Health Trials

A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A

ConditionHemophilia A
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age12+
SponsorHoffmann-La Roche

About this trial

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared with emicizumab prophylaxis in people age 12 years and older with severe or moderate congenital hemophilia A without factor VIII (FVIII) inhibitors or with hemophilia A of any severity (severe, moderate, and mild) with FVIII inhibitors.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosis of severe (FVIII:C <1 International Unit per decilitre [IU/dL]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A with or without inhibitors against FVIII

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

Documented historical FVIII inhibitor assay results within the 12 months prior to enrollment

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

Disqualifiers

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 HIV

Refusal to accept plasma-derived and/or blood product transfusion support in an emergency scenario

Planned surgery (excluding minor procedures, such as non-molar tooth extraction or incision and drainage) during the study

Trial design

Design model

Parallel

Treatments tested in this trial

  • NXT007

    Combination product

    NXT007 will be administered subcutaneously (SC) using an integrated drug-device combination product.

  • Emicizumab

    Drug

    Emicizumab will be administered subcutaneously (SC) using vial and syringe.

Treatment groups

360 Participants
are divided into 3 treatment groups
Group A: Main Study Treatment Period: NXT007 ProphylaxisExperimental treatment 1 intervention
Group B: Main Study Treatment Period: Emicizumab ProphylaxisActive comparator 1 intervention
Group C: Open-Label Extension Period: NXT007 ProphylaxisExperimental treatment 1 intervention

Trial outcomes

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

Adjusted Mean Treatment Burden Domain Score in Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire - Adult Version at Month 8

Time frame
Month 8

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Hoffmann-La Roche

Lead sponsor

Chugai Pharmaceutical

Collaborator

Source ClinicalTrials.gov
Apply now