CTRI/2023/02/049474已完成3 期
A multinational, open-label, randomised, controlled study to investigate efficacy and safety of NNC0365-3769 (Mim8) in adults and adolescents with haemophilia A with or without inhibitors.
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 267
- 试验地点
- 4
- 主要终点
- To confirm the haemostatic effect of Mim8 as bleeding prophylaxis for adults and adolescents with haemophilia A with or without inhibitors by demonstrating superiority in number of bleeding episodes when treated with Mim8 once-weekly versus no prophylaxis followed by Mim8 once-monthly versus no prophylaxis for participants on no prophylaxis treatment prior to enrolment.
研究概览
简要总结
This study is investigating how Mim8 works compared to other medicines in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used for prevention of bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII).
Whenand how often participants will receive Mim8 is dependent on their previoustreatment - but is otherwise decided by chance. Mim8 will be injected into askinfold on the stomach with a thin needle either once a week or once a month.
Thestudy will last 54-124 weeks (12-29 months) depending on how long participantswill be followed in run-in before they start treatment and if they continue inthe follow period or transfer to an open label extension study. Participantswill have 12-17 clinic visits.
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
- 12.00 Year(s) 至 99.00 Year(s)（—）
- 性别
- All
入选标准
- •â€¢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.
- •â€¢Male or female participants with diagnosis of congenital haemophilia A of any severity based on medical records.
- •â€¢Participant has been prescribed treatment with factor VIII concentrates or bypassing agent in the last 26 weeks prior to screening.
- •â€¢Age above or equal to 12 years at the time of signing informed consent.
- •â€¢Body weight greater than or equal to 30 kg.
- •â€¢Applicable to participants treated with on-demand/no prophylaxis prior to enrolment: ≥5 bleeds in the last 26 weeks prior to screening visit, for which factor VIII concentrates or bypassing agent has been prescribed.
- •â€¢Applicable to participants with FVIII activity ≥1% who are on prophylactic treatment: ≥1 bleed in the last 26 weeks prior to screening visit, for which factor VIII concentrates or bypassing agent has been prescribed.
- •â€¢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
- •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 the last dose within 6 months (or 5 half-lives of the investigational medicinal product, whichever is shorter) before planned randomisation.
- •Exposure to non-factor hemostatic 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.
- •Female who is pregnant, breast-feeding or intends to become pregnant or is of child-bearing potential and not using a highly effective contraceptive method.
- •Breast feeding is allowed only during the run-in period.
- •Any disorder, except for conditions associated with hemophilia A, which in the investigators opinion might jeopardise participants safety or compliance with the protocol.
- •Known or suspected hypersensitivity to trial product(s), any constituents of the product or to related products.
- •Receipt of gene therapy at any given time point.
- •Ongoing or planned immune tolerance induction (ITI) therapy.
- •Known congenital or acquired coagulation disorders other than hemophilia A.
- •Hepatic dysfunction defined as aspartate aminotransferase (AST) and or alanine aminotransferase (ALT) above 3 times the upper limit combined with total bilirubin above 1.5 times the upper limit measured at screening.
- •Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) below or equal to 30 ml per min per 1.73 meter square for serum creatinine measured at screening.
- •Previous or current thromboembolic disease or events (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or risk of thromboembolic disease, as evaluated by investigator.
- •Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation.
- •Other conditions (example, autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis as evaluated by the investigator.
结局指标
主要结局
To confirm the haemostatic effect of Mim8 as bleeding prophylaxis for adults and adolescents with haemophilia A with or without inhibitors by demonstrating superiority in number of bleeding episodes when treated with Mim8 once-weekly versus no prophylaxis followed by Mim8 once-monthly versus no prophylaxis for participants on no prophylaxis treatment prior to enrolment.
时间窗: Prophylaxis treatment (Arms 3 and 4): | From initiation of run-in (26-52 weeks prior to week 0) to | week 0 and from randomisation (week 0) to end of main | (Week 26)
Unit: Count
时间窗: Prophylaxis treatment (Arms 3 and 4): | From initiation of run-in (26-52 weeks prior to week 0) to | week 0 and from randomisation (week 0) to end of main | (Week 26)
次要结局
- Occurrence of anti-Mim8 antibodies(All participants receiving Mim8 (Arms 2a, 2b, 3 and 4): From randomisation (week 0) to end of extension (week 52))
- Number of treated spontaneous bleeds(No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26))
- Number of injection site reactions(All participants receiving Mim8 (Arms 2a, 2b, 3 and 4): From randomisation (week 0) to end of main (week 26))
- Number of treated joint bleeds(No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26))
- Number of treated traumatic bleeds(No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26))
- Number of target joint bleeds(No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26))
- Consumption of factor product per bleed treatment((number of injections))
- Change in physical function domain of PEDS-QL(All participants (Arms 1, 2a, 2b, 3 and 4): From randomisation (week 0) to the end of the main part (week 26))
- Change in patient’s treatment burden using the Hemo-TEM(All participants (Arms 1, 2a, 2b, 3 and 4): From randomisation (week 0) to the end of the main part (week 26))
- Change in patient’s joint pain score using(Joint Pain Rating Scale)
研究者
研究点 (4)
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