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 3ß-Hydroxy-?5-C27- steroid oxidoreductase deficiency or ?4-3-Oxosteroid-5ß-reductase deficiency.
- Conditions
- K76.8Other specified diseases of liver
- Registration Number
- DRKS00013645
- Lead Sponsor
- aboratoires CTRS
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 4
Males and females between 5 and 13 years old included.
Subject currently treated by Orphacol® commercial capsules or other primary bile acids for inborn errors in primary bile acid synthesis due to 3ß-Hydroxy-?5-C27-steroid oxidoreductase deficiency or ?4-3-Oxosteroid-5ß-reductase deficiency.
History of significant or ongoing psychiatric disorder (e.g. severe depression, schizophrenia, psychosis or manic depression).
Use of sedatives or antidepressants (including St. John’s Wort).
Sensory disorders affecting the mouth or local anaesthetics into the mouth within 48 hours before the study.
Previous surgery or radiotherapy for head and neck cancer.
Subject who has participated in another study and received any other investigational agent within 30 days prior to start of clinical dosing.
Subject and parents/legal representatives unable to provide written informed consent.
Failure to satisfy the investigator or eligibility to participate for any other reason.
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To document patient acceptability of the Orphacol® dispersible tablet.
- Secondary Outcome Measures
Name Time Method To document potential difficulties encountered by the parents during preparation and administration of the product.