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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04472169
Other study ID # 2019_75
Secondary ID 2020-A00584-35
Status Recruiting
Phase
First received
Last updated
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
Health authority
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

Study Design


Related Conditions & MeSH terms


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
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