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Treatment of Hemophilia A Patients With FVIII Inhibitors

MOdern Treatment of Inhibitor-PositiVe PATiEnts With Haemophilia A - An International Observational Study

Who can join

All ages · Men

Inclusion Criteria:

* Male persons with haemophilia A, of any severity, who have a historical inhibitor titer ≥ 0.6 BU/mL, including those who have failed previous immune tolerance induction (ITI) attempt(s)
* Persons undergoing ITI with Nuwiq, octanate, or wilateor undergoing ITI with Nuwiq®, octanate® or wilate® and receiving prophylactic therapy with emicizumab, activated prothrombin complex concentrate (aPCC), or activated recombinant factor VII (rFVIIa)
* Participants or participants' parent(s)/legal guardian(s) must be capable of giving signed informed consent and be able to understand the trial documents

Exclusion Criteria:

* Participants are excluded from the trial if any coagulation disorder other than haemophilia A is diagnosed
* Partly retrospective patients will be excluded if detailed documentation on treatment, all bleeding episodes, inhibitor titers, and FVIII levels is not available for the retrospective period

About the study

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.

What is being tested

• Nuwiq (biological)
• Octanate (biological)
• Wilate (biological)
• Emicizumab (biological)
• Recombinant factor VIIa (rFVIIa) (biological)
• Activated prothrombin complex concentrate (aPCC) (biological)

Sponsor: Emory University · Participants: 120 · Started: Mar 17, 2020

Contact the study team

• Robert Sidonio, MD, MSc · Phone: 404-785-1637
• Carmen Escuriola-Ettingshausen, MD · Phone: +4961059638909

Official record on ClinicalTrials.gov — NCT04023019

Locations in the U.S.

Conditions

• Hemophilia
• Blood Disorders

From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.