UPLC-MS/MS Monitoring of Emicizumab Therapy

Hemophilia A Clinical Trial

— EMICARE  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04472169
• Other study ID #: 2019_75
• Secondary ID: 2020-A00584-35
• Status: Recruiting
• Start date: April 13, 2022
• Est. completion date: February 2024

Study information

• Verified date: February 2023
• Source: University Hospital, Lille
• Contact: Antoine Rauch, MD,PhD
• Phone: 0320445962
• Email: antoine.rauch[@]chru-lille.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 100
• Est. completion date: February 2024
• Est. primary completion date: February 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A

Locations

Country	Name	City	State
France	CHU de Caen	Caen
France	Institut Coeur-Poumon, Pôle d'Hématologie-Transfusion, CHU	Lille

Sponsors (2)

Lead Sponsor	Collaborator
University Hospital, Lille	Groupement Interrégional de Recherche Clinique et d'Innovation

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Area under the curve ROC of Residual plasma level of emicizumab	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	At Week 5 (end of emicizumab loading period)
Secondary	Residual plasma level of emicizumab measured by UPLC-MS/MS	At least one hemarthrosis from loading period completion (week 5) to the end of study, an average of 1 year	At Week 5 (end of emicizumab loading period)
Secondary	Residual plasma level of emicizumab measured by UPLC-MS/MS	Post-traumatic or spontaneous nature of bleeding event	At each breakthrough bleeding until end of study
Secondary	Residual plasma level of emicizumab (UPLC-MS/MS dosing)	Emicizumab FVIII-like activity (chromogenic FVIII BIOPHEN™assay system with emicizumab calibration)	At Week 5 and at each breakthrough bleeding until end of study