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

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/48526
• 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 use in children with haemophilia A during routine clinical care, among pediatric patients enrolled in the European Pediatric Network for Haemophilia Management (PedNet) Registry. PedNet Registry is the largest registry in the world for pediatric patients with haemophilia, currently, 20 countries with approximately 32 treatment centers participate. The registry includes all age groups and severities (FVIII <25%), which includes 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 will be reported. In addition to safety, effectiveness of emicizumab will be evaluated by the annual bleeding rate, as reported in the PedNet Registry. The following criteria describe the population eligible for this study, which will be 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 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 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 will extract data indicated in this protocol, perform the statistical analysis, and provide results to the Marketing Authorization Holder (MAH) who will generate an annual PASS report and/or include the results in the PBRER.
• Study status:
  Ongoing

Research institution and networks

Institutions

F. Hoffmann-La Roche

Study timelines

Date when funding contract was signed

Actual: 24/03/2019

Study start date

Planned: 30/11/2019

Actual: 03/12/2019

Date of final study report

Planned: 30/09/2025

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

F. Hoffmann-La Roche, Ltd.

Study protocol

Initial protocol

Prot MO40685 (PEDNET) emicizumab v1_Redacted

Updated protocol

MO40685 (PEDNET)-Protocol v2_Redacted

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)

Other study registration identification numbers and links

MO40685, NCT02979119

PedNet Registry