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Observational Study for Hemophilia A Patients with Inhibitors

This observational study is for men with Hemophilia A (a bleeding disorder) who have developed inhibitors (antibodies that stop treatment from working) to Factor VIII (FVIII) products. The study aims to understand different ways doctors manage this condition and to evaluate how well treatments like Nuwiq, Octanate, Wilate, Emicizumab, and Recombinant factor VIIa (rFVIIa) work and if they are safe. You would be assigned to one of three groups based on the treatment you are already receiving, and you might switch groups if your treatment changes. The study will look at whether your inhibitor levels decrease and if your FVIII recovery and half-life improve over up to five years.

Study design
This is an observational study involving 120 male participants. It is multicenter and international, gathering information on current treatment approaches.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for a maximum observational period of five years.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04023019

Treatment of Hemophilia A Patients With FVIII Inhibitors

Recruiting
Not specifiedAll AgesObservational
Emory University
~120 participants
Updated 2025-09-05 on ClinicalTrials.gov
What's tested:NuwiqOctanateWilateEmicizumabRecombinant factor VIIa (rFVIIa)Activated prothrombin complex concentrate (aPCC)

At a glance

Recruiting sites
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Proportion of participants achieving inhibitor titer < 0.6 Bethesda units (BU)/mL L for at least 2 consecutive measurements
Measured over Up to 5 years
+3 more outcomes measured

Conditions

Hemophilia A

Explore related trials

Hemophilia AFVIII inhibitor

Where it's being run

2 sites across 2 states
Georgia1
Germany1

Study leadership

  • Robert Sidonio, MD, MSc · PRINCIPAL_INVESTIGATOR · Emory University

Who to contact

Robert Sidonio, MD, MSc
[email protected]
404-785-1637
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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Proportion of participants achieving inhibitor titer < 0.6 Bethesda units (BU)/mL L for at least 2 consecutive measurementsUp to 5 years

    The proportion of participants in Groups 1 and 2 achieving inhibitor titer \< 0.6 Bethesda units (BU)/mL L for at least 2 consecutive measurements will be determined. FVIII inhibitor titer is measured at baseline and throughout the study, according to standard of care.

  • Proportion of participants achieving FVIII recovery ≥ 66% of the predefined reference value of 1.5% IU/kg body weight (Groups 1 and 2)Up to 5 years

    The proportion of participants in Groups 1 and 2 achieving FVIII recovery ≥ 66% of the predefined reference value of 1.5% IU/kg body weight will be determined. Once inhibitor has become negative (\< 0.6 BU/mL), FVIII plasma levels are measured prior to and approximately 15 to 30 minutes after FVIII to evaluate FVIII recovery.

  • Proportion of participants achieving FVIII half-life ≥ 6 h (Groups 1 and 2)Up to 5 years

    The proportion of participants in Groups 1 and 2 achieving FVIII half-life ≥ 6 h will be determined. Once inhibitor has become negative (\< 0.6 BU/mL), FVIII plasma levels are measured prior to and at 15-30 minutes and 2, 4, 8-12, and 24 hours after administration of the immune tolerance induction (or prophylactic FVIII) to evaluate half-life; when FVIII trough levels are \> 1% during regular prophylaxis, half-life can be evaluated from fewer samples or using a population pharmacokinetic model.

  • Annualized bleeding rateUp to 5 years

    Annualized rate of all bleeding episodes will be reported and compared between all 3 study groups.