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

NCT05802836 | Recruiting

• 2 other identifiers: ML44394DRKS00031196
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Severe Hemophilia A; Severe Hemophilia A With Inhibitor; Severe Hemophilia A Without Inhibitor

Outcome Measures

Primary Outcomes (4)

• FVIII inhibitor development in inhibitor negative subjects

  Rate of FVIII inhibitor development during three years of emicizumab prophylaxis in inhibitor negative subjects. Assessed with Bethesda Assay (BU/ml). Number of patients who develop an FVIII inhibitor within the study period, but were FVIII inhibitor negative at start of the study.

  3 years

• FVIII antibody development in inhibitor negative subjects

  Rate of FVIII antibody development during three years of emicizumab prophylaxis in inhibitor negative subjects. FVIII anti drug antibody (ADA) is assessed by FVIII specific ELISA (OD=Optical Density). Number of patients who develop an FVIII antibody (ADA) within the study period, but were FVIII inhibitor negative at start of the study.

  3 years

• FVIII inhibitor disappearance in inhibitor positive subjects

  Rate of FVIII inhibitor disappearance during three years of emicizumab prophylaxis in inhibitor positive subjects. Assessed with Bethesda Assay (BU/ml). Number of patients who loose an FVIII inhibitor within the study period, but were FVIII inhibitor positive at start of the study.

  3 years

• FVIII antibody disappearance in inhibitor positive subjects

  Rate of FVIII antibody disappearance during three years of emicizumab prophylaxis in inhibitor positive subjects. FVIII anti drug antibody (ADA) is assessed by FVIII specific ELISA (OD=Optical Density). Number of patients who develop an FVIII antibody within the study period, but were FVIII inhibitor positive at start of the study.

  3 years

Secondary Outcomes (7)

• Anti-FVIII inhibitor development

  3 years

• Anti-FVIII antibody development

  3 years

• Time to negative inhibitor titers

  3 years

• Treatment of bleeds

  3 years

• Response to treatment

  3 years

Study Arms (3)

Inhibitor negative, FVIII on demand or regularly

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,

Other: no interventions

Inhibitor positive, FVIII therapy regularly (ITI)

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

Other: no interventions

Inhibitor positive, no FVIII therapy

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

Other: no interventions

Interventions

no interventions OTHER

no intervention, only 3 different patients groups

Inhibitor negative, FVIII on demand or regularly; Inhibitor positive, FVIII therapy regularly (ITI); Inhibitor positive, no FVIII therapy

Eligibility Criteria

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

Study Population

The study aims to recruit 100 patients with severe hemophilia A from 15 study sites in germany.

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Christoph Königs: lead
• Roche Pharma AG: collaborator
• Chugai Pharma Germany GmbH: collaborator

Study Sites (1)

University Hospital Frankfurt, Goethe University

Frankfurt am Main, Hesse, 60590, Germany

RECRUITING

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

• Christoph Koenigs, PD Dr. Dr

  Goethe University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Stephan Schultze-Strasser, Dr.

CONTACT

+496963016998; stephan.schultze-strasser@kgu.de

Christoph Koenigs, PD Dr. Dr

CONTACT

+4969630183030; christoph.koenigs@kgu.de

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator, Head of molecular and clinical Haemostasis

Study Record Dates

• First Submitted: March 6, 2023
• First Posted: April 7, 2023
• Study Start: April 26, 2023
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029
• Last Updated: July 10, 2023

Record last verified: 2023-07

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)