NL-OMON49869Not yet recruitingPhase 3
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 - XTEND-Kids
Trial Snapshot
- Phase
- Phase 3
- Status
- Not yet recruiting
- Sponsor
- Genzyme Europe BV
- Enrollment
- 3
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional
Eligibility Criteria
- Ages
- 2 to 11 (—)
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
- •- 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.
Investigators
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