A Research Study Investigating Mim8 in People With Haemophilia A (NCT04204408) | Clinical Trial Compass
CompletedPhase 2
A Research Study Investigating Mim8 in People With Haemophilia A
United States, Austria, Bulgaria275 participantsStarted 2020-01-10
Plain-language summary
This study is investigating how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medication that will be used for prevention of bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected with a thin needle in the skin of the stomach, using a pen-injector.
The study will last for up to 44 months. It consists of a main phase (part 1 and part 2) and an extension phase. In part 1, participants will be injected only once with either Mim8 or a "dummy" medicine (placebo) - which one will be decided by chance. In part 2 and the extension phase participants will get an Mim8 injection weekly or monthly.
Who can participate
Age range
12 Years
Sex
MALE
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:
Single ascending dose part 1:
* Male, aged 18-45 years (both inclusive) at the time of signing informed consent
* Considered to be generally healthy based on the medical history, physical examination, and the results of vital signs, electrocardiogram and clinical laboratory tests performed during the screening visit, as judged by the investigator
Multiple ascending dose part 2:
* Male, aged 12-64 years (both inclusive) at the time of signing informed consent (Germany and Japan have local requirements)
* Diagnosis of congenital haemophilia A with FVIII activity below 1% based on medical records
Exploratory biomarker cohort:
* Male, aged equal to or above 12 years at the time of signing informed consent (Germany and Japan have local requirements)
* Diagnosis of congenital haemophilia A with FVIII activity below 1% based on medical recordsv
Exclusion Criteria:
Part 1:
* Factor VIII activity equal to or above 150% at screening
* Increased risk of thrombosis, e.g. known history of personal or first degree relative(s) with unprovoked deep vein thrombosis
* Any clinical signs or established diagnosis of venous or arterial thromboembolic disease
Part 2:
* Known congenital or acquired coagulation disorders other than haemophilia A
* Increased risk of thrombosis as evaluated by the investigator. E.g. known history of personal or first degree relative(s) with unprovoked deep vein thrombosis with exception of previous catheter-associated thrombosis for whic…
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
Part 1: Number of treatment emergent adverse events
Timeframe: From time of dosing (Day 1) to Week 16
2
Part 2: Number of treatment emergent adverse events
Timeframe: From time of first dosing (Day 1) to Week 12
3
Part 2, extension: Number of treatment emergent adverse events
Timeframe: From Week 12 up to Week 176 (16 weeks after last dose)