Actively Recruiting
A Multicenter, Randomized, Open-Label Phase III Trial Evaluating NXT007 Prophylaxis Versus Factor VIII Prophylaxis in People With Hemophilia A Without Inhibitors
Led by Hoffmann-La Roche · Updated on 2026-06-08
126
Participants Needed
2
Research Sites
208 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 efficacy, safety, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared with Factor VIII (FVIII) prophylaxis in people aged 12 years and older with severe or moderate congenital hemophilia A without inhibitors. This Phase III randomized study aims to assess how NXT007, a newer treatment, compares to the current standard FVIII prophylaxis in preventing bleeding episodes in this population. Participants will be randomly assigned to receive either NXT007 prophylaxis, administered subcutaneously using a combined drug-device product, or standard FVIII prophylaxis given according to local guidelines. After the main treatment period of 6 months, those on NXT007 may continue with this dosing, while participants initially on FVIII can switch to NXT007 during an open-label extension phase. During the study, participants will be regularly monitored for bleeding rates, treatment burden, drug levels, and safety outcomes including adverse events and immune responses. Questionnaires will assess the impact of treatments on daily life and activities. The main measure is the annualized bleed rate over 6 months, with follow-up continuing for approximately 3.5 years to evaluate long-term effects and safety.
CONDITIONS
Brief Title
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of severe (FVIII:C <1 IU/dL) or moderate (FVIII:C between 651 IU/dL and 6545 IU/dL) congenital hemophilia A without inhibitors
- No documented inhibitor (<0.6 BU/mL), FVIII half-life 656 hours, or FVIII recovery >66% in the last 3 years prior to screening
- Documented negative test for FVIII inhibitor (<0.6 BU/mL) within 12 months prior to enrollment
- Documentation of prophylactic and episodic FVIII treatment and bleeding episodes for at least 6 months before screening
- Agreement to follow contraception requirements for those with childbearing potential
You will not qualify if you...
- Sensitivity or allergy to study drugs, devices, or procedures that contraindicate participation
- Use of systemic immunomodulators (e.g., interferon or rituximab) at enrollment or planned during study, except HIV antiretroviral therapy
- Planned surgery during the study, except minor procedures like non-molar tooth extraction or incision and drainage
- History or presence of significant abnormal ECG or prior heart attack
- Refusal to accept plasma-derived or blood product transfusions in emergency situations
- History of ventricular dysrhythmias or risk factors such as severe left ventricular dysfunction, hypertrophy, or coronary heart disease
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 - 6 months
Participants receive prophylaxis treatment with either NXT007 administered subcutaneously or Factor VIII prophylaxis according to local standard of care.
Visits as needed for treatment and assessments during the 6-month main study treatment period
Duration - Up to approximately 3 years
Participants who completed the main treatment period can continue or switch to NXT007 prophylaxis in an open-label extension to further assess safety and efficacy.
Visits at prespecified timepoints for ongoing assessments and dosing
Trial Site Locations
Total: 2 locations
1
Nara Medical University Hospital
Kashihara-shi, Nara, Japan, 634-8522
Actively Recruiting
2
Tokyo Medical University Hospital
Shinjuku-Ku, Tokyo, Japan, 160-0023
Actively Recruiting
Research Team
R
Reference Study ID Number: WO45886 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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