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Not yet recruitingNCT07838194ZEBRHA 3Updated Oct 19, 2026

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

A Phase 3 interventional study of NXT007 in Hemophilia A, sponsored by Hoffmann-La Roche. Not yet recruiting. Open to participants aged 0 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-10-19.

Sponsored by Hoffmann-La Roche · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
50
Allocation
Not applicable
Ages
0 Years to 17 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Hemophilia A

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Keywords

  • Factor VIII
  • Inhibitors
  • Bypassing agents
03

In context

Hemophilia A

844 studies on the registry are indexed under Hemophilia A; 138 are open to participants now.

This study's planned enrollment of 50 is above the median of 30 across 499 interventional studies indexed under Hemophilia A.

Browse Hemophilia A studies →

Lead sponsor

Hoffmann-La Roche is the lead sponsor of 2,101 studies on the registry; 87 are open to participants now.

Of its 330 completed or terminated interventional studies of FDA-regulated products, 248 (75%) have results posted.

Browse Hoffmann-La Roche studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
0 Years to 17 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Age \<12 years at the time of signing 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

Exclusion criteria

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 antiretroviral therapy to treat
  • 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
Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
50 participants (estimated)

Study arms

  • Experimental
    NXT007 Prophylaxis

    Drug: NXT007

Interventions

  • DrugNXT007

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

    Also known as: Zemocimig, RO7589655, RG6512

06

What researchers measure

Primary outcomes

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

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

Secondary outcomes

  1. ABR for All Bleeds Over the Main Study Treatment Period

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

  2. ABR for Treated Spontaneous Bleeds Over the Main Study Treatment Period

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

  3. ABR for Treated Joint Bleeds Over the Main Study Treatment Period

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

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

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

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

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

  6. Number of Injections and Dose per Bleed of Factor VIII or Bypassing Agent Administered to Treat a Bleed Over the Main Study Treatment Period

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

  7. Annualized Injection Rate of FVIII or Bypassing Agent Over the Main Study Treatment Period

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

  8. Annualized Consumption Rate of FVIII or Bypassing Agent Over the Main Study Treatment Period

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

  9. 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

    Time frame: Baseline and Month 8

  10. Change from Baseline in the Preoccupation Domain Score on the CATCH 2.0 Caregiver Questionnaire at Month 8

    Time frame: Baseline and Month 8

  11. 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

    Time frame: From Baseline until Study Completion (approximately 4 years)

  12. Incidence and Severity of Thromboembolic Events and Thrombotic Microangiopathy

    Time frame: From Baseline until Study Completion (approximately 4 years)

  13. Incidence and Severity of Injection-Site Reactions

    Time frame: From Baseline until Study Completion (approximately 4 years)

  14. Incidence of Adverse Events Leading to Study Drug Discontinuation

    Time frame: From Baseline until Study Completion (approximately 4 years)

  15. Incidence of Severe Hypersensitivity, Anaphylaxis, or Anaphylactoid Reactions

    Time frame: From Baseline until Study Completion (approximately 4 years)

  16. Plasma Concentrations of NXT007

    Time frame: At prespecified timepoints from Baseline until Study Completion (approximately 4 years)

  17. Percentage of Participants With Anti-Drug Antibodies (ADAs) Against NXT007 at Baseline and During the Study

    Time frame: At prespecified timepoints from Baseline until Study Completion (approximately 4 years)

  18. Percentage of Participants With Neutralizing ADAs Against NXT007

    Time frame: At prespecified timepoints from Baseline until Study Completion (approximately 4 years)

07

Study locations

No study locations are listed for this record.

08

References and documents

Individual participant data

Plan to share: 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

09

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Start is estimated for Nov 2026; primary completion for Sep 2028, 22 months later.

Of 89 industry-sponsored phase 3 interventional studies under Hemophilia A, half reached primary completion within 32 months of starting (the median), counting those still running; a quarter within 18 and three quarters within 56. This study's planned 22 months is below that.

Counting every sponsor, half of 91 studies reached it within 33 months; a quarter within 20 and three quarters within 56.

How these figures are worked out, and which studies are left out of them.

Registered
Sep 17, 2026
First posted
Sep 24, 2026
StartEst.
Nov 30, 2026
Primary completionEst.
Sep 30, 2028
CompletionEst.
Sep 30, 2031
Last update
Oct 19, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Minor edits
Nothing that changes what the study is or who can join. Edited: verification date
1 update, last Oct 19, 2026
Show all 1 update
  1. Oct 19, 2026
    Minor edits only
    + 1 other change: verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT07838194
Lead sponsor
Hoffmann-La Roche
Collaborators
Chugai Pharmaceutical
Responsible party
Sponsor
First posted
Sep 24, 2026
Start date
Nov 30, 2026 (estimated)
Primary completion
Sep 30, 2028 (estimated)
Completion
Sep 30, 2031 (estimated)
Last update
Oct 19, 2026

Study contacts

Reference Study ID Number: BO45888 https://forpatients.roche.com/ No attachments to email below.
Contact
global-roche-genentech-trials@gene.com
888-662-6728 (U.S. Only)
Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry
Contact
Clinical Trials
study director · Hoffmann-La Roche

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is not yet recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.

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Discussion

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