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

Christoph Königs

Status: Enrolling

Conditions: Severe Hemophilia A; Severe Hemophilia A Without Inhibitor; Severe Hemophilia A With Inhibitor

Treatments: Other: no interventions

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT05802836

DRKS00031196 (Registry Identifier)

ML44394

Details and patient eligibility

About

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.

Enrollment: 100 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

100 participants in 3 patient groups

Inhibitor negative, FVIII on demand or regularly

Description:

Patients with severe hemophilia A receiving emicizumab therapy which are negative for factor VIII Inhibitor (including patients post ITI) and are receiving factor VIII therapy either on demand or regularly,

Treatment:

Other: no interventions

Inhibitor positive, FVIII therapy regularly (ITI)

Description:

Patients with severe hemophilia A receiving emicizumab therapy which are positive for factor VIII Inhibitor and are receiving regularly factor VIII therapy (ITI)

Treatment:

Other: no interventions

Inhibitor positive, no FVIII therapy

Description:

Patients with severe hemophilia A receiving emicizumab therapy which are positive for factor VIII Inhibitor and are receiving no factor VIII therapy

Treatment:

Other: no interventions