UPLC-MS/MS Monitoring of Emicizumab Therapy

NCT04472169 | Unknown

• 2 other identifiers: 2019_752020-A00584-35
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 2

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.

Conditions

Hemophilia A

Keywords

Drug monitoring; Severe Hemophilia A; Emicizumab

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Residual plasma level of emicizumab measured by UPLC-MS/MS

  At Week 5 (end of emicizumab loading period)

• Residual plasma level of emicizumab measured by UPLC-MS/MS

  At each breakthrough bleeding until end of study

• Residual plasma level of emicizumab (UPLC-MS/MS dosing)

  At Week 5 and at each breakthrough bleeding until end of study

Study Arms (1)

Severe haemophila A patients with or without inhibitors

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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)

You may qualify if:

• Adult or child with Clinical diagnosis of severe hemophilia A (FVIII activity \< 1%) with or without inhibitor
• Clinical indication to emicizumab therapy

You may not qualify if:

• Refusal to give informed consent
• acquired hemophilia A
• other inherited or acquired bleeding disorder
• bodyweight \< 10 kgs

Sponsors & Collaborators

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

Study Sites (2)

CHU de Caen

Caen, France

NOT YET RECRUITING

Institut Coeur-Poumon, Pôle d'Hématologie-Transfusion, CHU

Lille, 59037, France

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Serum, poor-platelet plasma, whole blood

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Antoine Rauch, MD,PhD

  University Hospital, Lille

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Antoine Rauch, MD,PhD

CONTACT

0320445962; antoine.rauch@chru-lille.fr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 10, 2020
• First Posted: July 15, 2020
• Study Start: April 13, 2022
• Primary Completion: February 1, 2024
• Study Completion: February 1, 2024
• Last Updated: February 10, 2023

Record last verified: 2023-02

Locations

FR (2)