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

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

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.