NXT007 for Hemophilia A

Recruiting at 19 trial locations

Overseen By Reference Study ID Number: WP44714 https://forpatients.roche.com/

Age: < 65

Sex: Male

Trial Phase: Phase 1 & 2

Sponsor: Hoffmann-La Roche

Must be taking: Recombinant activated factor VII

Must not be taking: QT-prolonging medications

Disqualifiers: Bleeding disorders, Thromboembolic disease, Diabetes, Malignancies, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Variable duration based on dose escalation

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores NXT007, a potential new treatment for hemophilia A, a condition where blood doesn't clot properly. The study aims to assess the safety and effectiveness of NXT007 and its behavior in the body, including absorption, processing, and utilization. It consists of two parts: one for adult and adolescent males and another for young boys, all with severe or moderate hemophilia A. Participants should have a history of hemophilia A with frequent bleeding episodes that affect daily life and must be willing to adhere to the study's schedule and guidelines. As a Phase 1, Phase 2 trial, this research seeks to understand how NXT007 works in people and measure its effectiveness in an initial, smaller group, offering participants the chance to contribute to groundbreaking advancements in hemophilia A treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are using certain investigational drugs or medications that prolong the QT interval, you may need to stop those before participating.

Is there any evidence suggesting that NXT007 is likely to be safe for humans?

Research has shown that NXT007 is generally safe for humans. Studies found that increasing the dose did not lead to more side effects. Importantly, no serious issues like blood clots or problems at the injection site were reported. These findings suggest that NXT007 is safe for people with hemophilia A. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for hemophilia A?

NXT007 is unique because it targets Hemophilia A with a novel approach compared to traditional treatments like factor VIII replacement therapies. Most current treatments involve regular injections of clotting factor VIII to prevent bleeding episodes. However, NXT007 works differently by using a bispecific antibody that can bridge factors IXa and X, effectively mimicking the function of factor VIII without needing its frequent administration. This could mean fewer injections and potentially better management of bleeding episodes, which is why researchers are excited about its potential.

What evidence suggests that NXT007 might be an effective treatment for hemophilia A?

Research has shown that NXT007 might be promising for people with hemophilia A. Early data suggest it could help blood clot properly, a process known as hemostatic normalization. This potential benefit appears in people with or without factor VIII inhibitors, proteins that can sometimes interfere with treatment. Initial findings from the NXTAGE study showed positive results with different doses. In this trial, participants will receive varying doses of NXT007 to evaluate its effectiveness. Overall, these early studies suggest that NXT007 could be an effective treatment option for managing hemophilia A. ^{1 2 4 5 6}

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 males with severe or moderate Hemophilia A, weighing over 40 kg. They must have a history of bleeding episodes and be willing to follow the study's procedures. Participants can't join if they have poor kidney, liver, or blood function.

Inclusion Criteria

My kidney function is within the required range.

I weigh at least 40 kilograms.

Historic local FVIII inhibitor test results must be available during screening to confirm any previous inhibitor history and current status

Exclusion Criteria

My close family members had heart or brain blood vessel diseases early.

I have a history of significant heart issues noted on an ECG.

I have not taken any experimental drugs or had gene therapy recently.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple ascending doses of NXT007 to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy

Variable duration based on dose escalation

Follow-up

Participants are monitored for safety and effectiveness after treatment, including vital signs and ECG abnormalities

Up to 7.5 years

What Are the Treatments Tested in This Trial?

Interventions

• NXT007

Trial Overview The trial tests NXT007 in adults and teens with Hemophilia A to see how safe it is and how well it works at different doses. It's an early-stage (Phase I/II) global study where everyone gets the drug; there's no comparison group.

How Is the Trial Designed?

Group I: Part 2: Cohort A - NXT007 Experimental Treatment 1 Intervention

Group II: Part 1: Cohort 5 - NXT007 Dose Level 5 (High) Experimental Treatment 1 Intervention

Group III: Part 1: Cohort 4 - NXT007 Dose Level 4 Experimental Treatment 1 Intervention

Group IV: Part 1: Cohort 3 - NXT007 Dose Level 3 Experimental Treatment 1 Intervention

Group V: Part 1: Cohort 2 - NXT007 Dose Level 2 Experimental Treatment 1 Intervention

Group VI: Part 1: Cohort 1 - NXT007 Dose Level 1 (Low) Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Hoffmann-La Roche

Lead Sponsor

Trials

2,482

Recruited

1,107,000+

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Avastin, Herceptin, Rituxan, Accu-Chek

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

Published Research Related to This Trial

N8-GP, an extended half-life recombinant factor VIII, effectively maintains FVIII levels above 1 IU/dL in both adults and children with severe hemophilia A during prophylaxis, with dosing schedules of every 4 days for adults/adolescents and twice weekly for children.

The pharmacokinetic study involving 69 patients showed that N8-GP's half-life is positively correlated with von Willebrand factor levels in adults/adolescents, suggesting that this relationship may influence dosing strategies for optimal treatment outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fixed doses of N8-GP prophylaxis maintain moderate-to-mild factor VIII levels in the majority of patients with severe hemophilia A. Chowdary, P., Carcao, M., Holme, PA., et al.[2023]

N9-GP, a new recombinant factor IX treatment for hemophilia B, demonstrated a significantly prolonged half-life of 93 hours, which is five times longer than previous FIX products, suggesting it could reduce the frequency of dosing.

The treatment was well-tolerated in a trial with 16 patients, with no development of inhibitors and only one case of transient hypersensitivity, indicating a favorable safety profile for N9-GP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Negrier, C., Knobe, K., Tiede, A., et al.[2022]

Turoctocog alfa (NovoEight®) is a third-generation recombinant factor VIII concentrate that has shown excellent efficacy and safety in both prophylactic and on-demand treatment for patients with severe hemophilia A, based on clinical trials.

This treatment not only effectively prevents bleeding episodes but also demonstrates high hemostatic activity during surgical procedures, with ongoing post-marketing studies expected to provide further insights into its long-term benefits and the incidence of inhibitors.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An evaluation of the safety and efficacy of turoctocog alfa for hemophilia A. Boban, A., Hermans, C.[2021]

Blocking tissue factor pathway inhibitor (TFPI) may offer a new treatment strategy for hemophilia by enhancing clot formation, as shown in both in vitro and rabbit hemophilia models.

The monoclonal antibody mAb 2021, which targets the Kunitz-type protease inhibitor (KPI) 2 domain of TFPI, has demonstrated promising pro-hemostatic effects and is currently undergoing preclinical and clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hemostatic properties of a TFPI antibody. Petersen, LC.[2019]

A 9-month-old boy with hemophilia B developed inhibitors and an allergic reaction to factor IX after treatment, leading to a switch in therapy.

Prophylactic treatment with pdFVIIa/factor X successfully prevented life-threatening bleeding for over 2 years, suggesting it may be an effective option for hemophilia B patients with inhibitors and allergic reactions to FIX concentrates.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prophylaxis Using a Mixture of Plasma-Derived Activated Factor VII and Factor X (pdFVIIa/FX) in a Patient with Hemophilia B Complicated by Inhibitors and Allergy to Factor IX Concentrates: A Case Report. Uchida, E., Komori, K., Kurata, T., et al.[2021]

Citations

1. roche.com roche.com/media/releases/med-cor-2025-06-23

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 (without factor ...

2. sciencedirect.com sciencedirect.com/science/article/pii/S153878362500399X

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 ...

3. chugai-pharm.co.jp chugai-pharm.co.jp/english/news/detail/20250623113001_1165.html

Phase I/II Study in Hemophilia A Suggests NXT007 May ... Phase I/II Study in Hemophilia A Suggests NXT007 May Have the Potential to Provide Hemostatic Normalization. Results from The NXTAGE study part ...

4. genentech-clinicaltrials.com genentech-clinicaltrials.com/en/trials/Hemophilia/hemophilia-a/a-study-to-evaluate-the-safety--tolerability--pharmacok-88545.html

Clinical Study/NXT007-01 | ForPatients-Roche A clinical trial to look at how safely and how well NXT007 works at different doses in people with hemophilia A – and to understand how the body processes.

5. gene.com gene.com/media/press-releases/15088/2025-11-03/genentech-to-present-new-data-from-its-b

Genentech: Press Releases | Monday, Nov 3, 2025 NXT007: Positive Phase I/II results, including new data from a global study in people with hemophilia A with and without factor VIII inhibitors, ...

6. bleeding.org bleeding.org/news/trial-data-for-roche-investigational-subq-hemophilia-therapy-presented-at-isth

Read About New Hemophilia Trial Data on Investigational ... NXT007 is an investigational bispecific antibody therapy for people with hemophilia A who do not have factor VIII inhibitors.