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Recruiting NCT05802836

Dynamics of the Anti-factor VIII Antibody Signature During Treatment With Emicizumab

— Observational Severe Hemophilia A Severe Hemophilia A With Inhibitor Severe Hemophilia A Without Inhibitor

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: no interventions.
Who it may be relevant to
Registry conditions: Severe Hemophilia A, Severe Hemophilia A With Inhibitor, Severe Hemophilia A Without Inhibitor. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Germany
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Overview

The goal of this observational study is to learn about the changes of antibodies and inhibitors against the coagulation factor VIII in patients with severe hemophilia A receiving emicizumab therapy. No additional visits or procedures are planned. Patients in this study will continue to receive their routine care and analysis will be done from left over samples from routine visits.

Interventions

  • Other no interventions
    no intervention, only 3 different patients groups

Primary outcome measures

  • FVIII inhibitor development in inhibitor negative subjects [Time frame: 3 years]
  • FVIII antibody development in inhibitor negative subjects [Time frame: 3 years]
  • FVIII inhibitor disappearance in inhibitor positive subjects [Time frame: 3 years]
  • FVIII antibody disappearance in inhibitor positive subjects [Time frame: 3 years]
Secondary outcome measures (7)
  • Anti-FVIII inhibitor development [Time frame: 3 years]
  • Anti-FVIII antibody development [Time frame: 3 years]
  • Time to negative inhibitor titers [Time frame: 3 years]
  • Treatment of bleeds [Time frame: 3 years]
  • Response to treatment [Time frame: 3 years]
  • Quality of the antibody response (FVIII epitopes) [Time frame: 3 years]
  • Quality of the antibody response (IgG subclasses) [Time frame: 3 years]

Eligibility criteria

Inclusion criteria

  • Severe congenital hemophilia A (CHA)
  • Treatment with emicizumab irrespective of any other treatment
  • Informed consent

Exclusion criteria

  • No therapy with emicizumab
  • Immunosuppressive therapy
  • HIV-infection with CD4 (cluster of differentiation 4) cells <200/µl

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Other

Study locations

Germany · 1 center
  • University Hospital Frankfurt, Goethe University — Frankfurt am Main

Identifiers

NCT: NCT05802836 · ML44394 · DRKS00031196

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗