Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery - an International, Open-label, Non-controlled Study (NuDIMENSION)
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 发起方
- Octapharma
- 入组人数
- 28
- 试验地点
- 26
- 主要终点
- Overall haemostatic efficacy
研究概览
简要总结
Recombinant factor VIII for the prevention of bleeding in women/girls with haemophilia A undergoing major surgery
详细描述
Female patients with mild to moderate haemophilia A will often need FVIII concentrates to provide haemostatic cover during major surgery. This prospective, open-label, non-controlled, single-arm, multinational, multicentre study aims to evaluate the overall perioperative haemostatic efficacy of Nuwiq in women/girls over 12 with haemophilia A undergoing major surgery requiring FVIII treatment
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Women/girls with haemophilia A (FVIII:C ≥1-<40%) according to medical history. Additionally, women/girls with documented FVIII activity levels between ≥ 40% and 50% may be included if there is a documented history of clinically significant bleeding episodes consistent with haemophilia A; and/or either documented prior treatment with FVIII concentrates or a clinical indication that FVIII treatment would have been appropriate (e.g., use of FVIII from a family member, or treatment with alternative haemostatic agents due to access limitations)
- •At least 12 years of age
- •Scheduled to undergo major surgery* requiring FVIII treatment, including elective and emergency procedures and caesarean section in pregnant women with haemophilia A
- •Freely given written informed consent of the patient, or parent/legal representative where applicable, obtained in accordance with local regulations
排除标准
- •Coagulation disorder other than haemophilia A
- •Present or past FVIII inhibitor (≥0.6 Bethesda units [BU]/mL)
- •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)
- •Pregnancy, except in participants with a planned caesarean section
- •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
研究组 & 干预措施
Nuwiq
All patients receiving Nuwiq (recombinant FVIII). Nuwiq will be administered intravenously in accordance with the relevant prescribing information. Treatment will be repeated as necessary every 8-24 hours until adequate wound healing, then - if required - for at least another 7 days to maintain FVIII plasma levels of 30-60 IU/dL.
干预措施: Nuwiq (Drug)
结局指标
主要结局
Overall haemostatic efficacy
时间窗: 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
Overall haemostatic efficacy of treatment measured as binary "success" or "failure". The overall perioperative haemostatic efficacy of Nuwiq will be adjudicated by an Independent Data Monitoring Committee (IDMC) and determined using a composite assessment algorithm that considers the surgeon's assessment of intraoperative haemostatic efficacy and the investigator's assessment of postoperative haemostatic efficacy to classify the overall haemostatic efficacy as success or failure.
次要结局
- FVIII inhibitor formation(From start of first Nuwiq injection to 30 ±3 days following the surgical procedure)
- Intraoperative haemostatic efficacy(During surgery: From first skin incision to last suture)
- Postoperative haemostatic efficacy(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)
- Allogenic blood products(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)
- FVIII plasma levels(≤30 minutes before and 15-30 minutes after Nuwiq injection)
- Perioperative haemostatic efficacy per WFH criteria(≤30 minutes before and 15-30 minutes after Nuwiq injection)
- Adverse events(From start of first Nuwiq injection to 30 ±3 days following the surgical procedure)
- Thrombotic events(From start of first Nuwiq injection to 30 ±3 days following the surgical procedure)