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

Официальное название

A Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Emicizumab Prophylaxis in People With Hemophilia A

Обзор

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.

Вмешательства

• Комбинированный продукт NXT007
  NXT007 will be administered subcutaneously (SC) using an integrated drug-device combination product.
• Препарат Emicizumab
  Emicizumab will be administered subcutaneously (SC) using vial and syringe.

Первичные конечные точки

• Annualized Bleed Rate (ABR) for Treated Bleeds Over the Main Study Treatment Period [Срок оценки: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)]

Вторичные конечные точки (12)

• ABR for All Bleeds Over the Main Study Treatment Period [Срок оценки: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)]
• ABR for Treated Spontaneous Bleeds Over the Main Study Treatment Period [Срок оценки: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)]
• ABR for Treated Joint Bleeds Over the Main Study Treatment Period [Срок оценки: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)]
• Adjusted Mean Treatment Burden Domain Score in Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire - Adult Version at Month 8 [Срок оценки: Month 8]
• ABR for Treated Target Joint Bleeds Over the Main Study Treatment Period [Срок оценки: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)]
• Percentage of Participants with Zero Treated Bleeds Over the Main Study Treatment Period [Срок оценки: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)]
• Number of Injections and Dose per Bleed of Coagulation Factors or Bypassing Agent Administered to Treat a Bleed Over the Main Study Treatment Period [Срок оценки: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)]
• Annualized Injection Rate of FVIII or Bypassing Agent Over the Main Study Treatment Period [Срок оценки: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)]
• Annualized Consumption Rate of FVIII or Bypassing Agent Over the Main Study Treatment Period [Срок оценки: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)]
• Mean Treatment Burden Domain Score in CATCH Questionnaire - Adolescent Version at Month 8 [Срок оценки: Month 8]
• Change From Baseline in Preoccupation Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions) [Срок оценки: 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) [Срок оценки: At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years)]

Критерии участия

Критерии включения

• 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
• For potential participants taking on-demand treatments prior to study entry: agreement to move to a prophylaxis treatment with either emicizumab or NXT007, according to assigned randomization

Критерии исключения

• 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
• 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)
• 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 evidence or clinical history of prior myocardial infarction

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 6 центров

• Center for Inherited Blood Disorders — Orange
• University of Colorado Hemophilia and Thrombosis Center — Aurora
• St Joseph's Children's Hospital of Tampa — Tampa
• Innovative Hematology, Inc. — Indianapolis
• University Of Iowa Hospitals And Clinics — Iowa City
• Washington Center for Bleeding Disorders — Seattle

Испания · 6 центров

• Hospital Universitario Vall d'Hebron - PPDS — Barcelona
• Hospital de la Santa Creu i Sant Pau — Barcelona
• Hospital Universitario La Paz - PPDS — Madrid
• Hospital Regional Universitario de Malaga ? Hospital General — Málaga
• Hospital Universitario Virgen del Rocio - PPDS — Seville
• Hospital Universitari i Politecnic La Fe de Valencia — Valencia

Япония · 4 центра

• Nagoya University Hospital — Nagoya
• Gunma University Hospital — Maebashi
• Nara Medical University Hospital — Kashihara-shi
• Ogikubo Hospital — Suginami-Ku

Италия · 3 центра

• Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico — Milan
• IRCCS Istituto Clinico Humanitas — Rozzano
• Azienda Ospedaliera Universitaria Careggi — Florence

Бельгия · 1 центр

• UZ Leuven — Leuven

Китай · 1 центр

• Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences — Тяньцзинь

Франция · 1 центр

• Hospices Civils de Lyon — Bron

Германия · 1 центр

• Universitätsklinikum Bonn — Bonn

Венгрия · 1 центр

• Debreceni Egyetem Klinikai Kozpont Nagyerdei Campus — Debrecen

Израиль · 1 центр

• The Chaim Sheba Medical Center - PPDS — Ramat Gan

Нидерланды · 1 центр

• Universitair Medisch Centrum Utrecht Cancer Center - PPDS — Utrecht

Новая Зеландия · 1 центр

• Auckland City Hospital — Auckland

South Korea · 1 центр

• Kyungpook National University Hospital — Daegu

Тайвань · 1 центр

• National Taiwan University Hospital — Taipei

Великобритания · 1 центр

• University Hospital of Wales — Cardiff

Идентификаторы

NCT: NCT07416604 · BO45887 · 2025-522435-33-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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