UPLC-MS/MS Monitoring of Emicizumab Therapy

NCT04472169 · tracked via the Priya Life Science France tracker

Sponsor

University Hospital, Lille

Phase

Observational

Started

2022-04-13

Last updated

2026-05-20

Condition(s) studied

Hemophilia A

Study 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.

Eligibility

Sex

ALL

Min age

—

Max age

—

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

Primary outcome measure(s)

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

Trial sites (2)

Facility	City	Region	Status
CHU de Caen	Caen	France
Institut Coeur-Poumon, Pôle d'Hématologie-Transfusion, CHU	Lille	France

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04472169 on ClinicalTrials.gov ↗ ← All trials in France