Study identification

EU PAS number

EUPAS1000000876

Study ID

1000000876

Official title and acronym

An Observational Cohort Study to Assess Long-Term Safety of Danicopan Add-on Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data

DARWIN EU® study

No

Study countries

Argentina
Australia
Austria
Belgium
Canada
China
Denmark
Finland
France
Germany
Greece
Italy
Japan
Korea, Republic of
Netherlands
Norway
Spain
Sweden
Switzerland
Taiwan
Türkiye
United Kingdom
United States

Study description

This is a non-interventional cohort study that utilizes data from the International PNH Interest Group (IPIG) PNH Registry to assess the long-term safety of danicopan as add-on therapy to ravulizumab or eculizumab for the treatment of adult patients with PNH who have residual hemolytic anemia.

The study utilizes both primary and secondary data from the IPIG PNH
Core Registry including the danicopan (Voydeya) Silo registry. The study population will consist of participants treated with danicopan as add-on therapy compared with participants treated with Soliris/Ultomiris monotherapy.

Study status

Planned
Research institutions and networks

Institutions

Alexion Europe SANS

Contact details

Ami Patel

Primary lead investigator

Study timelines

Date when funding contract was signed

Actual:

Study start date

Planned:

Data analysis start date

Planned:

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

Alexion Pharmaceuticals, Inc.
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)