EUCTR2020-003467-26-PT进行中（未招募）1 期
Safety, efficacy and exposure of subcutaneously administered NNC0365-3769 (Mim8) prophylaxis in children with haemophilia A with or without FVIII inhibitors
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 70
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1.Informed consent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
- •2.Male and female participants with the diagnosis of congenital haemophilia A of any severity based on medical records.
- •3.Aged 1-11 years (both inclusive) at the time of signing informed consent.
- •4.For previously treated participants:
- •a.Participant has been prescribed treatment with FVIII concentrate or bypassing agent in the last 26 weeks prior to screening.
- •b.Participants with endogenous FVIII activity =1%, based on medical records, must have at least 1 treated bleed during the previous 26 weeks before screening for which factor VIII concentrate or bypassing agent has been prescribed (No requirements for participants with FVIII activity <1%).
- •5.For previously untreated participants:
- •a.Diagnosis of severe haemophilia A (endogenous FVIII activity < 1%) based on medical records.
- •6.Child and parent/caregiver willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 70
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1.Known or suspected hypersensitivity to trial product or related products.
- •2.Previous participation in this study. Participation is defined as signed informed consent.
- •3.Participation (i.e., signed informed consent) in any interventional clinical study with receipt of last dose within 6 months (or 5 half-lives of the investigational medicinal product, whichever is shorter) before planned randomisation.
- •4.Exposure to non-factor haemostatic products for bleeding prophylaxis within 6 months (or 5 half-lives of the medicinal product, whichever is shorter) before planned randomisation, for participants not included in the run-in.
- •5.Known congenital or acquired coagulation disorders other than haemophilia A.
- •6.Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis, as evaluated by the investigator.
- •7.Any disorder, except for conditions associated with haemophilia A, that in the investigator’s opinion might jeopardise the participant’s safety or compliance with the protocol.
- •8.Mental incapacity, unwillingness to cooperate or a language barrier precluding adequate understanding and cooperation.
- •9.Lack of adequate parental/caregiver support to enter accurately and timely information regarding treatment and bleeding episodes into an (electronic) diary.
- •10.Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease.
- •11.Major surgery planned to take place after screening. For definition of major surgery, see Table ?6-7.
- •12.Immune tolerance induction planned to take place after treatment initiation.
- •13.Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) >3 times the upper limit of normal combined with total bilirubin >1.5 times the upper limit of normal measured at screening.
- •14.Serum creatinine above 1.5 x upper limit of normal (ULN), measured at screening.
- •15.Pregnancy (female participants).
研究者
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