A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants 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 Factor VIII Prophylaxis in People With Hemophilia A Without Inhibitors

Обзор

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.

Вмешательства

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

Первичные конечные точки

• Annualized Bleed Rate (ABR) for Treated Bleeds Over the Main Study Treatment Period [Срок оценки: 6 months]

Вторичные конечные точки (12)

• ABR for All Bleeds Over the Main Study Treatment Period [Срок оценки: 6 months]
• ABR for Treated Spontaneous Bleeds Over the Main Study Treatment Period [Срок оценки: 6 months]
• ABR for Treated Joint Bleeds Over the Main Study Treatment Period [Срок оценки: 6 months]
• Adjusted Mean Treatment Burden Domain Score in Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire - Adult Version at Month 7 [Срок оценки: Month 7]
• ABR for Treated Target Joint Bleeds Over the Main Study Treatment Period [Срок оценки: 6 months]
• Percentage of Participants with Zero Treated Bleeds Over the Main Study Treatment Period [Срок оценки: 6 months]
• Number of Injections and Dose per Bleed of Coagulation Factors Administered to Treat a Bleed Over the Main Study Treatment Period [Срок оценки: 6 months]
• Annualized FVIII Injection Rate Over the Main Study Treatment Period [Срок оценки: 6 months]
• Annualized FVIII Consumption Rate Over the Main Study Treatment Period [Срок оценки: 6 months]
• Mean Treatment Burden Domain Score in CATCH Questionnaire - Adolescent Version at Month 7 [Срок оценки: Month 7]
• 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 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)

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 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)

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

Испания · 4 центра

• 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

Италия · 3 центра

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

США · 2 центра

• University of Colorado Hemophilia and Thrombosis Center — Aurora
• University of Iowa Hospitals and Clinics — Iowa City

Япония · 2 центра

• Nara Medical University Hospital — Kashihara-shi
• Tokyo Medical University Hospital — Shinjuku-Ku

South Korea · 2 центра

• Kyung Hee University Hospital at Gangdong — Gangdong-gu
• Chonnam National University Hwasun Hospital — Hwasun-gun

Тайвань · 2 центра

• Taichung Veterans General Hospital — Taichung
• Tri-Service General Hospital — Taipei

Германия · 1 центр

• Universitätsklinikum Bonn — Bonn

Венгрия · 1 центр

• Eszak-Pesti Centrumkorhaz - Honvedkorhaz;Diabetologiai Szakrendeles — Budapest

Нидерланды · 1 центр

• Universitair Medisch Centrum Utrecht — Utrecht

Великобритания · 1 центр

• University Hospital of Wales — Cardiff

Идентификаторы

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

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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