A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors (NCT05306418) | Clinical Trial Compass
CompletedPhase 3
A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors
United States, Canada, China70 participantsStarted 2022-04-04
Plain-language summary
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.
Who can participate
Age range
1 Year – 11 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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 :
. Participant has been prescribed treatment with FVIII concentrate or bypassing agent in the last 26 weeks prior to screening.
. Participants with endogenous FVIII activity greater than or equal to 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 below 1%).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of treatment emergent adverse events
Timeframe: From treatment initiation to follow up visit (week 0 to week 72)
. 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.( For China mainland; assessed at the investigator's discretion unless otherwise stated.)
Exclusion criteria
. Known or suspected hypersensitivity to trial product or related products.(For China mainland; assessed at the investigator's discretion unless otherwise stated.)
. Previous participation in this study. Participation is defined as signed informed consent.
. 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.
. 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 (e.g. 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's discretion unless otherwise stated.)
. 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.(For China mainland; assessed at the investigator's 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's discretion unless otherwise stated.)