Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes

Hemophilia A Clinical Trial

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Official title:

A Global, Multicenter, Observational Study Evaluating the Impact of rFVIIIFc and rFIXFc on Patient-reported Treatment Burden and Health Economic Outcomes

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02392156
• Other study ID #: 997HA401
• Status: Terminated
• Start date: July 2015
• Est. completion date: December 2015

Study information

• Verified date: August 2018
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 3
• Est. completion date: December 2015
• Est. primary completion date: December 2015
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Biological:
• rFVIIIFc
As described in the treatment arm
• rFIXFc
As described in the treatment arm

Drug:
• non-Fc FVIII replacement products
Standard plasma-derived (pd) or recombinant (r) FVIII concentrate and other traditional treatment options
• non-Fc FIX replacement products
Standard plasma-derived (pd) or recombinant (r) FIX concentrate and other traditional treatment options

Locations

Country	Name	City	State
United States	Research Site	East Lansing	Michigan
United States	Research Site	Los Angeles	California

Sponsors (2)

Lead Sponsor	Collaborator
Bioverativ Therapeutics Inc.	Swedish Orphan Biovitrum

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Annualized number of injections for prophylactic treatment with a Factor VIII or Factor IX replacement product		Month 2 to month 14
Secondary	The total annualized factor consumption (in International Units [IU] per kilogram [IU/kg]) calculated for each participant		Month 2 to month 14
Secondary	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.	Baseline to 14 months
Secondary	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.	Baseline to 14 months
Secondary	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.	Baseline to 14 months
Secondary	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.	Baseline to 14 months
Secondary	Change in Work Productivity and Impairment Questionnaire plus Classroom Impairment Questions: Hemophilia Specific (WPAI+CIQ:HS)	WPAI and classroom impairment questionnaire version is a 9-item questionnaire designed to measure hemophilia-related impairment in work, school, and activity level within the past 7 days. Visual analogue scales and fill-in responses are used to compute scores for absenteeism, impairment at work/school, losses in work/school productivity, and activity impairment based on scoring algorithms and presented as impairment percentages. Higher percentages indicate greater disease-related impairment	Baseline to 14 months
Secondary	Change in Missed planned activity and productivity	The purpose of these free standing questions are to capture any missed activities due to hemophilia that are not captured in other questionnaires included in the study, especially among children and adolescents.	Baseline to 14 months
Secondary	Change in Caregiver burden	The purpose of this survey is to assess how the intensive nature of caring for a hemophilia patient affects caregiver burden, providing scores on the following scales: Emotional burden, financial burden, lifestyle, social burden, and positive emotions. This will be completed by caregivers of children and adolescents younger than 18 years of age.	Baseline to 14 months
Secondary	Number of inpatient and outpatient healthcare visits	Number of hospitalizations and number of emergency room visits or number of physician outpatient clinic visits	Baseline to 14 months