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Recruiting NCT07416526

A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A

Phase III Interventional Hemophilia A

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: NXT007, Human Coagulation Factor VIII.
Who it may be relevant to
Registry conditions: Hemophilia A. Basic parameters: from 12 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Germany, Hungary, Italy, Japan +5
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Factor VIII Prophylaxis in People With Hemophilia A Without Inhibitors

Overview

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of NXT007 prophylaxis compared with Factor VIII (FVIII) prophylaxis in participants with severe or moderate congenital hemophilia A without inhibitors. The study will include people aged ≥12 years old with severe or moderate congenital hemophilia A without inhibitors on previous FVIII prophylaxis treatment.

Interventions

  • Combination product NXT007
    NXT007 will be administered subcutaneously (SC) using an integrated drug-device combination product.
  • Drug Human Coagulation Factor VIII
    Factor VIII (FVIII) prophylaxis standard of care (SOC) will be administered at the dose and frequency as stated in the local labels and per local country practice.

Primary outcome measures

  • Annualized Bleed Rate (ABR) for Treated Bleeds Over the Main Study Treatment Period [Time frame: 6 months]
Secondary outcome measures (12)
  • ABR for All Bleeds Over the Main Study Treatment Period [Time frame: 6 months]
  • ABR for Treated Spontaneous Bleeds Over the Main Study Treatment Period [Time frame: 6 months]
  • ABR for Treated Joint Bleeds Over the Main Study Treatment Period [Time frame: 6 months]
  • Adjusted Mean Treatment Burden Domain Score in Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire - Adult Version at Month 7 [Time frame: Month 7]
  • ABR for Treated Target Joint Bleeds Over the Main Study Treatment Period [Time frame: 6 months]
  • Percentage of Participants with Zero Treated Bleeds Over the Main Study Treatment Period [Time frame: 6 months]
  • Number of Injections and Dose per Bleed of Coagulation Factors Administered to Treat a Bleed Over the Main Study Treatment Period [Time frame: 6 months]
  • Annualized FVIII Injection Rate Over the Main Study Treatment Period [Time frame: 6 months]
  • Annualized FVIII Consumption Rate Over the Main Study Treatment Period [Time frame: 6 months]
  • Mean Treatment Burden Domain Score in CATCH Questionnaire - Adolescent Version at Month 7 [Time frame: Month 7]
  • Change From Baseline in Preoccupation Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions) [Time frame: At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years)]
  • Change From Baseline in Social Activity Impact Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions) [Time frame: At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years)]

Eligibility criteria

Inclusion criteria

  • Diagnosis of severe (FVIII:C <1 IU/dL \[International Unit per decilitre\]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A without inhibitors against FVIII
  • No documented inhibitor (i.e., <0.6 BU/mL \[Bethesda unit per millilitre\]), FVIII half-life ≥6 hours, or FVIII recovery >66% in the last 3 years prior to screening
  • Documented historical negative test for FVIII inhibitor (i.e., <0.6 BU/mL) within 12 months prior to enrollment
  • Documentation of the details of prophylactic and episodic FVIII treatment and of the number and type of bleeding episodes for at least the last 6 months prior to screening
  • Agreement to adhere to the contraception requirements (for potential participants with childbearing potential)

Exclusion criteria

  • 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 anti-retroviral therapy to treat HIV
  • Planned surgery (excluding minor procedures such as non-molar tooth extraction, incision and drainage) during the study
  • History or presence of an abnormal ECG that is deemed clinically significant, (e.g., complete left bundle branch block, second- or third- degree atrioventricular heart block) or ECG evidence or clinical history of prior myocardial infarction
  • Refusal to accept plasma-derived and/or blood product transfusion support in an emergency scenario
  • History of ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease (e.g., severe left ventricular systolic dysfunction, left ventricular hypertrophy), coronary heart disease (symptomatic or with ischemia demonstrated by diagnostic testing)

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Treatment

Study locations

Spain · 4 centers
  • Hospital Universitario Vall d'Hebron - PPDS — Barcelona
  • Hospital Universitario La Paz - PPDS — Madrid
  • Hospital Regional Universitario de Malaga ? Hospital General — Málaga
  • Hospital Universitari i Politecnic La Fe de Valencia — Valencia
Italy · 3 centers
  • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico — Milan
  • IRCCS Istituto Clinico Humanitas — Rozzano
  • Azienda Ospedaliera Universitaria Careggi — Florence
United States · 2 centers
  • University of Colorado Hemophilia and Thrombosis Center — Aurora
  • University of Iowa Hospitals and Clinics — Iowa City
Japan · 2 centers
  • Nara Medical University Hospital — Kashihara-shi
  • Tokyo Medical University Hospital — Shinjuku-Ku
South Korea · 2 centers
  • Kyung Hee University Hospital at Gangdong — Gangdong-gu
  • Chonnam National University Hwasun Hospital — Hwasun-gun
Taiwan · 2 centers
  • Taichung Veterans General Hospital — Taichung
  • Tri-Service General Hospital — Taipei
Germany · 1 center
  • Universitätsklinikum Bonn — Bonn
Hungary · 1 center
  • Eszak-Pesti Centrumkorhaz - Honvedkorhaz;Diabetologiai Szakrendeles — Budapest
Netherlands · 1 center
  • Universitair Medisch Centrum Utrecht — Utrecht
United Kingdom · 1 center
  • University Hospital of Wales — Cardiff

Identifiers

NCT: NCT07416526 · WO45886 · 2025-522434-32-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗