A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A

Phase 3RecruitingNCT07416526

Hoffmann-La Roche126 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

126

Conditions

Hemophilia A

Interventions

NXT007COMBINATION_PRODUCT

NXT007 will be administered subcutaneously (SC) using an integrated drug-device combination product.

Human Coagulation Factor VIIIDRUG

Factor 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.

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: * 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) Exclusion Criteria: * 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 * Refusal to accept plasma-derived and/or blood product transfusion support in an emergency scenario * History of ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease (e.g., severe left ventricular systolic dysfunction, left ventricular hypertrophy), coronary heart disease (symptomatic or with ischemia demonstrated by diagnostic testing)

Locations (2)

Nara Medical University Hospital

Kashihara-shi, Nara, Japan

RECRUITING

Tokyo Medical University Hospital

Shinjuku-Ku, Tokyo, Japan

RECRUITING

Outcomes

Primary Outcomes

Annualized Bleed Rate (ABR) for Treated Bleeds Over the Main Study Treatment Period

Time frame: 6 months

Secondary Outcomes

ABR for All Bleeds Over the Main Study Treatment Period

Time frame: 6 months

ABR for Treated Spontaneous Bleeds Over the Main Study Treatment Period

Time frame: 6 months

ABR for Treated Joint Bleeds Over the Main Study Treatment Period

Time frame: 6 months

Adjusted Mean Treatment Burden Domain Score in Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire - Adult Version at Month 7

Time frame: Month 7

ABR for Treated Target Joint Bleeds Over the Main Study Treatment Period

Time frame: 6 months

Percentage of Participants with Zero Treated Bleeds Over the Main Study Treatment Period

Time frame: 6 months

Number of Injections and Dose per Bleed of Coagulation Factors Administered to Treat a Bleed Over the Main Study Treatment Period

Time frame: 6 months

Annualized FVIII Injection Rate Over the Main Study Treatment Period

Time frame: 6 months

Annualized FVIII Consumption Rate Over the Main Study Treatment Period

Time frame: 6 months

Central Contacts

Reference Study ID Number: WO45886 https://forpatients.roche.com/

CONTACT

888-662-6728 (U.S. Only)global-roche-genentech-trials@gene.com

Data from ClinicalTrials.gov

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