Actively Recruiting
A Multicenter, Randomized, Open-Label Phase III Trial Evaluating NXT007 Versus Emicizumab Prophylaxis in People Aged 12 and Older With Hemophilia A
Led by Hoffmann-La Roche · Updated on 2026-06-08
360
Participants Needed
8
Research Sites
204 weeks
Total Duration
On this page
Sponsors
H
Hoffmann-La Roche
Lead Sponsor
C
Chugai Pharmaceutical
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the effects of NXT007 prophylaxis compared with emicizumab prophylaxis in people aged 12 years and older who have severe or moderate congenital hemophilia A without factor VIII (FVIII) inhibitors, or mild hemophilia A with FVIII inhibitors. The study aims to assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of these treatments in managing hemophilia A. This is a Phase III, randomized, open-label clinical trial sponsored by Hoffmann-La Roche. Participants are randomly assigned to receive either NXT007 prophylaxis or emicizumab prophylaxis during the main study treatment period. NXT007 is given by subcutaneous injection using a combined drug-device product. Emicizumab is administered subcutaneously with a vial and syringe, starting with weekly loading doses of 3 mg/kg for 4 weeks, followed by maintenance doses at varying intervals and amounts depending on prior treatment. After the main treatment period, those on NXT007 may continue its use, and those on emicizumab can switch to NXT007 during an open-label extension phase. Participants will be monitored for at least 7 months during the main treatment period, with assessments focusing on the annualized bleed rate for treated bleeds. Additional evaluations include treatment burden, safety events such as adverse reactions and thromboembolic events, and measurement of drug levels and antibodies. Questionnaires will track the impact on daily activities and treatment satisfaction. The total study duration is approximately 3.5 years, with ongoing safety and efficacy follow-up throughout.
CONDITIONS
Brief Title
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of severe or moderate congenital hemophilia A without factor VIII inhibitors, or mild hemophilia A with chronic factor VIII inhibitors
- Documented factor VIII inhibitor test results within 12 months prior to enrollment
- Documentation of prophylactic and episodic factor VIII treatment and bleeding episodes for at least 6 months before screening
- Agreement to switch to prophylaxis with emicizumab or NXT007 if previously on on-demand treatment
- Age 12 years or older
You will not qualify if you...
- Sensitivity or allergy to study drugs or components that contraindicates participation
- Use or planned use of systemic immunomodulators like interferon or rituximab, except HIV antiretroviral therapy
- Refusal of plasma-derived or blood product transfusion in emergencies
- Planned surgery beyond minor procedures during the study
- History of serious ventricular dysrhythmias or heart disease such as severe left ventricular dysfunction or coronary heart disease
- History or presence of clinically significant abnormal ECG or prior myocardial infarction
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - At least 7 months
Participants receive prophylactic treatment with either NXT007 or Emicizumab to manage Hemophilia A.
Weekly or biweekly visits depending on assigned treatment dosing schedule
Duration - Up to approximately 3 years
Participants who complete the main study treatment period may continue or switch to NXT007 prophylaxis in this extension period.
Visits scheduled at prespecified timepoints during the extension period
Trial Site Locations
Total: 8 locations
1
Center for Inherited Blood Disorders
Orange, California, United States, 92868
Actively Recruiting
2
Innovative Hematology, Inc.
Indianapolis, Indiana, United States, 46260
Actively Recruiting
3
Washington Center for Bleeding Disorders
Seattle, Washington, United States, 98101-3932
Actively Recruiting
4
The Chaim Sheba Medical Center - PPDS
Ramat Gan, Central District, Israel, 5262100
Actively Recruiting
5
Nagoya University Hospital
Nagoya, Aiti, Japan, 466-8560
Actively Recruiting
6
Gunma University Hospital
Maebashi, Gunma, Japan, 371-8511
Actively Recruiting
7
Nara Medical University Hospital
Kashihara-shi, Nara, Japan, 634-8522
Actively Recruiting
8
Ogikubo Hospital
Suginami-Ku, Tokyo, Japan, 167-0035
Actively Recruiting
Research Team
R
Reference Study ID Number: BO45887 https://forpatients.roche.com/
F
Fastest response: use the inquiry form. No email attachments. https://www.gene.com/contact-us/submit-medical-inquiry
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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