UPLC-MS/MS Monitoring of Emicizumab Therapy (EMICARE)

February 9, 2023 updated by: University Hospital, Lille

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

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.

Study Overview

Status

Recruiting

Conditions

Study Type

Observational

Enrollment (Anticipated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Caen, France
        • Not yet recruiting
        • CHU de Caen
      • Lille, France, 59037
        • Recruiting
        • Institut Coeur-Poumon, Pôle d'Hématologie-Transfusion, CHU

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • ADULT
  • OLDER_ADULT
  • CHILD

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

100 patients with severe hemophilia A will be included in the study on 2 french Hemophilia Treatement Centers (CHU Lille and CHU Caen)

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Severe haemophila A patients with or without inhibitors

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
At Week 5 (end of emicizumab loading period)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
At Week 5 (end of emicizumab loading period)
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
At each breakthrough bleeding until end of study
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)
At Week 5 and at each breakthrough bleeding until end of study

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital, Lille

Collaborators

Groupement Interrégional de Recherche Clinique et d'Innovation

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

April 13, 2022

Primary Completion (ANTICIPATED)

February 1, 2024

Study Completion (ANTICIPATED)

February 1, 2024

Study Registration Dates

First Submitted

July 10, 2020

First Submitted That Met QC Criteria

July 10, 2020

First Posted (ACTUAL)

July 15, 2020

Study Record Updates

Last Update Posted (ACTUAL)

February 10, 2023

Last Update Submitted That Met QC Criteria

February 9, 2023

Last Verified

February 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2019_75
  • 2020-A00584-35 (OTHER: ID-RCB number,ANSM)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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