Clinical Assessment Study in Crigler-Najjar Syndrome
Purpose
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.
Condition
- Crigler-Najjar Syndrome
Eligibility
- Eligible Ages
- Over 1 Year
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
More Details
- Status
- Completed
- Sponsor
- Audentes Therapeutics