Hemophilia APhase 3ZEBRHA 3NCT07838194
Clinical Trial of NXT007 for the Prophylactic Treatment of Hemophilia A in Infants and Children
A Phase 3 study testing NXT007 in people with Hemophilia A, run by Hoffmann-La Roche.
Trial 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… Read more about this study
- Who is it for?
- Ages 0 to 17 years
- Conditions the study is for
- Hemophilia A
- What is being tested?
- NXT007
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- 30 Nov 2026to 30 Sep 2031
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
Reference Study ID Number: BO45888 https://forpatients.roche.com/ No attachments to email below. contact
Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry contact
About this study
Official title: A Multicenter, Open-Label, Single-Arm, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of NXT007 Prophylaxis in Pediatric Patients With Hemophilia A
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
- Being tested
- NXT007
- Keywords
- Factor VIII, Inhibitors, Bypassing agents
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NXT007Drug
Also called Zemocimig, RO7589655, RG6512
NXT007 will be administered subcutaneously (SC) according to the schedule in the protocol.
| Study group | Role | What they receive |
|---|---|---|
| NXT007 Prophylaxis | Experimental | Not described Receives: Drug: NXT007 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Bleed Rate (ABR) for Treated Bleeds Over the Main Study Treatment Period
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)
Number of Injections and Dose per Bleed of Factor VIII or Bypassing Agent Administered to Treat a Bleed Over the Main Study Treatment Period
Measured: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)
Annualized Injection Rate of FVIII or Bypassing Agent Over the Main Study Treatment Period
Measured: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)
Annualized Consumption Rate of FVIII or Bypassing Agent Over the Main Study Treatment Period
Measured: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)
Change from Baseline in the Treatment Burden Domain Score on the Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) 2.0 Caregiver Questionnaire at Month 8
Measured: Baseline and Month 8
Change from Baseline in the Preoccupation Domain Score on the CATCH 2.0 Caregiver Questionnaire at Month 8
Measured: Baseline and Month 8
Incidence and Severity of Adverse Events, With Severity Determined According To National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0 Grading Scale
Measured: From Baseline until Study Completion (approximately 4 years)
Incidence and Severity of Thromboembolic Events and Thrombotic Microangiopathy
Measured: From Baseline until Study Completion (approximately 4 years)
Incidence and Severity of Injection-Site Reactions
Measured: From Baseline until Study Completion (approximately 4 years)
Incidence of Adverse Events Leading to Study Drug Discontinuation
Measured: From Baseline until Study Completion (approximately 4 years)
Incidence of Severe Hypersensitivity, Anaphylaxis, or Anaphylactoid Reactions
Measured: From Baseline until Study Completion (approximately 4 years)
Plasma Concentrations of NXT007
Measured: At prespecified timepoints from Baseline until Study Completion (approximately 4 years)
Percentage of Participants With Anti-Drug Antibodies (ADAs) Against NXT007 at Baseline and During the Study
Measured: At prespecified timepoints from Baseline until Study Completion (approximately 4 years)
Percentage of Participants With Neutralizing ADAs Against NXT007
Measured: At prespecified timepoints from Baseline until Study Completion (approximately 4 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2026
- Study start30 Nov 2026 (estimated)
- Primary completion30 Sep 2028 (estimated)
- Study completion30 Sep 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Chugai Pharmaceutical
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07838194
- Sponsor's protocol ID
- BO45888
- Other IDs
- 2026-526103-29-00
- Acronym
- ZEBRHA 3
- Status last verified
- Oct 2026
- First submitted
- 17 Sep 2026
- Last updated
- 19 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing