UPLC-MS/MS Monitoring of Emicizumab Therapy (NCT04472169) | Clinical Trial Compass
Active — Not RecruitingNot Applicable
UPLC-MS/MS Monitoring of Emicizumab Therapy
France100 participantsStarted 2022-04-13
Plain-language summary
Emicizumab is a monoclonal bispecific antibody with a terminal half-life of 28 days which is now licensed in the treatment of severe haemophilia A with or without inhibitors. Some heterogeneity in residual emicizumab concentrations have been reported according to age, body mass index or drug therapeutic regimen. Some cases of neutralizing antidrug antibodies have been also reported. Whether monitoring emicizumab plasma concentration could predict the residual bleeding risk under emicizumab is unknown. As conventional coagulation assays are not adapted for emicizumab monitoring, this study aims to assess the value of monitoring residual emicizumab plasma concentration by UPLC-MS/MS in bleeding risk prediction.
Who can participate
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:
* Adult or child with Clinical diagnosis of severe hemophilia A (FVIII activity \< 1%) with or without inhibitor
* Clinical indication to emicizumab therapy
Exclusion Criteria:
* Refusal to give informed consent
* acquired hemophilia A
* other inherited or acquired bleeding disorder
* bodyweight \< 10 kgs
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
Area under the curve ROC of Residual plasma level of emicizumab
Timeframe: At Week 5 (end of emicizumab loading period)