Study to investigate the effectiveness of emicizumab under real-world conditions in paediatric, adolescent, adult and elderly participants with haemophilia A with and without Factor VIII (FVIII) inhibitors

Not Applicable

• Conditions: Haemophilia A; Haematological Disorders; Hereditary factor VIII deficiency

• Registration Number: ISRCTN58752772
• Lead Sponsor: Roche (Germany)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-02-09
• Last Update Posted: 19 December 2023
• Study Completion: 2025-01-31

Recruitment & Eligibility

• Status: Ongoing
• Sex: Male
• Target Recruitment: 168

Inclusion Criteria

1. Participants of any age with congenital severe haemophilia A with or without FVIII inhibitors
2. Participants undergoing treatment with emicizumab according to Summary of Product Characteristics (SPC) (start of treatment with emicizumab maximum 3 months prior to study entry)
3. Must sign informed consent by the legal representative or participant or both, as required
4. Selection criteria for Cohort A include participants diagnosed with severe congenital haemophilia A (<1% FVIII activity) and no present FVIII inhibitor at the start of emicizumab treatment, patients who completed successful ITI before the start of Emicizumab treatment are eligible.
5. Selection criteria for Cohort B include participants diagnosed with congenital haemophilia A (any severity) with FVIII inhibitor activity at the start of emicizumab treatment or ongoing ITI at the start of emicizumab treatment

Exclusion Criteria

1. Participants having bleeding disorder other than congenital haemophilia A
2. Treatment with emicizumab outside of the SPC at study entry
3. Any contraindication for treatment with emicizumab according to current SPC
4. Current participation in an interventional study

Study & Design

• Study Type: Observational
• Study Design: Not specified