LUSTRO: Clinical Assessment Study of Crigler-Najjar Syndrome
Research type
Research Study
Full title
LUSTRO: Clinical Assessment Study of Crigler-Najjar Syndrome
IRAS ID
227403
Contact name
Anil Dhawan
Contact email
Sponsor organisation
Audentes Therapeutics
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 1 months, 31 days
Research summary
This is a prospective, non-interventional clinical assessment study to evaluate Crigler-Najjar syndrome subjects requiring daily phototherapy. The prospective evaluation of disease presentation will provide a longitudinal baseline and within-patient control for the planned interventional study. Subjects will be assessed according to the schedule of events for a minimum of 2 months. Bilirubin variability will be assessed by twice-weekly blood draws for a period of 10 weeks. At Week 6, 5-7 (based on subject age) blood samples will be collected over a 24-hour period to assess changes in bilirubin during phototherapy and time off phototherapy. Following the initial 10-week period in the study, bilirubin will be assessed twice-daily before and after phototherapy at Week 11 and 12. Following the initial 12-week period in the study, bilirubin will be assessed every 2 weeks for the duration of the study.
REC name
South Central - Berkshire Research Ethics Committee
REC reference
17/SC/0250
Date of REC Opinion
23 Jun 2017
REC opinion
Further Information Favourable Opinion