Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes

NCT02392156 | Terminated

• 1 other identifier: 997HA401
• Study Type: observational
• Target: 3
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Hemophilia A; Hemophilia B

Outcome Measures

Primary Outcomes (1)

• Annualized number of injections for prophylactic treatment with a Factor VIII or Factor IX replacement product

  Month 2 to month 14

Secondary Outcomes (9)

• The total annualized factor consumption (in International Units [IU] per kilogram [IU/kg]) calculated for each participant

  Month 2 to month 14

• Change in Treatment Satisfaction Questionnaire for Medication (TSQM)

  Baseline to 14 months

• Change in Hemophilia Activities List (HAL)

  Baseline to 14 months

• Change in Validated Hemophilia Regimen Treatment Adherence Scale-Prophylaxis (VERITAS-Pro)

  Baseline to 14 months

• Change in Wong-Baker Faces Pain Rating Scale

  Baseline to 14 months

Study Arms (4)

rFVIIIFc for hemophilia A

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

Biological: rFVIIIFc

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

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

Biological: rFVIIIFc; Drug: non-Fc FVIII replacement products

rFIXFc for hemophilia B

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

Biological: rFIXFc

non-Fc factor replacement products for hemophilia B

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

Biological: rFIXFc; Drug: non-Fc FIX replacement products

Interventions

rFVIIIFc BIOLOGICAL

As described in the treatment arm

Also known as: antihemophilic factor (recombinant) Fc fusion protein, Eloctate, recombinant coagulation factor VIII Fc fusion protein, efmoroctocog alfa, BIIB031

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

rFIXFc BIOLOGICAL

As described in the treatment arm

Also known as: coagulation factor IX (recombinant) Fc fusion protein, Alprolix, BIIB029

non-Fc factor replacement products for hemophilia B; rFIXFc for hemophilia B

non-Fc FVIII replacement products DRUG

Standard plasma-derived (pd) or recombinant (r) FVIII concentrate and other traditional treatment options

Also known as: rFVIII, pdFVIII

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

non-Fc FIX replacement products DRUG

Standard plasma-derived (pd) or recombinant (r) FIX concentrate and other traditional treatment options

Also known as: rFIX, pdFIX

non-Fc factor replacement products for hemophilia B

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Participants will be recruited at various locations by prescribing physicians.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Bioverativ Therapeutics Inc.: lead
• Swedish Orphan Biovitrum: collaborator

Study Sites (2)

Research Site

Los Angeles, California, 90007, United States

Location

Research Site

East Lansing, Michigan, 48823, United States

Location

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Interventions

factor VIII-Fc fusion protein; factor IX Fc fusion protein; Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Medical Director

  Bioverativ Therapeutics Inc.

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 12, 2015
• First Posted: March 18, 2015
• Study Start: July 1, 2015
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2015
• Last Updated: December 19, 2020

Record last verified: 2018-08

Locations

US (2)