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 FVIII inhibitors. The study will include people aged ≥12 years old who have been on FVIII prophylaxis treatment prior to study entry.
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
Sensitivity to any of the study investigations, or components thereof, or drug or other allergy that, in the opinion of the investigator, contraindicates participation in the study
Use of systemic immunomodulators (e.g., interferon or rituximab) at the time of enrollment or planned use during the study, except for anti-retroviral therapy to treat HIV
Planned surgery (excluding minor procedures such as non-molar tooth extraction, incision and drainage) during the study
History or presence of an abnormal ECG that is deemed clinically significant, (e.g., complete left bundle branch block, second- or third- degree atrioventricular heart block) or ECG evidence or clinical history of prior myocardial infarction
Parallel
NXT007
Combination productNXT007 will be administered subcutaneously (SC) using an integrated drug-device combination product.
Human Coagulation Factor VIII
DrugFactor VIII (FVIII) prophylaxis standard of care (SOC) will be administered at the dose and frequency as stated in the local labels and per local country practice.
Annualized Bleed Rate (ABR) for Treated Bleeds Over the Main Study Treatment Period
ABR for All Bleeds Over the Main Study Treatment Period
ABR for Treated Spontaneous Bleeds Over the Main Study Treatment Period
ABR for Treated Joint Bleeds Over the Main Study Treatment Period
Adjusted Mean Treatment Burden Domain Score in Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire - Adult Version at Month 7
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Hoffmann-La Roche
Lead sponsor
Chugai Pharmaceutical
Collaborator
