Study identification

EU PAS number

EUPAS37229

Study ID

49962

Official title and acronym

Orphacol® patient surveillance database (Orphabase)

DARWIN EU® study

No

Study countries

France
Germany
Italy
Spain
Switzerland
United Kingdom

Study description

Multi-centre, multi-national, retrospective and prospective observational study to collect patients’ clinical information (treatment, safety and efficacy data) of patients receiving Orphacol® for the treatment of inborn errors in primary bile acid synthesis due to 3β-hydroxy-Δ5-C27-steroid oxidoreductase (3β-HSD) deficiency or Δ4-3-oxosteroid 5β-reductase (Δ4-3-oxoR) deficiency.

Study status

Ongoing
Research institutions and networks

Institutions

Hannover Medical School (MHH)
First published:
01/02/2024
Institution Educational Institution Hospital/Clinic/Other health care facility
Kremlin Bicêtre Hospital, France France, Hôpital La Timone, Marseille France, Hôpital Beaujon, Paris France, Westfällische Wilhelms-Universität Münster Germany, Universitätsklinikum Heidelberg Germany, University Hospital Hamburg-Eppendorf, Hamburg Germany, Medizinische Hochschule Hannover Germany, Ospedale Infantile Regina Margherita, Torino Italy, Fondazione I.R.C.C.S. Policlinico S.Matteo, Pavia Italy, Azienda Ospedaliera Padova Italy

Contact details

Theravia Medical Affairs Direction

Primary lead investigator

Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Theravia
Regulatory

Was the study required by a regulatory body?

Yes

Is the study required by a Risk Management Plan (RMP)?

EU RMP category 2 (specific obligation of marketing authorisation)

Regulatory procedure number

Initial marketing authorisation procedure (EU/1/13/870/001-006)