Study identification

PURI

https://redirect.ema.europa.eu/resource/106458

EU PAS number

EUPAS106243

Study ID

106458

Official title and acronym

Prospective Registry-Based Study of the Long-Term Safety of Odevixibat in Patients with Progressive Familial Intrahepatic Cholestasis (PFIC)

DARWIN EU® study

No

Study countries

Austria
Belgium
Brazil
Canada
China
France
Hungary
India
Ireland
Israel
Italy
Netherlands
Poland
Portugal
Slovenia
South Africa
Spain
Sweden
Switzerland
Türkiye
United Arab Emirates
United Kingdom
United States

Study description

A registry-based safety study to examine the long-term, real-world safety profile of odevixibat in patients with PFIC compared to patients not receiving odevixibat. Data for this study will be obtained from the TreatFIC registry. The overall objectives of this registry-based safety study are to evaluate the long-term safety of odevixibat and to evaluate the impact of odevixibat on the occurrence of severe diarrhoea, the impact of odevixibat on the clinical manifestations of fat-soluble vitamin deficiency, the impact of odevixibat on the effectiveness of fat-soluble drugs, the impact of odevixibat on nutritional status and the impact of odevixibat on hepatic function and signs of hepatotoxicity.

Study status

Ongoing
Research institutions and networks

Institutions

Ipsen Pharma
First published:
01/02/2024
Institution
TreatFIC Registry

Contact details

Medical Director

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

Ipsen
Study protocol
Initial protocol

A4250-019 - Protocol Version 24 Mar 2023_ Redacted_PDFA.pdf

English (503.79 KB - PDF)View document
Regulatory

Was the study required by a regulatory body?

Yes

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

EU RMP category 3 (required)