Study identification

EU PAS number

EUPAS3636

Study ID

34601

Official title and acronym

A non-interventional Post Authorization Safety Study (PASS) to evaluate long-term safety of Orfadin treatment in hypertyrosinemia type 1 (HT-1) patients in standard clinical care (OPAL)

DARWIN EU® study

No

Study countries

Austria
Belgium
Denmark
Finland
France
Germany
Hungary
Ireland
Italy
Netherlands
Norway
Poland
Portugal
Spain
Sweden
United Kingdom

Study description

A non-interventional, non-comparative, multicenter post authorization safety study (PASS) to collect retrospective and prospective longitudinal safety data in hypertyrosinemia type 1 (HT-1) patients on Orfadin treatment in standard clinical care at study entry as well as patients diagnosed and starting Orfadin treatment during the time of the study. All patients included will be followed throughout the study period. Data will be captured from each patient at each follow-up at least once yearly.

Study status

Finalised
Research institutions and networks

Institutions

Department of Pediatric and Adolescent Medicine
Multiple centres: 77 centres are involved in the study

Contact details

Spiekerkötter Ute

Primary lead investigator

Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Data analysis start date

Planned:
Actual:

Date of final study report

Planned:
Actual:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Swedish Orphan Biovitrum AB (publ)
Regulatory

Was the study required by a regulatory body?

No

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

EU RMP category 3 (required)