Open-label, long-term safety and efficacy study of Mim8 in participants withhaemophilia A with or without inhibitors
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 425
- 试验地点
- 7
- 主要终点
- Number of
研究概览
简要总结
This study is looking at how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used to avoid bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). When and how often the participants will receive Mim8 in this study depends on the treatment participant receives in the current Mim8 study participant is taking part in. The study will last for up to 5.5 years. The duration of the study depends on when the participant enrolled in this study. The study will end if Mim8 is approved and marketed in participants country during the study, or the study will end in 2028, whichever comes first. Mim8 will be injected under the skin with a thin needle either once a week, once every two weeks or once a month. Participants will get up to 262 injections; the number of injections depends on how often participants will get injections. While taking part in this study, there are some restrictions about what medicine participants can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.
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 99.00 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 or female with diagnosis of congenital haemophilia A based on medical records.
- •Ongoing participation in study 4513, 4514, 4516, or 4728 at the time of transfer.
- •Participant should qualify either of the following criteria: a.
- •Participant from study 4513, who has participated in the extension part of the study for at least 12 weeks prior to enrolment in study 4532, or, b.
- •Participant has completed the end of treatment visit for study 4514,4516, or
- •Participant and/or participants parent(s)/participants LAR willingness and ability to comply with scheduled visits and study procedures, including the completion of diary.
排除标准
- •Any disorder, except for conditions associated with haemophilia, which in the investigators opinion might jeopardise participants safety or compliance with the protocol.
- •Participant who has discontinued or been withdrawn from studies 4513, 4514, 4516, or
- •Previous participation in this study.
- •Participation is defined as signed informed consent.
- •Female who is pregnant, breast-feeding or intends to become pregnant.
- •Female of child-bearing potential and not using a highly effective contraceptive method (highly effective contraceptive measures as defined in Appendix 4 or as required by local regulation or practice).
- •Participation (i.e., signed informed consent) in any other interventional clinical study (except from study 4513, 4514, 4516, or 4728) of an approved or non-approved investigational medicinal product.
- •Any planned major surgery, during part 1 of the study.
- •Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation.
结局指标
主要结局
Number of
时间窗: From Visit 1 (week 0) until end | of study (up to 283 weeks)
treatment-emergent adverse events
时间窗: From Visit 1 (week 0) until end | of study (up to 283 weeks)
次要结局
- Number of injection site reactions(From Visit 1 (week 0) until end)
- Number of treated bleeding episodes(From Visit 1 (week 0) until end)
- Number of treated joint bleeding(episodes)
- Mim8 plasma concentration(From Visit 1 (week 0) until end)
- Change in participants’ treatment burden using the Hemo-TEM (Applicable for participants in Arm 2 only excluding Chinese participants)(From Visit 1 (week 0) until end)
- Change in participant’s joint pain score using JPRS (Applicable for adult & adolescent participants in Arm 2 only excluding Chinese participants)(From Visit 1 (week 0) until end)
- Occurrence of anti-Mim8 antibodies(From Visit 1 (week 0) until end)
- Number of treated target joint bleeding episodes (Arm 2)(From Visit 1 (week 0) until end)
- Number of treated spontaneous(bleeding episodes)
- Number of treated traumatic bleeding episodes(From Visit 1 (week 0) until end)
- Device handling experience using HDAT (applicable for participants in Arm 2 only)(At Visit 8 (week 26) & Visit)
- Change in physical function domain of PedsQL Generic Core Scales (Applicable for participants in Arm 2 only excluding Chinese participants)(From Visit 1 (week 0) until end)