- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02392156
Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes (FORWARD)
December 16, 2020 updated by: Bioverativ Therapeutics Inc.
A Global, Multicenter, Observational Study Evaluating the Impact of rFVIIIFc and rFIXFc on Patient-reported Treatment Burden and Health Economic Outcomes
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 Overview
Status
Terminated
Conditions
Study Type
Observational
Enrollment (Actual)
3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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California
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Los Angeles, California, United States, 90007
- Research Site
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Michigan
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East Lansing, Michigan, United States, 48823
- Research Site
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Genders Eligible for Study
Male
Sampling Method
Probability Sample
Study Population
Participants will be recruited at various locations by prescribing physicians.
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
rFVIIIFc for hemophilia A
Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label
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As described in the treatment arm
Other Names:
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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
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As described in the treatment arm
Other Names:
Standard plasma-derived (pd) or recombinant (r) FVIII concentrate and other traditional treatment options
Other Names:
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rFIXFc for hemophilia B
Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label
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As described in the treatment arm
Other Names:
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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
|
As described in the treatment arm
Other Names:
Standard plasma-derived (pd) or recombinant (r) FIX concentrate and other traditional treatment options
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Annualized number of injections for prophylactic treatment with a Factor VIII or Factor IX replacement product
Time Frame: Month 2 to month 14
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Month 2 to month 14
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
The total annualized factor consumption (in International Units [IU] per kilogram [IU/kg]) calculated for each participant
Time Frame: Month 2 to month 14
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Month 2 to month 14
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Change in Treatment Satisfaction Questionnaire for Medication (TSQM)
Time Frame: Baseline to 14 months
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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.
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Baseline to 14 months
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Change in Hemophilia Activities List (HAL)
Time Frame: Baseline to 14 months
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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.
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Baseline to 14 months
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Change in Validated Hemophilia Regimen Treatment Adherence Scale-Prophylaxis (VERITAS-Pro)
Time Frame: Baseline to 14 months
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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.
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Baseline to 14 months
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Change in Wong-Baker Faces Pain Rating Scale
Time Frame: Baseline to 14 months
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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.
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Baseline to 14 months
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Change in Work Productivity and Impairment Questionnaire plus Classroom Impairment Questions: Hemophilia Specific (WPAI+CIQ:HS)
Time Frame: Baseline to 14 months
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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
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Baseline to 14 months
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Change in Missed planned activity and productivity
Time Frame: Baseline to 14 months
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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.
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Baseline to 14 months
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Change in Caregiver burden
Time Frame: Baseline to 14 months
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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.
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Baseline to 14 months
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Number of inpatient and outpatient healthcare visits
Time Frame: Baseline to 14 months
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Number of hospitalizations and number of emergency room visits or number of physician outpatient clinic visits
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Baseline to 14 months
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Study Director: Medical Director, Bioverativ Therapeutics Inc.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
July 1, 2015
Primary Completion (Actual)
December 1, 2015
Study Completion (Actual)
December 1, 2015
Study Registration Dates
First Submitted
March 12, 2015
First Submitted That Met QC Criteria
March 12, 2015
First Posted (Estimate)
March 18, 2015
Study Record Updates
Last Update Posted (Actual)
December 19, 2020
Last Update Submitted That Met QC Criteria
December 16, 2020
Last Verified
August 1, 2018
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 997HA401
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Hemophilia A
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Christoph KönigsRoche Pharma AG; Chugai Pharma Germany GmbHRecruitingSevere Hemophilia A | Severe Hemophilia A With Inhibitor | Severe Hemophilia A Without InhibitorGermany
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GWT-TUD GmbHHannover Medical School; Hoffmann-La RocheCompleted
-
Kathelijn FischerRadboud University Medical Center; University Medical Center Groningen; Maastricht... and other collaboratorsRecruitingAdolescent | Child | Hemophilia A With Inhibitor | Adult | Hemophilia A Without Inhibitor | Hemophilia A, SevereNetherlands
-
Hoffmann-La RocheActive, not recruitingSevere Hemophilia A | Moderate Hemophilia ABrazil, Germany, Italy, Spain, United States, Turkey, United Kingdom, Tunisia, Canada, Hungary, Morocco, Serbia
-
Catalyst BiosciencesCompletedHemophilia A | Hemophilia B | Hemophilia A With Inhibitor | Hemophilia B With Inhibitor | Hemophilia A Without Inhibitor | Hemophilia B Without InhibitorBulgaria, Russian Federation
-
JW PharmaceuticalRecruitingHemophilia A With Inhibitor | Hemophilia A Without InhibitorKorea, Republic of
-
PfizerCompletedFactor VIII Deficiency, Congenital | Hemophilia A, Congenital | Factor 8 Deficiency, Congenital | Autosomal Hemophilia A | Classic Hemophilia
-
BioMarin PharmaceuticalRecruitingHemophilia A With Inhibitor | Hemophilia A With Anti Factor VIIIUnited States, United Kingdom, Taiwan, Korea, Republic of, Brazil, Italy
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American Thrombosis and Hemostasis NetworkTakeda; CSL Behring; OctapharmaCompletedHemophilia A | Hemophilia B | Hemophilia | Hemophilia A With Inhibitor | Haemophilia | Hemophilia B With Inhibitor | Haemophilia A Without Inhibitor | Haemophilia B Without InhibitorUnited States
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BayerCompletedHemophilia A; Hemophilia BIsrael
Clinical Trials on rFVIIIFc
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Margaret RagniWithdrawn
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Swedish Orphan BiovitrumCerner EnvizaCompletedHemophilia A With InhibitorFrance, Ireland, Italy, Norway, Germany, Kuwait, Saudi Arabia, Switzerland
-
Swedish Orphan BiovitrumActive, not recruitingHemophilia ASpain, Germany, Czechia, Italy, Estonia, Finland, Greece, Netherlands, Saudi Arabia, Slovenia, Sweden, Switzerland, United Kingdom
-
Margaret RagniHealth Resources and Services Administration (HRSA)Terminated
-
Swedish Orphan BiovitrumCompletedHaemophilia A | Haemophilia BGermany
-
Swedish Orphan BiovitrumBioverativ Therapeutics Inc.CompletedHemophilia AIreland, United Kingdom, Germany, United States, Canada, Slovenia, Sweden
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Bioverativ Therapeutics Inc.CompletedSevere Hemophilia AUnited States, Hong Kong, Israel
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Bioverativ, a Sanofi companySwedish Orphan BiovitrumCompletedHemophilia A With InhibitorsUnited States, Spain, Canada, Belgium, France, Bulgaria, Italy, United Kingdom, Japan, Germany
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Margaret RagniHealth Resources and Services Administration (HRSA)Terminated
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Bioverativ Therapeutics Inc.Swedish Orphan BiovitrumCompletedSevere Hemophilia AUnited States, Australia, New Zealand