LUSTRO: A Clinical Assessment Study in Crigler Najjar syndrome
Research type
Research Study
Full title
LUSTRO: A Clinical Assessment Study in Crigler Najjar syndrome
IRAS ID
219374
Contact name
Anil Dhawan
Contact email
Sponsor organisation
Audentes Therapeutics
Duration of Study in the UK
2 years, 6 months, 31 days
Research 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. This study is collecting clinical, disease and treatment data that will be used to help design future clinical studies.
Subjects will be assessed according to the schedule of events for a minimum of 3 months, and/or until the subject chooses to enroll in an AT342 interventional study Crigler-Najjar syndrome (if inclusion/exclusion criteria allow), withdraws from the study, or Audentes terminates the study.
This study will characterize bilirubin levels in subjects with Crigler-Najjar syndrome who require daily phototherapy. Initially, bilirubin variability will be assessed by twice-weekly blood draws for a period of 10 weeks. Following the initial 10-week period in the study, bilirubin will be assessed twice-daily before and after phototherapy at a frequency of twice a week through week 12.
Following the initial 12-week period in the study, bilirubin will be assessed every 2 weeks for the duration of the study. At Week 6, to assess changes in bilirubin levels over a 24-hour period during phototherapy and also off phototherapy, bilirubin levels will be assessed by frequent monitoring over a 24-hour period.REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
17/WM/0040
Date of REC Opinion
2 Mar 2017
REC opinion
Unfavourable Opinion