Study identification

PURI

https://redirect.ema.europa.eu/resource/1000000457

EU PAS number

EUPAS1000000457

Study ID

1000000457

Official title and acronym

Post-authorization safety study of iptacopan in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) using data from the non-interventional IPIG PNH Registry

DARWIN EU® study

No

Study countries

Canada
China
France
Germany
Italy
Japan
Spain
Switzerland
United Kingdom
United States

Study description

This post-authorization safety study (PASS) is an observational single-arm descriptive cohort study based on the secondary use of data collected on iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the IPIG PNH registry.
The primary objective of the study is to describe the risk of infections due to encapsulated bacteria in patients with PNH treated with iptacopan. The secondary objectives are to describe the risk of other safety outcomes in patients with PNH treated with iptacopan, and to describe the course of pregnancies exposed to iptacopan and pregnancy outcomes. In addition, the study aims to describe in an observational setting the proportion of iptacopan-treated PNH patients not compliant with mandatory and recommended vaccinations against encapsulated bacteria.

Study status

Ongoing
Research institutions and networks

Institutions

Contact details

Novartis Clinical Disclosure Officer

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:
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

Novartis Pharma AG
Study protocol
Initial protocol

02.01.0201 Protocol_Redacted.pdf

English (3.23 MB - 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)