Study identification

EU PAS number

EUPAS31954

Study ID

48526

Official title and acronym

Emicizumab Use in Pediatric Patients in the Real World: an Analysis of the PedNet Registry

DARWIN EU® study

No

Study countries

Austria
Belgium
Canada
Czechia
Denmark
Finland
France
Germany
Greece
Ireland
Israel
Italy
Netherlands
Norway
Portugal
Spain
Sweden
Switzerland
United Kingdom

Study description

The main aim of this non-interventional, secondary data use, post-authorization safety study (PASS) is to assess safety of emicizumab prophylaxis in real-world conditions, among pediatric patients with hemophilia A enrolled in the European Pediatric Network for Haemophilia Management (PedNet) Registry.

PedNet Registry is the largest registry in the world for pediatric patients with hemophilia. Currently, 19 countries with approximately 34 treatment centers participate. The registry includes all age groups and severities (Factor VIII <25%), which provides substantial coverage and an adequate representation of the pediatric patient population.

The primary safety events of interest in this study are thromboembolic events (TEs), thrombotic microangiopathy (TMA), and anaphylaxis. However, all safety events collected in the PedNet Registry are reported.
In addition to safety, effectiveness of emicizumab is evaluated by the annual bleeding rate, as reported in the PedNet Registry. The following criteria describe the population eligible for this study, which is a subset of the overall population participating in the PedNet Registry.

Inclusion criteria for inclusion in the PedNet Registry:
- Diagnosis of haemophilia A
- Factor VIII (FVIII) activity <25%
- Treated in one of the participating centers.
Additional inclusion for emicizumab-specific analysis:
- Received prophylactic treatment with emicizumab.

Exclusion criteria for the PedNet Registry:
- Referral to a participating center after development of FVIII inhibitors
- Informed consent for participation in the PedNet Registry not obtained.
Exclusion criteria for emicizumab-specific analysis:
- Inherited or acquired bleeding disorder other than haemophilia A.

The PedNet Registry extracts and analyzes data according to the protocol and provides the Marketing Authorization Holder (MAH) with annual emicizumab-specific reports.

Study status

Finalised
Research institutions and networks

Institutions

Contact details

Letizia Polito

Primary lead investigator

Study timelines

Date when funding contract was signed

Actual:

Study start date

Planned:
Actual:

Date of final study report

Planned:
Actual:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

F. Hoffmann-La Roche, Ltd.
Regulatory

Was the study required by a regulatory body?

No

Is the study required by a Risk Management Plan (RMP)?

EU RMP category 3 (required)