A Phase 3 open-label, multicenter study of the safety, efficacy, and pharmacokinetics of intravenous recombinant coagulation Factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; BIVV001) in previously treated pediatric patients <12 years of age with severe hemophilia A

Phase 3

• Conditions: 10064477; hemophilia A; 10005330

• Registration Number: NL-OMON49869
• Lead Sponsor: Genzyme Europe BV

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Pending
• Sex: Not specified
• Target Recruitment: 3

Inclusion Criteria

- Participant must be younger than 12 years of age, at the time of signing the
informed consent
- Severe hemophilia A defined as <1 IU/dL (<1%) endogenous FVIII as documented
either by central laboratory testing at Screening or in historical medical
records from a clinical laboratory demonstrating <1% FVIII coagulant activity
(FVIII:C) or a documented genotype known to produce severe hemophilia A.
- Previous treatment for hemophilia A (prophylaxis or on-demand) with any
recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150
EDs for patients aged 6-11 years and above 50 EDs for patients aged below 6
years
- Weight above or equal to 10 kg.

Exclusion Criteria

- History of hypersensitivity or anaphylaxis associated with any FVIII product.
- History of a positive inhibitor (to FVIII) test defined as >=0.6 BU/mL, or any
value greater than or equal to the lower sensitivity cut-off for laboratories
with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical
signs or symptoms of decreased response to FVIII administrations. Family
history of inhibitors will not exclude the participant.
- Positive inhibitor test result, defined as >=0.6 BU/mL at Screening.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Occurrence of inhibitor development</p><br>