UPLC-MS/MS Monitoring of Emicizumab Therapy

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Sex eligibility

All sexes

Study type

Observational

Primary location

CHU de Caen, Caen, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

Area under the curve ROC of Residual plasma level of emicizumab

Time frame: 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 outcomes

Residual plasma level of emicizumab measured by UPLC-MS/MS

Time frame: 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 measured by UPLC-MS/MS

Time frame: At each breakthrough bleeding until end of study

Post-traumatic or spontaneous nature of bleeding event
Residual plasma level of emicizumab (UPLC-MS/MS dosing)

Time frame: At Week 5 and at each breakthrough bleeding until end of study

Emicizumab FVIII-like activity (chromogenic FVIII BIOPHEN™assay system with emicizumab calibration)

Sponsors and collaborators

Lead sponsor

University Hospital, Lille

Other

Collaborators

Groupement Interrégional de Recherche Clinique et d'Innovation

Registry information

Official study title

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

Acronym: EMICARE

Important dates

Study start: 2022
Primary completion: 2026
Study completion: 2026
First posted: Jul 15, 2020
Registry last updated: May 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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