Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes

Bioverativ Therapeutics Inc.3 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Hemophilia A Hemophilia B

Interventions

rFVIIIFcBIOLOGICAL

As described in the treatment arm

rFIXFcBIOLOGICAL

As described in the treatment arm

non-Fc FVIII replacement productsDRUG

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

Eligibility

Sex: MALE

Medical Language ↔ Plain English

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.

Locations (2)

Research Site

Los Angeles, California, United States

Research Site

East Lansing, Michigan, United States

Outcomes

Primary Outcomes

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

Time frame: Month 2 to month 14

Secondary Outcomes

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

Time frame: Month 2 to month 14

Change in Treatment Satisfaction Questionnaire for Medication (TSQM)

TSQM V2 is a 11-item instrument designed to measure participants' satisfaction with medication within the past 2 to 3 weeks, or since their last medication use. Treatment satisfaction is assessed through side effects, effectiveness, convenience, and global satisfaction scale domains.

Time frame: Baseline to 14 months

Change in Hemophilia Activities List (HAL)

HAL is an instrument designed to measure and quantify a participant's perceived functional ability to perform activities in daily life. The HAL consists of 42 multiple choice questions within seven domains: lying/sitting/kneeling/standing, function of legs, function of arms, use of transportation, self-care, household tasks, and leisure activities.

Time frame: Baseline to 14 months

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

VERITAS-Pro is a 24-item questionnaire designed to measure treatment adherence through six sub-scales: Time, Dose, Plan, Remember, Skip, and Communicate. Each question is quantified on a 5-point Likert scale ("Always" to "Never") that correlates with a numeric score from 1 to 5, where higher scores indicate lower treatment adherence.

Time frame: Baseline to 14 months

Change in Wong-Baker Faces Pain Rating Scale

Wong-Baker Faces Scale is a 1-item questionnaire consisting of six facial caricatures, each depicting a different level of current perceived pain intensity, ranging from a score of 0 to 10. Higher scores indicate greater pain.

Data from ClinicalTrials.gov

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