LONG-TERM NON-INTERVENTIONAL SAFETY STUDY OF EMICIZUMAB TREATMENT IN PATIENTS WITH MODERATE HAEMOPHILIA A AND SEVERE BLEEDING PHENOTYPE (STUDY BO44691, PASS)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/1000000174
• EU PAS number:
  EUPAS1000000174
• Study ID:
  1000000174
• Official title and acronym:
  LONG-TERM NON-INTERVENTIONAL SAFETY STUDY OF EMICIZUMAB TREATMENT IN PATIENTS WITH MODERATE HAEMOPHILIA A AND SEVERE BLEEDING PHENOTYPE (STUDY BO44691, PASS)
• DARWIN EU® study:
  No
• Study countries:
  Canada

  France

  Netherlands

  United Kingdom

  United States
• Study description:
  This non-interventional (NI) post-authorization safety study (PASS) is a multi-country, registry-based, longitudinal cohort study based on secondary use of data collected for patients of all ages with moderate congenital Haemophilia A (Factor VIII [FVIII] 1%-5%) without FVIII inhibitors and with severe bleeding phenotype and who are treated with emicizumab. The aim of the study is to evaluate the long-term safety profile of emicizumab in patients who are exposed to emicizumab in real-world settings, with a specific focus on thromboembolic (TE) events.
• Study status:
  Planned

Research institutions and networks

Institutions

IQVIA

United Kingdom

Study timelines

Date when funding contract was signed

Planned: 20/10/2024

Study start date

Planned: 01/05/2025

Data analysis start date

Planned: 01/10/2030

Date of final study report

Planned: 01/05/2031

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

F. Hoffmann-La Roche, Ltd.

Study protocol

Initial protocol

Prot BO44691 emicizumab v2, Published Output-1_Redacted.pdf

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)