Study identification

PURI

https://redirect.ema.europa.eu/resource/40418

EU PAS number

EUPAS40417

Study ID

40418

Official title and acronym

Prospective observational study to monitor and assess the safety of Onpattro® patisiran-LNP in a real-world cohort of hATTR amyloidosis patients

DARWIN EU® study

No

Study countries

Brazil
Bulgaria
Denmark
France
Germany
Israel
Italy
Netherlands
Portugal
Spain
Taiwan
United Kingdom
United States

Study description

The purpose of this observational study is to assess the safety of patisiran-lipid nanoparticle (LNP) (Onpattro®) in real-world clinical practice by creating and monitoring a longitudinal cohort of hereditary transthyretin-mediated (hATTR) amyloidosis patients, including both patients treated with patisiran-LNP and comparator patients (treated with a competitor and untreated), following local standard of care.
The primary objective of the study is to characterise the safety of patisiran-LNP under real-world conditions, including determining and comparing the incidence of protocol-specified events of interest in patients exposed to patisiran-LNP.

Study status

Ongoing
Research institutions and networks

Institutions

Multiple centres: 40 centres are involved in the study

Contact details

Emily Brouwer

Primary lead investigator
Study timelines

Date when funding contract was signed

Actual:

Study start date

Actual:

Date of interim report, if expected

Planned:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Alnylam
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)