Actively Recruiting

MALE
NCT04023019

Treatment of Hemophilia A Patients With FVIII Inhibitors

Led by Emory University · Updated on 2025-09-05

120

Participants Needed

2

Research Sites

480 weeks

Total Duration

On this page

Sponsors

E

Emory University

Lead Sponsor

O

Octapharma

Collaborating Sponsor

AI-Summary

What this Trial Is About

This is a non-interventional, multicenter, observational, international study in male persons with haemophilia A who have developed inhibitors to any replacement coagulation factor VIII (FVIII) product. The purpose of the study is to capture different approaches in the management of persons with haemophilia A and FVIII inhibitors, document current immune tolerance induction approaches, and evaluate the efficacy and safety of immune tolerance induction, including the combination of FVIII and emicizumab. Patients will be assigned to 1 of 3 groups based on the treatments they receive, and may switch to another group if their treatment is changed. Participants will be followed after a maximum observational period of 5 years.

CONDITIONS

Official Title

Treatment of Hemophilia A Patients With FVIII Inhibitors

Who Can Participate

MALE

Eligibility Criteria

Eligible

You may qualify if you...

  • Male persons with haemophilia A, any severity, with a historical inhibitor titer  0.6 BU/mL, including those who failed previous immune tolerance induction attempts
  • Currently undergoing immune tolerance induction with Nuwiq, octanate, or wilate, or undergoing immune tolerance induction with these plus prophylactic emicizumab, aPCC, or rFVIIa
  • Able to give signed informed consent and understand trial documents (participant or parent/legal guardian)
Not Eligible

You will not qualify if you...

  • Diagnosis of any coagulation disorder other than haemophilia A
  • Partly retrospective patients without detailed documentation on treatment, bleeding episodes, inhibitor titers, and FVIII levels for the retrospective period

AI-Screening

AI-Powered Screening

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Trial Site Locations

Total: 2 locations

1

Arthur M. Blank Hospital

Atlanta, Georgia, United States, 30329

Actively Recruiting

2

HZRM Hämophilie-Zentrum Rhein Main

Mörfelden-Walldorf, Germany, 64546

Actively Recruiting

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Research Team

R

Robert Sidonio, MD, MSc

CONTACT

C

Carmen Escuriola-Ettingshausen, MD

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

3

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