Treatment of Hemophilia A Patients With FVIII Inhibitors

Summary

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

• Hemophilia A

Interventions

BIOLOGICAL

Nuwiq

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.

BIOLOGICAL

Octanate

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.

BIOLOGICAL

Wilate

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.

BIOLOGICAL

Emicizumab

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.

BIOLOGICAL

Recombinant factor VIIa (rFVIIa)

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.

BIOLOGICAL

Activated prothrombin complex concentrate (aPCC)

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.

Sponsors & Collaborators

• Octapharma

  collaborator INDUSTRY

• Emory University

  lead OTHER

Principal Investigators

• Robert Sidonio, MD, MSc · Emory University

Eligibility

Sex

MALE

Healthy Volunteers

No

Timeline & Regulatory

Start

2020-03-17

Primary Completion

2028-12-31

Completion

2029-06-30

FDA Drug

Yes

Countries

• United States
• Germany

Study Locations