Treatment of Hemophilia A Patients With FVIII Inhibitors

Part of paid clinical trials in Atlanta, Georgia.

Sponsor
Emory University
Study ID
NCT04023019
Status
Recruiting
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Conditions

  • Hemophilia A

Eligibility Criteria

Sex
MALE
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Nuwiq — BIOLOGICAL
    Nuwiq is a recombinant FVIII concentrate from a human cell line. It is administered via intravenous injection at a dosage determined by the investigator's discretion, in consideration of the participants' clinical condition and prescribing information.
  • Octanate — BIOLOGICAL
    Octanate is a high-purity human Factor VIII / von Willebrand Factor (VWF) concentrate. It is administered via intravenous injection at a dosage determined by the investigator's discretion, in consideration of the participants' clinical condition and prescribing information.
  • Wilate — BIOLOGICAL
    Wilate is a high-purity human von Willebrand Factor (VWF)/Factor VIII concentrate. It is administered via intravenous injection at a dosage determined by the investigator's discretion, in consideration of the participants' clinical condition and prescribing information.
  • Emicizumab — BIOLOGICAL
    Emicizumab is a therapeutic antibody which brings activated factor IX and factor X together It is administered via subcutaneous injection at a dosage determined by the investigator's discretion, in consideration of the participants' clinical condition and prescribing information.
  • Recombinant factor VIIa (rFVIIa) — BIOLOGICAL
    Recombinant factor VIIa (rFVIIa) is a blood factor VII manufactured using recombinant technology. It is administered via intravenous injection at a dosage determined by the investigator's discretion, in consideration of the participants' clinical condition and prescribing information.
  • Activated prothrombin complex concentrate (aPCC) — BIOLOGICAL
    Activated prothrombin complex concentrate (aPCC) is an anti-inhibitor coagulant complex acting on multiple pathways to facilitate coagulation. It is administered via intravenous injection at a dosage determined by the investigator's discretion, in consideration of the participants' clinical condition and prescribing information.

Study Details

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.

Key Dates

Start date
Mar 17, 2020
Status verified
Aug 2025
Primary completion
Dec 31, 2028
Completion
Jun 30, 2029

Study Design

Enrollment
120 participants (estimated)

Arms

  • Arm: Group 1: ITI with Nuwiq, octanate, or wilate
    Participants receiving immune tolerance induction with either Nuwiq, octanate, or wilate. As needed, aPCC/rFVIIa will be administered to treat bleeding episodes or during surgery and for prophylaxis.
  • Arm: Group 2: ITI with Nuwiq, octanate, or wilate with emicizumab
    Participants receiving immune tolerance induction with either Nuwiq, octanate, or wilate, in combination with emicizumab prophylaxis. As needed, aPCC/rFVIIa will be administered to treat bleeding episodes or during surgery.
  • Arm: Group 3: Prophylaxis with emicizumab, aPCC, or rFVIIa
    Participants receiving routine prophylaxis with emicizumab, aPCC, or rFVIIa without immune tolerance induction. On-demand aPCC/rFVIIa can be used as needed to treat bleeding episodes or during surgery.

Primary Outcome Measure

Proportion of participants achieving inhibitor titer < 0.6 Bethesda units (BU)/mL L for at least 2 consecutive measurements [ Time Frame: Up to 5 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Arthur M. Blank HospitalAtlantaGeorgia30329
Robert Sidonio, MD, MSc
[email protected]
404-785-1637
Robert Sidonio, MD, MSc (PRINCIPAL_INVESTIGATOR)

Find similar trials in Atlanta, GA

By research site
Arthur M. Blank Hospital· Atlanta, GA

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