NXT007 for Hemophilia A
(ZEBRHA 1 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new treatment called NXT007 for individuals with hemophilia A, a condition that affects blood clotting. The goal is to determine if NXT007 is more effective and safer than the current standard treatment, Factor VIII, in preventing bleeding episodes. Participants will be divided into groups: one will receive NXT007, while the other will continue with standard Factor VIII treatment. The trial seeks individuals with severe or moderate hemophilia A who have been using Factor VIII to manage their condition. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that NXT007 was safe in earlier studies. In one study, a single injection under the skin was well tolerated, with no reports of blood clots. Another study confirmed these results, showing no blocked blood vessels. This suggests that NXT007 is generally safe for humans so far.
For those considering joining this trial, past research indicates that NXT007 has been well tolerated. However, discussing any concerns or questions with the trial team or a doctor remains important.12345Why are researchers excited about this study treatment for hemophilia A?
Researchers are excited about NXT007 for Hemophilia A because it offers a potential new approach compared to the standard treatment, which typically involves regular infusions of Factor VIII. NXT007 is unique as it may provide prophylaxis with a different mechanism, potentially requiring less frequent dosing while still effectively preventing bleeding episodes. This could mean fewer injections and a more convenient treatment schedule for patients, improving their quality of life. Additionally, the possibility of switching patients from the standard Factor VIII treatment to NXT007 in an open-label extension suggests researchers are hopeful about its long-term benefits and safety.
What evidence suggests that this trial's treatments could be effective for hemophilia A?
Studies have shown that NXT007 has promising effects for people with hemophilia A. In this trial, participants will receive either NXT007 prophylaxis or FVIII standard of care (SOC) prophylaxis during the main study treatment period. Earlier research demonstrated that patients who switched to NXT007 maintained stable blood clotting levels similar to normal. Early data also suggest that NXT007 effectively controls bleeding. Additionally, NXT007 is generally well-tolerated, meaning most people's bodies handle it well. Although more research is needed, these findings support the potential effectiveness of NXT007 in treating hemophilia A.12367
Who Is on the Research Team?
Clinical Trials
Principal Investigator
Hoffmann-La Roche
Are You a Good Fit for This Trial?
This trial is for people aged 12 and older with severe or moderate hemophilia A who do not have inhibitors against Factor VIII. Participants must have been on regular Factor VIII preventive treatment, have no history of FVIII inhibitors, and be able to follow contraception guidelines if needed.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Main Study Treatment
Participants receive either NXT007 prophylaxis or FVIII standard of care prophylaxis
Open-Label Extension
Participants in the NXT007 arm continue with NXT007 dosing, and participants in the FVIII arm switch to NXT007
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Human Coagulation Factor VIII
- NXT007
Trial Overview
The study compares a new medication called NXT007 to standard Factor VIII therapy in preventing bleeding episodes in people with hemophilia A. Participants are randomly assigned to receive either NXT007 or Factor VIII as ongoing preventive treatment.
How Is the Trial Designed?
3
Treatment groups
Experimental Treatment
Active Control
After the main study treatment period, participants in the NXT007 arm will be able to continue with NXT007 dosing, and participants in the FVIII arm will be able to switch to NXT007, in the open-label extension period.
Participants randomized to this arm will receive NXT007 prophylaxis for the main study treatment period.
Participants randomized to this arm will receive FVIII standard of care (SOC) prophylaxis for the main study treatment period.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Hoffmann-La Roche
Lead Sponsor
Dr. Levi Garraway
Hoffmann-La Roche
Chief Medical Officer since 2019
MD from the University of Basel
Dr. Thomas Schinecker
Hoffmann-La Roche
Chief Executive Officer since 2023
PhD in Molecular Biology from New York University
Chugai Pharmaceutical
Industry Sponsor
Dr. Osamu Okuda
Chugai Pharmaceutical
Chief Executive Officer since 2020
MD from Kyoto University
Dr. Mariko Y. Momoi
Chugai Pharmaceutical
Chief Medical Officer
MD from Jichi Medical University
Citations
Early data suggest Roche's NXT007 may have the ...
Early data from the NXTAGE study suggest that NXT007 may have the potential to provide haemostatic normalisation in people with haemophilia A ( ...
NXT007 Suggests Favorable Tolerability and Efficacy in ...
NXT007 Suggests Favorable Tolerability and Efficacy in Phase I/II Study for Hemophilia A Following Direct Switch from Emicizumab without Washout ...
NXTAGE: a phase 1/2 study of NXT007 to assess safety ... - PMC
In a study of 30 emicizumab-naive patients with severe hemophilia A, they show persistent levels of hemostasis (>40 IU of FVIII per dL) with the higher NXT007 ...
4.
ashpublications.org
ashpublications.org/blood/article/147/19/2261/566441/NXTAGE-a-phase-1-2-study-of-NXT007-to-assessNXTAGE: a phase 1/2 study of NXT007 to assess safety ...
Participants' plasma NXT007 concentration showed a dose-dependent increase, and predicted FVIII-equivalent activity reached a nonhemophilic ...
A first-in-human study of NXT007, a next-generation ...
Emicizumab is well tolerated and effective in people with congenital hemophilia A regardless of age, severity of disease, or inhibitor status: a scoping ...
A first-in-human study of NXT007, a next-generation ...
A single subcutaneous injection of NXT007 was well tolerated without occurrence of thrombotic events. The long half-life, pharmacological effect, and safety ...
7.
genentech-clinicaltrials.com
genentech-clinicaltrials.com/en/trials/Hemophilia/hemophilia-a/a-study-to-evaluate-the-safety--tolerability--pharmacok-88545.htmlClinical Study/NXT007-01 | ForPatients-Roche
This clinical trial aims to test the safety of NXT007, to understand how the body processes NXT007, and to measure the effects, good or bad, of NXT007 in people ...
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