UPLC-MS/MS Monitoring of Emicizumab Therapy

Value of Emicizumab Monitoring With UPLC-MS/MS for Bleeding Risk Prediction in Severe Hemophilia A

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04472169

Acronym

EMICARE

Enrollment

100

Registered

2020-07-15

Start date

2022-04-13

Completion date

2026-11-01

Last updated

2026-05-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia A

Keywords

Drug monitoring, Severe Hemophilia A, Emicizumab

Brief 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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
Area under the curve ROC of Residual plasma level of emicizumab	At Week 5 (end of emicizumab loading period)	At least one clinically significant bleeding (defined as any bleeding treated with FVIII, rFVIIa or aPCC) from loading period completion (week 5) to the end of study, an average of 1 year

Secondary

Measure	Time frame	Description
Residual plasma level of emicizumab measured by UPLC-MS/MS	At Week 5 (end of emicizumab loading period)	At least one hemarthrosis from loading period completion (week 5) to the end of study, an average of 1 year
Residual plasma level of emicizumab (UPLC-MS/MS dosing)	At Week 5 and at each breakthrough bleeding until end of study	Emicizumab FVIII-like activity (chromogenic FVIII BIOPHEN™assay system with emicizumab calibration)

Countries

France

Contacts

PRINCIPAL_INVESTIGATORAntoine Rauch, MD,PhD

University Hospital, Lille

Outcome results