A Phase I/II Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hoffmann-La Roche
Summary
WP44714 is a Phase I/II, open-label, non-randomized, global, multicenter trial consisting of two parts: * Part 1 is a multiple-ascending dose (MAD) study in adult and adolescent male participants with severe or moderate hemophilia A with or without factor VIII (FVIII) inhibitors. * Part 2 is a multiple-dose study in pediatric male participants with severe or moderate hemophilia A with or without FVIII inhibitors. The overall aim of the study is to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and efficacy of NXT007.
Eligibility
- Age range
- 2–59 years
- Sex
- Male
- Healthy volunteers
- No
Inclusion Criteria: * Diagnosis of severe (Factor VIII \[FVIII\] coagulant activity \<1 IU/dL) or moderate (FVIII coagulant activity ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A with or without inhibitors against FVIII * Participants with FVIII inhibitors: participants using recombinant activated factor VII (rFVIIa) or willing to switch to rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds, trauma, or procedures * Historic local FVIII inhibitor test results being available during screening to confirm any previous inhibitor history and current status * Participants wh…
Interventions
- DrugNXT007
Participants will receive NXT007 administered subcutaneously (SC), 2 loading doses once every two weeks (Q2W) followed by once every 4 weeks (Q4W) maintenance doses based on the schedule.
Locations (14)
- UC Davis Cancer CenterSacramento, California
- Georgetown Uni Medical CenterWashington D.C., District of Columbia
- Indiana Hemophilia & Thrombosis centerIndianapolis, Indiana
- University of Iowa Hospitals and Clnics Dept of PediatricsIowa City, Iowa
- British Columbia Children's HospitalVancouver, British Columbia
- Hamilton Health Sciences CorporationHamilton, Ontario