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

NCT07416526 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: WO458862025-522434-32-00
• Study Type: interventional
• Target: 126
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

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

  6 months

Secondary Outcomes (22)

• ABR for All Bleeds Over the Main Study Treatment Period

  6 months

• ABR for Treated Spontaneous Bleeds Over the Main Study Treatment Period

  6 months

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

  6 months

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

  Month 7

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

  6 months

Study Arms (3)

Main Study Treatment Period: NXT007 Prophylaxis

EXPERIMENTAL

Participants randomized to this arm will receive NXT007 prophylaxis for the main study treatment period.

Combination Product: NXT007

Main Study Treatment Period: FVIII SOC Prophylaxis

ACTIVE COMPARATOR

Participants randomized to this arm will receive FVIII standard of care (SOC) prophylaxis for the main study treatment period.

Drug: Human Coagulation Factor VIII

Open-Label Extension Period: NXT007 Prophylaxis

EXPERIMENTAL

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.

Combination Product: NXT007

Interventions

NXT007 COMBINATION_PRODUCT

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

Also known as: Zemocimig, RO7589655, RG6512

Main Study Treatment Period: NXT007 Prophylaxis; Open-Label Extension Period: NXT007 Prophylaxis

Human Coagulation Factor VIII DRUG

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.

Main Study Treatment Period: FVIII SOC Prophylaxis

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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)

Sponsors & Collaborators

• Hoffmann-La Roche: lead
• Chugai Pharmaceutical: collaborator

Study Sites (2)

Nara Medical University Hospital

Kashihara-shi, Nara, 634-8522, Japan

RECRUITING

Tokyo Medical University Hospital

Shinjuku-Ku, Tokyo, 160-0023, Japan

RECRUITING

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Central Study Contacts

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

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 11, 2026
• First Posted: February 18, 2026
• Study Start: May 9, 2026
• Primary Completion (Estimated): September 23, 2027
• Study Completion (Estimated): September 23, 2031
• Last Updated: May 7, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

JP (2)