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

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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

  • United States
    • California
      • Los Angeles, California, United States, 90007
        • Research Site
    • Michigan
      • East Lansing, Michigan, United States, 48823
        • Research Site

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
rFVIIIFc for hemophilia A
Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label
Biological: rFVIIIFc
As described in the treatment arm
Other Names:
  • Eloctate
  • recombinant coagulation factor VIII Fc fusion protein
  • BIIB031
  • antihemophilic factor (recombinant) Fc fusion protein
  • efmoroctocog alfa
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
As described in the treatment arm
Other Names:
  • Eloctate
  • recombinant coagulation factor VIII Fc fusion protein
  • BIIB031
  • antihemophilic factor (recombinant) Fc fusion protein
  • efmoroctocog alfa
Drug: non-Fc FVIII replacement products
Standard plasma-derived (pd) or recombinant (r) FVIII concentrate and other traditional treatment options
Other Names:
  • rFVIII
  • pdFVIII
rFIXFc for hemophilia B
Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label
Biological: rFIXFc
As described in the treatment arm
Other Names:
  • Alprolix
  • BIIB029
  • coagulation factor IX (recombinant) Fc fusion protein
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
As described in the treatment arm
Other Names:
  • Alprolix
  • BIIB029
  • coagulation factor IX (recombinant) Fc fusion protein
Drug: non-Fc FIX replacement products
Standard plasma-derived (pd) or recombinant (r) FIX concentrate and other traditional treatment options
Other Names:
  • rFIX
  • pdFIX

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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
Month 2 to month 14

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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
Month 2 to month 14
Change in Treatment Satisfaction Questionnaire for Medication (TSQM)
Time Frame: Baseline to 14 months
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
Change in Hemophilia Activities List (HAL)
Time Frame: Baseline to 14 months
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
Change in Validated Hemophilia Regimen Treatment Adherence Scale-Prophylaxis (VERITAS-Pro)
Time Frame: Baseline to 14 months
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
Change in Wong-Baker Faces Pain Rating Scale
Time Frame: Baseline to 14 months
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
Change in Work Productivity and Impairment Questionnaire plus Classroom Impairment Questions: Hemophilia Specific (WPAI+CIQ:HS)
Time Frame: Baseline to 14 months
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
Change in Missed planned activity and productivity
Time Frame: Baseline to 14 months
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
Change in Caregiver burden
Time Frame: Baseline to 14 months
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
Number of inpatient and outpatient healthcare visits
Time Frame: Baseline to 14 months
Number of hospitalizations and number of emergency room visits or number of physician outpatient clinic visits
Baseline to 14 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Bioverativ Therapeutics Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Swedish Orphan Biovitrum

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2015

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2015

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 12, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 12, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 18, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 19, 2020

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 16, 2020

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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.

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