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.

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. - ORPH-PAED-01

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

DRKS

Registry ID

DRKS00013645

Enrollment

Registered

2017-12-22

Start date

2017-12-13

Completion date

Unknown

Last updated

2025-04-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

K76.8

Interventions

Group 1: The investigator will explain the study and its objectives during a visit, and will answer any question from the subject and the parents. Review of inclusion/exclusion criteria will be perfor

Eligibility

Sex/Gender

All

Age

5 Years to 13 Years

Inclusion criteria

Inclusion criteria: 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.

Exclusion criteria

Exclusion criteria: 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.

Design outcomes

Primary

Measure	Time frame
To document patient acceptability of the Orphacol® dispersible tablet.	—

Secondary

Measure	Time frame
To document potential difficulties encountered by the parents during preparation and administration of the product.	—

Countries

Germany, Italy, Switzerland, United Kingdom

Contacts

Public ContactMarie Fey

Laboratoires CTRS

mfey@ctrs.fr+33 6 40 54 36 15

Outcome results

None listed

Source: DRKS (via WHO ICTRP) · Data processed: Feb 4, 2026