Study identification

PURI

https://redirect.ema.europa.eu/resource/1000000533

EU PAS number

EUPAS1000000533

Study ID

1000000533

Official title and acronym

Characterization of Participants treated with Ultomiris and Long term safety outcomes: an IPIG registry based study

DARWIN EU® study

No

Study countries

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

Study description

This is a noninterventional cohort study utilizing data from the IPIG PNH Registry.
Adult and pediatric participants with PNH with a detected proportion of PNH cells (PNH clone) of at least 1% at registry enrollment, who have provided written informed consent, and who are not participating in an interventional clinical trial specific to PNH, are eligible for participation in the IPIG PNH Registry.

The primary study objectives are to characterize the safety of Ultomiris in participants in PNH and the incidence of targeted clinical outcomes among participants with PNH. Secondary objectives include describing the demographic and clinical profile at treatment initiation for Ultomiris treated participants with PNH and assessing Ultomiris treatment patterns among participants with PNH.

Study status

Planned
Research institutions and networks

Institutions

Alexion Europe SAS
NA

Networks

NA

Contact details

Alexion Europe SAS

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:

Study start date

Planned:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 
Study protocol
Updated protocol

ALX-PNH-501 PASS Protocol - Final - 14 June 2023.pdf

English (529.74 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)