Study identification

EU PAS number

EUPAS20599

Study ID

35885

Official title and acronym

A Prospective, Observational Registry of Patients with Fabry Disease (AT1001-030)

DARWIN EU® study

No

Study countries

Australia
Austria
Canada
Croatia
Denmark
Finland
Germany
Greece
Hungary
Ireland
Israel
Italy
Norway
Portugal
Spain
Switzerland
United Kingdom
United States

Study description

This is a prospective, multi centre, multinational, observational, safety, effectiveness, and outcomes registry enrolling approximately 450 Fabry disease patients (approximately 250 patients in the migalastat treated group, 100 patients in the ERT-treated group, and 100 patients in the untreated group). All patients will be followed for up to 5 years after enrolment.This is a registry to evaluate the effects of treatment on long-term safety, effectiveness, and quality of life (QOL) in patients with Fabry disease, with a main focus on migalastat, a medicine with a novel mechanism of action. Occurrence of key indicators of safety and effectiveness will be evaluated, such as cardiac, cerebrovascular and renal events, serious adverse events, and overall survival. A comparison of these events will be evaluated over a period of 5 years in migalastat-treated, ERT-treated (with amenable and non-amenable mutations), and untreated patients with Fabry disease who have amenable mutations.

Study status

Ongoing
Research institutions and networks

Institutions

Contact details

Jasmine Rutecki

Primary lead investigator

Study timelines

Date when funding contract was signed

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

Amicus Therapeutics UK Limited
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)