Clinical Assessment Study in Crigler-Najjar Syndrome (LUSTRO)

Audentes Therapeutics

Status: Completed

Conditions: Crigler-Najjar Syndrome

Study type: Observational

Funder types: Industry

Identifiers

NCT03078881

AT342-01

Details and patient eligibility

About

This is a Pre-Phase 1 prospective, non-interventional clinical assessment study to evaluate Crigler-Najjar syndrome subjects requiring daily phototherapy, aged 1 year and older.

Enrollment: 8 patients

Sex: All

Ages: 1+ year old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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)

Exclusion Criteria:

• 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