JPRN-jRCT2031220670进行中（未招募）3 期
Safety, efficacy and exposure of subcutaneously administered NNC0365-3769 (Mim8) prophylaxis in children with haemophilia A with or without FVIII inhibitors (NN7769-4516) - Frontier 3
Esaki Risa0 个研究点目标入组 70 人开始时间: 2023年3月2日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 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
- 年龄范围
- >= 1age old 至 <= 11age old（—）
- 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
- •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
排除标准
- •1. Known or suspected hypersensitivity to trial product or related products.a
- •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.a
- •7. Any disorder, except for conditions associated with haemophilia A, that in the opinion of investigator might jeopardise the safety of participants or compliance with the protocol.a
- •8. Mental incapacity, unwillingness to cooperate or a language barrier precluding adequate
- •understanding and cooperation.a
- •9. Lack of adequate parental/caregiver support to enter accurately and timely information
- •regarding treatment and bleeding episodes into an (electronic) diary.a
- •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.a For definition of major surgery, see Table 6-7.
- •12. Immune tolerance induction planned to take place after treatment initiation.a
- •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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