Brief Title
UPLC-MS/MS Monitoring of Emicizumab Therapy
Official Title
Value of Emicizumab Monitoring With UPLC-MS/MS for Bleeding Risk Prediction in Severe Hemophilia A
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.
Study Type
Observational
Primary Outcome
Area under the curve ROC of Residual plasma level of emicizumab
Secondary Outcome
Residual plasma level of emicizumab measured by UPLC-MS/MS
Condition
Hemophilia A
Study Arms / Comparison Groups
Severe haemophila A patients with or without inhibitors
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
100
Start Date
August 2020
Completion Date
February 2022
Primary Completion Date
February 2022
Eligibility Criteria
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
Gender
All
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
Antoine Rauch, MD,PhD, 0320445962, [email protected]
Location Countries
France
Location Countries
France
Administrative Informations
NCT ID
NCT04472169
Organization ID
2019_75
Secondary IDs
2020-A00584-35
Responsible Party
Sponsor
Study Sponsor
University Hospital, Lille
Collaborators
Groupement Interrégional de Recherche Clinique et d'Innovation
Study Sponsor
Antoine Rauch, MD,PhD, Principal Investigator, University Hospital, Lille
Verification Date
June 2020