A Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Factor VIII Prophylaxis in People With Hemophilia A Without Inhibitors
Status: Recruiting
Location: See all (19) locations...
Intervention Type: Drug, Combination product
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of NXT007 prophylaxis compared with Factor VIII (FVIII) prophylaxis in participants with severe or moderate congenital hemophilia A without inhibitors. The study will include people aged ≥12 years old with severe or moderate congenital hemophilia A without inhibitors on previous FVIII prophylaxis treatment.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Healthy Volunteers: f
View:
• Diagnosis of severe (FVIII:C \<1 IU/dL \[International Unit per decilitre\]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A without inhibitors against FVIII
• No documented inhibitor (i.e., \<0.6 BU/mL \[Bethesda unit per millilitre\]), FVIII half-life ≥6 hours, or FVIII recovery \>66% in the last 3 years prior to screening
• Documented historical negative test for FVIII inhibitor (i.e., \<0.6 BU/mL) within 12 months prior to enrollment
• Documentation of the details of prophylactic and episodic FVIII treatment and of the number and type of bleeding episodes for at least the last 6 months prior to screening
• Agreement to adhere to the contraception requirements (for potential participants with childbearing potential)
Locations
United States
Colorado
University of Colorado Hemophilia and Thrombosis Center
After the main study treatment period, participants in the NXT007 arm will be able to continue with NXT007 dosing, and participants in the FVIII arm will be able to switch to NXT007, in the open-label extension period.