Acceptability study of a new paediatric formulation of Orphacol®
Research type
Research Study
Full title
Acceptability study of a new paediatric formulation of Orphacol® (cholic acid) in paediatric patients treated for inborn errors in primary bile acid synthesis due to 3β-Hydroxy-Δ5-C27-steroid oxidoreductase deficiency or Δ4-3-Oxosteroid-5β-reductase deficiency.
IRAS ID
234601
Contact name
Anil Dhawan
Contact email
Sponsor organisation
Laboratoires CTRS
Duration of Study in the UK
0 years, 2 months, 2 days
Research summary
This is a study to evaluate how patients aged 5-13 will accept a new formulation of the medicine Orphacol®. This new formulation is taste-masked and will help to develop a product to be administered specifically to children. Patients will be asked how they find the product and parents will be asked how easy it is to prepare and give it to their children
This study will be conducted in several countries in patients suffering from inborn errors in primary bile acid synthesis due to 3β-Hydroxy-Δ5 C27-steroid oxidoreductase deficiency or Δ4-3-Oxosteroid-5β-reductase deficiency, and currently treated with Orphacol® capsules or other primary bile acids.
Investigators are Hospital-based physicians, managing paediatric patients suffering from inborn errors in primary bile acid synthesis and located in Germany, Switzerland, Italy and United-Kingdom.
Only one visit is expected for each patient and parents at the study centre. This visit will last for about 2 hours. It is expected that the entire duration of this study globally will be 6 months.
A total of 5 to 7 patients will be enrolled in the study.REC name
East of Scotland Research Ethics Service REC 2
REC reference
17/ES/0149
Date of REC Opinion
4 Dec 2017
REC opinion
Further Information Favourable Opinion