Orphacol® patient surveillance database (Orphabase)

Study summary

No information provided.

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

THERAVIA

Hannover Medical School (MHH)

University Medical Centre Hamburg-Eppendorf

Germany

Heidelberg University Hospital

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

Study timelines

Date when funding contract was signed

Planned:17/11/2006

Actual:17/11/2006

Study start date

Planned:20/11/2006

Actual:20/11/2006

Date of final study report

Planned:13/02/2030

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)