Clinical Trial: Clinical Assessment Study in Crigler-Najjar Syndrome
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational
Official Title: LUSTRO: A Clinical Assessment Study in Crigler-Najjar Syndrome
Brief Summary: 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.
Detailed Summary:
Sponsor: Audentes Therapeutics
Current Primary Outcome:
- Characterize the disease course and natural history of subjects with Crigler-Najjar syndrome [ Time Frame: Up to 2 years ]
- Assess variation in bilirubin levels over the course of the study and the variation of bilirubin levels over a 24-hr period [ Time Frame: Up to 2 years ]
- Assess phototherapy usage over the course of the study [ Time Frame: Up to 2 years ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Assess the humanistic and clinical burden of disease in Crigler-Najjar subjects and caregivers as measured by PedsQL [ Time Frame: Up to 2 years ]
Original Secondary Outcome: Same as current
Information By: Audentes Therapeutics
Dates:
Date Received: March 8, 2017
Date Started: March 10, 2017
Date Completion: February 2019
Last Updated: May 2, 2017
Last Verified: May 2017
