject has significant cholestatic disease, in the opinion of the investigator
Subject is receiving phenobarbital or other known inducer of UGT1A1 within 30 days of screening
Subject has any clinically significant underlying liver disease (other than Crigler-Najjar syndrome), in the opinion of the investigator
Subject has a history of, or currently has, a clinically important condition other than Crigler-Najjar syndrome, in the opinion of the investigator