A Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Emicizumab Prophylaxis in People With Hemophilia A
Hoffmann-La Roche
Summary
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared with emicizumab prophylaxis in people age 12 years and older with severe or moderate congenital hemophilia A without factor VIII (FVIII) inhibitors or with hemophilia A of any severity (severe, moderate, and mild) with FVIII inhibitors.
Eligibility
- Age range
- 12+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Diagnosis of severe (FVIII:C \<1 International Unit per decilitre \[IU/dL\]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A with or without inhibitors against FVIII * Diagnosis of mild (FVIII:C between \>5 IU/dL and \<40 IU/dL) congenital hemophilia A with chronic FVIII inhibitors, defined as documented FVIII inhibitor ( ≥0.6 BU/mL or ≥1.0 BU/mL only for laboratories with a historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) and chronic reduction of endogenous baseline FVIII:C to \<5 IU/dL for ≥12 months * Documented historica…
Interventions
- Combination ProductNXT007
NXT007 will be administered subcutaneously (SC) using an integrated drug-device combination product.
- DrugEmicizumab
Emicizumab will be administered subcutaneously (SC) using vial and syringe.
Locations (6)
- Center for Inherited Blood DisordersOrange, California
- Innovative Hematology, Inc.Indianapolis, Indiana
- Washington Center for Bleeding DisordersSeattle, Washington
- Gunma University HospitalMaebashi, Gunma
- Nara Medical University HospitalKashihara-shi, Nara
- Ogikubo HospitalSuginami-Ku, Tokyo