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EMICARE: UPLC-MS/MS Monitoring of Emicizumab Therapy

Sponsor
University Hospital, Lille (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT04472169
Collaborator
Groupement Interrégional de Recherche Clinique et d'Innovation (Other)
100
Enrollment
2
Locations
18
Anticipated Duration (Months)
50
Patients Per Site
2.8
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    100 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Value of Emicizumab Monitoring With UPLC-MS/MS for Bleeding Risk Prediction in Severe Hemophilia A
    Anticipated Study Start Date :
    Aug 1, 2022
    Anticipated Primary Completion Date :
    Feb 1, 2024
    Anticipated Study Completion Date :
    Feb 1, 2024

    Arms and Interventions

    Arm Intervention/Treatment
    Severe haemophila A patients with or without inhibitors

    Outcome Measures

    Primary Outcome Measures

    1. 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 Outcome Measures

    1. 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

    2. Residual plasma level of emicizumab measured by UPLC-MS/MS [At each breakthrough bleeding until end of study]

      Post-traumatic or spontaneous nature of bleeding event

    3. 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)

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 CHU de Caen Caen France
    2 Institut Coeur-Poumon, Pôle d'Hématologie-Transfusion, CHU Lille France 59037

    Sponsors and Collaborators

    • University Hospital, Lille
    • Groupement Interrégional de Recherche Clinique et d'Innovation

    Investigators

    • Principal Investigator: Antoine Rauch, MD,PhD, University Hospital, Lille

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    University Hospital, Lille
    ClinicalTrials.gov Identifier:
    NCT04472169
    Other Study ID Numbers:
    • 2019_75
    • 2020-A00584-35
    First Posted:
    Jul 15, 2020
    Last Update Posted:
    Mar 17, 2022
    Last Verified:
    Feb 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by University Hospital, Lille
    Drug monitoring
    Severe Hemophilia A
    Emicizumab
    Additional relevant MeSH terms:
    Hemophilia A

    Study Results

    No Results Posted as of Mar 17, 2022