Clinical trial NCT02392156: Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes
Official title: A Global, Multicenter, Observational Study Evaluating the Impact of rFVIIIFc and rFIXFc on Patient-reported Treatment Burden and Health Economic Outcomes
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Hemophilia A
Hemophilia B
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
Collaborators: name: Swedish Orphan Biovitrum
First posted: 2015-03-18
Enrollment: 3
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Participants will be recruited at various locations by prescribing physicians.

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.


Eligibility 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.

Primary outcomes:
measure: Annualized number of injections for prophylactic treatment with a Factor VIII or Factor IX replacement product
timeFrame: Month 2 to month 14
Secondary outcomes:
measure: The total annualized factor consumption (in International Units [IU] per kilogram [IU/kg]) calculated for each participant
timeFrame: Month 2 to month 14
measure: Change in Treatment Satisfaction Questionnaire for Medication (TSQM)
timeFrame: Baseline to 14 months
measure: Change in Hemophilia Activities List (HAL)
timeFrame: Baseline to 14 months
measure: Change in Validated Hemophilia Regimen Treatment Adherence Scale-Prophylaxis (VERITAS-Pro)
timeFrame: Baseline to 14 months
measure: Change in Wong-Baker Faces Pain Rating Scale
timeFrame: Baseline to 14 months
measure: Change in Work Productivity and Impairment Questionnaire plus Classroom Impairment Questions: Hemophilia Specific (WPAI+CIQ:HS)
timeFrame: Baseline to 14 months
measure: Change in Missed planned activity and productivity
timeFrame: Baseline to 14 months
measure: Change in Caregiver burden
timeFrame: Baseline to 14 months
measure: Number of inpatient and outpatient healthcare visits
timeFrame: Baseline to 14 months
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Research Site
state: California
city: East Lansing
country: United States
facility: Research Site
state: Michigan
Study officials:
affiliation: Bioverativ Therapeutics Inc.
name: Medical Director
role: STUDY_DIRECTOR
References: