Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study

Официальное название

Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study (NuPOWER)

Обзор

Recombinant factor VIII for the prevention of bleeding in patients with severe haemophilia A undergoing major surgery while receiving emicizumab prophylaxis

Подробное описание

Patients with severe haemophilia A receiving emicizumab will often need concomitant FVIII to provide haemostatic cover during major surgery. This prospective, open-label, uncontrolled, single-arm, multinational, multicentre study aims to evaluate the overall perioperative haemostatic efficacy of Nuwiq, a recombinant factor VIII, in combination with emicizumab prophylaxis in male patients over 12 with severe haemophilia A undergoing major surgery.

Вмешательства

• Препарат Nuwiq
  Nuwiq is a purified B-domain-deleted FVIII glycoprotein synthesised by a genetically engineered human embryonic kidney cell line (HEK 293F).

Первичные конечные точки

• Overall haemostatic efficacy [Срок оценки: During surgery and until completion of wound healing (e.g. removal of sutures, cessation of drainage, etc.) as defined by the investigator, during the study duration of 30 ± 3 days after the day of surgical procedure]

Вторичные конечные точки (9)

• Intraoperative haemostatic efficacy [Срок оценки: During surgery: From first skin incision to last suture]
• Postoperative haemostatic efficacy [Срок оценки: From end of surgery until completion of wound healing (e.g. removal of sutures, cessation of drainage, etc.) as defined by the investigator, during the study duration of 30 ± 3 days after the day of surgical procedure]
• Blood product transfusion levels [Срок оценки: From day of surgery until completion of wound healing (e.g. removal of sutures, cessation of drainage, etc.) as defined by the investigator, during the study duration of 30 ± 3 days after the day of surgical procedure]
• FVIII plasma levels [Срок оценки: ≤30 minutes before and 15-30 minutes after Nuwiq injection]
• Thrombin generation [Срок оценки: ≤30 minutes before and 15-30 minutes after Nuwiq injection]
• Perioperative haemostatic efficacy per World Federation of Hemophilia (WFH) criteria [Срок оценки: Perioperative]
• Thrombotic events [Срок оценки: From start of first Nuwiq injection to 30 ± 3 days following the surgical procedure or until day of discharge, whichever is later]
• FVIII inhibitor formation [Срок оценки: From start of first Nuwiq injection to 30 ± 3 days following the surgical procedure or until day of discharge, whichever is later]
• Adverse events [Срок оценки: From start of first Nuwiq injection to 30 ± 3 days following the surgical procedure or until day of discharge, whichever is later]

Критерии участия

Критерии включения

• Severe haemophilia A (FVIII activity \[FVIII:C\] <1%) according to medical history
• Male patients at least 12 years of age
• Previous treatment with any FVIII product(s) for at least 150 exposure days
• On regular prophylaxis with emicizumab for at least 1 month prior to a scheduled major elective surgery requiring FVIII treatment
• Freely given written informed consent of the patient, or parent/legal representative where applicable, obtained in accordance with local regulations

Критерии исключения

• Coagulation disorder other than haemophilia A
• Present or past FVIII inhibitor (≥0.6 Bethesda units \[BU\]/mL) according to medical history
• Severe liver or kidney disease (alanine aminotransferase \[ALT\] and/or aspartate aminotransferase \[AST\] levels >5 times the upper limit of normal; or creatinine >120 μmol/L)
• Known hypersensitivity to Nuwiq's active substance or its excipients (sucrose, sodium chloride, calcium chloride dihydrate, arginine hydrochloride, sodium citrate dihydrate, poloxamer 188)
• Already had surgery in this study
• Current participation in another interventional clinical trial
• Treatment with any investigational medicinal product (IMP) within 30 days prior to screening visit

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Германия · 3 центра

• Vivantes Klinikum im Friedrichshein (KFH) — Berlin
• Gerinnungszentrum Rhein-Ruhr — Duisburg
• Universitätsklinikum Hamburg Eppendorf (UKE) — Hamburg

Италия · 3 центра

• Azienda Ospedaliera Pugliese Ciaccio — Catanzaro
• Azienda Ospedaliero Universitaria Careggi - Centro Emofilia — Florence
• Centro Trombosi e Malattie Emorragiche, ITCCS Humanitas Research Hospital — Milan

Франция · 2 центра

• CHU de Nantes Hôtel-Dieu — Nantes
• CHRU de Tours — Tours

Индия · 2 центра

• Christian Medical College Vellore — Vellore
• St. John's Medical College Hospital — Bengaluru

Испания · 2 центра

• Hospital Universitario La Paz — Madrid
• Hospital Universitario Virgen del Rocio — Seville

США · 1 центр

• UT Southwestern Medical Center — Dallas

Хорватия · 1 центр

• University Hospital Centre Zagreb — Zagreb

Финляндия · 1 центр

• Helsinki University Hospital — Helsinki

North Macedonia · 1 центр

• Centre for Haemopilia, Institute for transfusion medicine of Republic of North Macedonia — Skopje

Сербия · 1 центр

• Clinical Center for Serbia Belgrade — Belgrade

Великобритания · 1 центр

• St. James's University Hospital — Leeds

Публикации

• Srivastava A, Kanny A, Langer F, Kubicek-Hofmann C, Alvarez Roman MT, Nunez Vazquez R, Boban A, Dejanova-Ilijevska V, Miljic P, Garcia J, Halimeh S, Drillaud N, Valentin JB, Mancuso ME, Castaman G, Santoro RC, Lehtinen AE, Abraham A, Hashimoto M, Knaub S. NuPOWER (Nuwiq for Perioperative management Of patients With haemophilia A on Emicizumab Regular prophylaxis): protocol for an open-label, singl PMID 41151952

Идентификаторы

NCT: NCT05935358 · GENA-22

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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