CTRI/2023/01/048708已完成3 期
Safety, efficacy and exposure of subcutaneously administered NNC0365-3769 (Mim8) prophylaxis in children with haemophilia A with or without FVIII inhibitors.
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 70
- 试验地点
- 4
- 主要终点
- Number of treatment emergent adverse events
研究概览
简要总结
This study is looking at how Mim8 works compared to other medicines in children with haemophilia A, who either have inhibitors or do not have inhibitors.
Mim8 is a new medicine that will be used for prevention of bleeds. Mim8 will be injected with a thin needle into the skin. The study will last for about 54-98 weeks, from screening to follow-up visit, In case the participant experiences bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor.
研究设计
- 研究类型
- Interventional
- 分配方式
- Computer generated randomization
- 盲法
- Open Label
入排标准
- 年龄范围
- 1.00 Year(s) 至 11.00 Year(s)（—）
- 性别
- All
入选标准
- •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. Male and female participants with the diagnosis of congenital haemophilia A of any severity based on medical records. Aged 1-11 years (both inclusive) at the time of signing informed consent. For previously treated participants.
- •a. Participant has been prescribed treatment with Factor Eight concentrate or bypassing agent in the last 26 weeks prior to screening. b. Participants with endogenous factor eight activity greater than or equal to 1 percentage, based on medical records, must have at least 1 treated bleed during the previous 26 weeks before screening for which factor eight concentrate or bypassing agent has been prescribed (No requirements for participants with factor right activity below 1 percentage). For previously untreated participants.
- •a. Diagnosis of severe haemophilia A (endogenous Factor eight activity below 1 percentage) based on medical records. Child and parent or caregiver willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires. (For China mainland, assessed at the investigator discretion unless otherwise stated.).
排除标准
- •Known or suspected hypersensitivity to trial product or related products.
- •(For China mainland, assessed at the investigator discretion unless otherwise stated.) Previous participation in this study.
- •Participation is defined as signed informed consent.
- •Participation (that is, 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.
- •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.
- •Known congenital or acquired coagulation disorders other than haemophilia A.
- •Other conditions (eg, autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis, as evaluated by the investigator.
- •(For China mainland, assessed at the investigator discretion unless otherwise stated.) Any disorder, except for conditions associated with haemophilia A, that in the investigator opinion might jeopardise the participant safety or compliance with the protocol.
- •(For China mainland, assessed at the investigator discretion unless otherwise stated.) Mental incapacity, unwillingness to cooperate or a language barrier precluding adequate understanding and cooperation.
- •(For China mainland; assessed at the investigator discretion unless otherwise stated.) Lack of adequate parental or caregiver support to enter accurately and timely information regarding treatment and bleeding episodes into an (electronic) diary.
- •(For China mainland, assessed at the investigator discretion unless otherwise stated.) 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.
- •(For China mainland, assessed at the investigator discretion unless otherwise stated.) Immune tolerance induction planned to take place after treatment initiation.
- •(For China mainland, assessed at the investigator discretion unless otherwise stated.) Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) greater than 3 times the upper limit of normal combined with total bilirubin greater than 1.5 times the upper limit of normal measured at screening.
- •Hepatic dysfunction defined as aspartate aminotransferase (AST) and or alanine aminotransferase (ALT) greater than 3 times the upper limit of normal combined with total bilirubin greater than 1.5 times the upper limit of normal measured at screening.
- •Serum creatinine above 1.5 multiplied by the upper limit of normal (ULN), measured at screening.
- •Pregnancy (female participants).
- •(Will be assessed at investigator discretion, according to suspicion of pregnancy.).
结局指标
主要结局
Number of treatment emergent adverse events
时间窗: Time Frame: From treatment initiation to follow up visit (week 0 to week 72) | Count of events
次要结局
- Number of treated bleeds(Time Frame: From treatment initiation to end of treatment (week 0 to week 52))
- Consumption of factor product per bleed treatment(Time Frame: From run-in initiation to end of treatment (week -26 to week 52))
- Occurrence of anti-Mim8 antibodies(Time Frame: From treatment initiation to end of treatment (week 0 to week 52))
- Mim8 plasma concentration(Time Frame: From treatment initiation to end of treatment (week 0 to week 52))
- Change in physical function domain of PEDS QL (Paediatric Quality of Life inventory) Generic Core Scales(Time Frame: From treatment initiation to end of treatment (week 0 to week 52))
- Treatment preference for Mim8 versus previous treatment using Caregiver H PPQ (Caregiver Haemophilia Patient Preference )(Time Frame: Once during treatment (week 26))
- Change in participants treatment burden using the Hemo TEM (Haemophilia treatment experience measure)(Time Frame: From treatment initiation to end of treatment (week 0 to week 52))
- Number of treated spontaneous bleeds(Time Frame: From treatment initiation to end of treatment (week 0 to week 52)
- Number of treated traumatic bleeds(Time Frame: From treatment initiation to end of treatment (week 0 to week 52))
- Number of treated joint bleeds(Time Frame: From treatment initiation to end of treatment (week 0 to week 52))
- Number of treated target joint bleeds(Time Frame: From treatment initiation to end of treatment (week 0 to week 52))
- Number of injection site reactions(Time Frame: From treatment initiation to end of treatment (week 0 to week 52))
研究者
研究点 (4)
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