Natascha Marquardt, Florian Langer, Katharina Holstein, María Teresa Álvarez Román, Ramiro Núñez Vázquez, Predrag Miljić, Nicolas Drillaud, Laurent Ardillon, Anna-Elina Lehtinen, Rita Carlotta Santoro, Mariasanta Napolitano, Sergio Siragusa, Gillian Gidley, Martina Jansen, Sigurd Knaub, Johannes Oldenburg
{"title":"设计一项国际、IV期、开放标签的研究，研究对象是正在接受手术的A型血友病妇女/女孩（NuDIMENSION）。","authors":"Natascha Marquardt, Florian Langer, Katharina Holstein, María Teresa Álvarez Román, Ramiro Núñez Vázquez, Predrag Miljić, Nicolas Drillaud, Laurent Ardillon, Anna-Elina Lehtinen, Rita Carlotta Santoro, Mariasanta Napolitano, Sergio Siragusa, Gillian Gidley, Martina Jansen, Sigurd Knaub, Johannes Oldenburg","doi":"10.1177/20406207241300040","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Although hemophilia A mainly affects males, carriers (defined as females with hemophilia A, as well as symptomatic or asymptomatic hemophilia A carriers) are at risk of excessive bleeding, particularly during trauma or during surgical procedures. Clinical trials have focused on male patients with severe disease, and data for females are limited. Improved, evidence-based treatment guidelines for management of hemophilia A carriers are required.</p><p><strong>Objectives and design: </strong>The NuDIMENSION study is a phase IV, prospective, open-label, single-arm study that will evaluate the perioperative efficacy and safety of simoctocog alfa (Nuwiq<sup>®</sup>), a recombinant factor VIII (FVIII), in women/girls with hemophilia A undergoing major surgery. The study will be conducted at approximately 15 centers worldwide. Women/girls aged ⩾12 years, with mild or moderate hemophilia A (residual FVIII activity (FVIII:C) ⩾1% to <40%) and with no current/past FVIII inhibitors are eligible. All patients must be scheduled to undergo a major surgical procedure during which simoctocog alfa will be administered.</p><p><strong>Methods and analysis: </strong>The primary endpoint is overall perioperative hemostatic efficacy (\"success\" or \"failure\") of simoctocog alfa. Hemostatic efficacy will be assessed at the end of surgery and at the end of the postoperative period (i.e., completion of wound healing), with overall adjudication by an Independent Data Monitoring Committee. Safety endpoints will include the incidences of thrombotic events and FVIII inhibitor development. The aim is to recruit 28 patients to achieve 26 evaluable surgeries.</p><p><strong>Ethics: </strong>Ethical approval will be received from institutional review boards/independent ethics committees, and the study will be conducted in compliance with the Declaration of Helsinki.</p><p><strong>Discussion: </strong>Data from NuDIMENSION will generate much-needed evidence on surgical management of women/girls with hemophilia A, which will help to enable the development of treatment guidelines specific for such patients.</p><p><strong>Trial registration: </strong>CT EU 2022-502061-17-00; NCT05936580.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241300040"},"PeriodicalIF":3.4000,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11610030/pdf/","citationCount":"0","resultStr":"{\"title\":\"Design of an international, phase IV, open-label study of simoctocog alfa in women/girls with hemophilia A undergoing surgery (NuDIMENSION).\",\"authors\":\"Natascha Marquardt, Florian Langer, Katharina Holstein, María Teresa Álvarez Román, Ramiro Núñez Vázquez, Predrag Miljić, Nicolas Drillaud, Laurent Ardillon, Anna-Elina Lehtinen, Rita Carlotta Santoro, Mariasanta Napolitano, Sergio Siragusa, Gillian Gidley, Martina Jansen, Sigurd Knaub, Johannes Oldenburg\",\"doi\":\"10.1177/20406207241300040\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Although hemophilia A mainly affects males, carriers (defined as females with hemophilia A, as well as symptomatic or asymptomatic hemophilia A carriers) are at risk of excessive bleeding, particularly during trauma or during surgical procedures. Clinical trials have focused on male patients with severe disease, and data for females are limited. Improved, evidence-based treatment guidelines for management of hemophilia A carriers are required.</p><p><strong>Objectives and design: </strong>The NuDIMENSION study is a phase IV, prospective, open-label, single-arm study that will evaluate the perioperative efficacy and safety of simoctocog alfa (Nuwiq<sup>®</sup>), a recombinant factor VIII (FVIII), in women/girls with hemophilia A undergoing major surgery. The study will be conducted at approximately 15 centers worldwide. Women/girls aged ⩾12 years, with mild or moderate hemophilia A (residual FVIII activity (FVIII:C) ⩾1% to <40%) and with no current/past FVIII inhibitors are eligible. All patients must be scheduled to undergo a major surgical procedure during which simoctocog alfa will be administered.</p><p><strong>Methods and analysis: </strong>The primary endpoint is overall perioperative hemostatic efficacy (\\\"success\\\" or \\\"failure\\\") of simoctocog alfa. Hemostatic efficacy will be assessed at the end of surgery and at the end of the postoperative period (i.e., completion of wound healing), with overall adjudication by an Independent Data Monitoring Committee. Safety endpoints will include the incidences of thrombotic events and FVIII inhibitor development. The aim is to recruit 28 patients to achieve 26 evaluable surgeries.</p><p><strong>Ethics: </strong>Ethical approval will be received from institutional review boards/independent ethics committees, and the study will be conducted in compliance with the Declaration of Helsinki.</p><p><strong>Discussion: </strong>Data from NuDIMENSION will generate much-needed evidence on surgical management of women/girls with hemophilia A, which will help to enable the development of treatment guidelines specific for such patients.</p><p><strong>Trial registration: </strong>CT EU 2022-502061-17-00; NCT05936580.</p>\",\"PeriodicalId\":23048,\"journal\":{\"name\":\"Therapeutic Advances in Hematology\",\"volume\":\"15 \",\"pages\":\"20406207241300040\"},\"PeriodicalIF\":3.4000,\"publicationDate\":\"2024-12-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11610030/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Therapeutic Advances in Hematology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1177/20406207241300040\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Therapeutic Advances in Hematology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/20406207241300040","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/1/1 0:00:00","PubModel":"eCollection","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
背景：虽然A型血友病主要影响男性，但携带者（定义为患有A型血友病的女性，以及有症状或无症状的A型血友病携带者）有出血过多的风险，特别是在创伤或手术过程中。临床试验主要集中在患有严重疾病的男性患者身上，而女性患者的数据有限。需要改进以证据为基础的A型血友病携带者治疗指南。目的和设计：NuDIMENSION研究是一项IV期、前瞻性、开放标签、单组研究，将评估重组因子VIII (FVIII) simotocog alfa （Nuwiq®）在接受大手术的a型血友病妇女/女孩中的围手术期疗效和安全性。这项研究将在全球大约15个中心进行。年龄小于或等于12岁的妇女/女孩，患有轻度或中度血友病A(剩余FVIII活性（FVIII:C）小于或等于1%的方法和分析：主要终点是simotocog α的总体围手术期止血疗效（“成功”或“失败”）。止血效果将在手术结束和术后（即伤口愈合完成）结束时进行评估，并由独立数据监测委员会进行总体裁决。安全性终点将包括血栓事件的发生率和FVIII抑制剂的发展。目标是招募28名患者进行26次可评估的手术。伦理：将收到机构审查委员会/独立伦理委员会的伦理批准，研究将按照赫尔辛基宣言进行。讨论：NuDIMENSION的数据将为患有A型血友病的妇女/女孩的手术治疗提供急需的证据，这将有助于制定针对此类患者的治疗指南。试验注册：CT EU 2022-502061-17-00；NCT05936580。
Design of an international, phase IV, open-label study of simoctocog alfa in women/girls with hemophilia A undergoing surgery (NuDIMENSION).
Background: Although hemophilia A mainly affects males, carriers (defined as females with hemophilia A, as well as symptomatic or asymptomatic hemophilia A carriers) are at risk of excessive bleeding, particularly during trauma or during surgical procedures. Clinical trials have focused on male patients with severe disease, and data for females are limited. Improved, evidence-based treatment guidelines for management of hemophilia A carriers are required.
Objectives and design: The NuDIMENSION study is a phase IV, prospective, open-label, single-arm study that will evaluate the perioperative efficacy and safety of simoctocog alfa (Nuwiq®), a recombinant factor VIII (FVIII), in women/girls with hemophilia A undergoing major surgery. The study will be conducted at approximately 15 centers worldwide. Women/girls aged ⩾12 years, with mild or moderate hemophilia A (residual FVIII activity (FVIII:C) ⩾1% to <40%) and with no current/past FVIII inhibitors are eligible. All patients must be scheduled to undergo a major surgical procedure during which simoctocog alfa will be administered.
Methods and analysis: The primary endpoint is overall perioperative hemostatic efficacy ("success" or "failure") of simoctocog alfa. Hemostatic efficacy will be assessed at the end of surgery and at the end of the postoperative period (i.e., completion of wound healing), with overall adjudication by an Independent Data Monitoring Committee. Safety endpoints will include the incidences of thrombotic events and FVIII inhibitor development. The aim is to recruit 28 patients to achieve 26 evaluable surgeries.
Ethics: Ethical approval will be received from institutional review boards/independent ethics committees, and the study will be conducted in compliance with the Declaration of Helsinki.
Discussion: Data from NuDIMENSION will generate much-needed evidence on surgical management of women/girls with hemophilia A, which will help to enable the development of treatment guidelines specific for such patients.
Trial registration: CT EU 2022-502061-17-00; NCT05936580.
期刊介绍：
Therapeutic Advances in Hematology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of hematology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in hematology, providing a forum in print and online for publishing the highest quality articles in this area.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
