Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes (FORWARD)

Bioverativ

Status: Terminated

Conditions: Hemophilia B; Hemophilia A

Treatments: Drug: non-Fc FVIII replacement products; Biological: rFIXFc; Drug: non-Fc FIX replacement products; Biological: rFVIIIFc

Study type: Observational

Funder types: Industry

Identifiers

NCT02392156

997HA401

Details and patient eligibility

About

The primary objective of the study is to evaluate the effectiveness of prophylactic treatment with recombinant Factor VIII Fc fusion protein (rFVIIIFc) and recombinant Factor IX Fc fusion protein (rFIXFc) therapy as assessed by patient treatment burden and health economic outcomes while maintaining disease control in males with hemophilia A or B.

Enrollment: 3 patients

Sex: Male

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Have a medically documented diagnosis of hemophilia A or B that is being treated prophylactically with a factor replacement product not designed to have a prolonged half-life and satisfy a therapeutic indication for rFVIIIFc/rFIXFc per the approved local label
• Have at least 50 prior exposure days (EDs) to any combination of factor replacement products
• Have documented pre-study data available that confirm fulfillment of the eligibility criteria
• Have no measurable inhibitor activity in a sample obtained within 4 weeks prior to the Baseline visit, and absence of clinical signs or symptoms of decreased response to the current factor replacement product

Key Exclusion Criteria:

• Have a diagnosis of any bleeding disorder other than hemophilia A or hemophilia B or an additional coagulation disorder(s) in addition to hemophilia A or hemophilia B
• Have a prior history of anaphylaxis associated with any factor VIII (FVIII)/ factor IX (FIX) or intravenous immunoglobin administration
• Had an inhibitor within 5 years before the Baseline visit. Note: A family history of inhibitors will not exclude the patient.
• Past or current treatment with any factor replacement product with a prolonged half-life, including an Fc product, for the treatment of hemophilia

NOTE: Other Protocol Defined Inclusion/ Exclusion Criteria May Apply.

Trial design

3 participants in 4 patient groups

rFVIIIFc for hemophilia A

Description:

Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label

Treatment:

Biological: rFVIIIFc

non-Fc (fusion protein) replacement products for hemophilia A

Description:

Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label

Treatment:

Biological: rFVIIIFc

Drug: non-Fc FVIII replacement products

rFIXFc for hemophilia B

Description:

Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label

Treatment:

Biological: rFIXFc

non-Fc factor replacement products for hemophilia B

Description:

Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label

Treatment:

Drug: non-Fc FIX replacement products

Biological: rFIXFc