Clinical Trial of NXT007 for the Prophylactic Treatment of Hemophilia A in Infants and Children

NCT07838194 | Not Yet Recruiting Phase 3 FDA Drug

• 2 other identifiers: BO458882026-526103-29-00
• Study Type: interventional
• Target: 50
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of NXT007 prophylaxis in pediatric patients aged 0 to 11 years with severe or moderate congenital hemophilia A without factor VIII inhibitors or congenital hemophilia A of any severity (severe, moderate, and mild) with inhibitors (emicizumab-naïve and treated). Patients with hemophilia A aged ≥12 to \<18 years old with a body weight of \<40 kilograms (kg) are also able to participate.

Conditions

Hemophilia A

Keywords

Factor VIII; Inhibitors; Bypassing agents

Outcome Measures

Primary Outcomes (1)

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

  From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

Secondary Outcomes (18)

• ABR for All Bleeds Over the Main Study Treatment Period

  From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

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

  From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

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

  From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

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

  From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

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

  From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

Study Arms (1)

NXT007 Prophylaxis

EXPERIMENTAL

Drug: NXT007

Interventions

NXT007 DRUG

NXT007 will be administered subcutaneously (SC) according to the schedule in the protocol.

Also known as: Zemocimig, RO7589655, RG6512

NXT007 Prophylaxis

Eligibility Criteria

• Age: 0 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age \<12 years at the time of signing Informed Consent Form; or age ≥12 and \<18 years with body weight \<40 kg
• Diagnosis of severe (Factor VIII coagulation protein activity \[FVIII:C\] \<1 International Unit per decilitre \[IU/dL\]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A (HA) with or without inhibitors against factor VIII (FVIII)
• For potential participants with moderate HA without inhibitors, fulfillment of at least one of the following criteria: Current prophylaxis with long-term prophylaxis intention due to a severe bleed phenotype; For those not treated with prophylaxis: at least one traumatic joint or critical muscle bleed in the last 6 months or ≥2 spontaneous bleeds/year or 5 bleeds/year, including traumatic bleeds; Signs of joint degeneration compatible with hemophilic arthropathy (e.g., subchondral bone changes or the presence of synovitis) as assessed by any imaging assessment (e.g., ultrasound, MRI, radiograph); Bleeding at a critical site (e.g., intracranial).
• Diagnosis of mild (FVIII:C between \>5 IU/dL and \<40 IU/dL) congenital hemophilia A with chronic FVIII inhibitors, defined as documented FVIII inhibitor (≥0.6 BU/mL or ≥1.0 BU/mL only for laboratories with a historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) and chronic reduction of endogenous baseline FVIII:C to \<5 IU/dL for ≥12 months
• Documentation of the details of prophylactic and episodic FVIII treatment, bypassing agent (BPA) treatment, emicizumab prophylaxis treatment, and the number and type of bleeding episodes for at least the last 6 months prior to screening if appropriate
• For potential participants taking on-demand treatments prior to study entry: agreement to move to a prophylaxis treatment with NXT007
• Adequate renal, hepatic, and hematologic function, as defined in the protocol

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 antiretroviral therapy to treat HIV
• Refusal to accept plasma-derived and/or blood product transfusion support in an emergency scenario
• History or conditions, other than HA, which may indicate hypo- or hypercoagulopathy risk
• Planned surgery (excluding minor procedures, such as non-molar tooth extraction or incision and drainage) during the study
• 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)
• Any serious medical condition or abnormality in clinical laboratory tests that precludes an individual's safe participation in and completion of the study

Sponsors & Collaborators

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

Related Links

• Please use this form to submit your questions for a faster response: https://www.gene.com/contact-us/submit-medical-inquiry. Do not include or attach any medical records when emailing or completing the form. A nurse will respond within 24 business hours.

MeSH Terms

Conditions

Hemophilia A

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

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Central Study Contacts

Reference Study ID Number: BO45888 https://forpatients.roche.com/ No attachments to email below.

CONTACT

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

Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry

CONTACT

Study Design

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

Study Record Dates

• First Submitted: September 17, 2026
• First Posted: September 24, 2026
• Study Start (Estimated): November 30, 2026
• Primary Completion (Estimated): September 30, 2028
• Study Completion (Estimated): September 30, 2031
• Last Updated: September 24, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will share