Hemophilia AFORWARDNCT02392156
Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes
An observational study of Hemophilia A and Hemophilia B, run by Bioverativ Therapeutics Inc. at 2 sites in United States.
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… Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Hemophilia A, Hemophilia B
- What is being tested?
- rFVIIIFc, rFIXFc and non-Fc FVIII replacement products
- How many people
- 3 participants
- Where
- 2 sitesUnited States
- Study dates
- Jul 2015to Dec 2015
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants will be recruited at various locations by prescribing physicians.
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Research Site
East Lansing, Michigan, 48823
Research Site
Los Angeles, California, 90007
About this study
Official title: 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.
- Conditions
- Hemophilia A, Hemophilia B
- Being tested
- rFVIIIFc, rFIXFc, non-Fc FVIII replacement products, non-Fc FIX replacement products
- Medical subject headings
- Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rFVIIIFcBiological
Also called antihemophilic factor (recombinant) Fc fusion protein, Eloctate, recombinant coagulation factor VIII Fc fusion protein, efmoroctocog alfa
As described in the treatment arm
rFIXFcBiological
Also called coagulation factor IX (recombinant) Fc fusion protein, Alprolix, BIIB029
As described in the treatment arm
non-Fc FVIII replacement productsDrug
Also called rFVIII, pdFVIII
Standard plasma-derived (pd) or recombinant (r) FVIII concentrate and other traditional treatment options
non-Fc FIX replacement productsDrug
Also called rFIX, pdFIX
Standard plasma-derived (pd) or recombinant (r) FIX concentrate and other traditional treatment options
| Study group | Role | What they receive |
|---|---|---|
| rFVIIIFc for hemophilia A | Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label Receives: Biological: rFVIIIFc | |
| 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 Receives: Biological: rFVIIIFc; Drug: non-Fc FVIII replacement products | |
| rFIXFc for hemophilia B | Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label Receives: Biological: rFIXFc | |
| 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 Receives: Biological: rFIXFc; Drug: non-Fc FIX replacement products |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized number of injections for prophylactic treatment with a Factor VIII or Factor IX replacement product
Measured: Month 2 to month 14
Secondary outcomes (9)
The total annualized factor consumption (in International Units [IU] per kilogram [IU/kg]) calculated for each participant
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline to 14 months
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
Measured: Baseline to 14 months
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.
Measured: Baseline to 14 months
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.
Measured: Baseline to 14 months
Number of inpatient and outpatient healthcare visits
Number of hospitalizations and number of emergency room visits or number of physician outpatient clinic visits
Measured: Baseline to 14 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2015 (estimated)
- Study startJul 2015
- Primary completionDec 2015
- Study completionDec 2015
Why it stopped: Study terminated due to recruitment challenges.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioverativ Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Swedish Orphan Biovitrum
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Bioverativ Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02392156
- Sponsor's protocol ID
- 997HA401
- Acronym
- FORWARD
- Status last verified
- Aug 2018
- First submitted
- 12 Mar 2015
- Last updated
- 19 Dec 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Global, Multicenter, Observational Study Evaluating the Impact of rFVIIIFc and rFIXFc on Patient-reported Treatment Burden and Health Economic Outcomes
- ClinicalTrials.gov ID
- NCT02392156
- Acronym / sponsor ID
- FORWARD / 997HA401
- Conditions
- Hemophilia A, Hemophilia B
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Study terminated due to recruitment challenges.
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Bioverativ Therapeutics Inc.Industry (pharma, biotech, devices)
- Collaborators
- Swedish Orphan Biovitrum
- Study start
- Jul 2015
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 18 Mar 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| rFVIIIFc for hemophilia A | – | Biological: rFVIIIFcAdministered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label |
| non-Fc (fusion protein) replacement products for hemophilia A | – | Biological: rFVIIIFc, Drug: non-Fc FVIII replacement productsAdministered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label |
| rFIXFc for hemophilia B | – | Biological: rFIXFcAdministered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label |
| non-Fc factor replacement products for hemophilia B | – | Biological: rFIXFc, Drug: non-Fc FIX replacement productsAdministered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants will be recruited at various locations by prescribing physicians.
Key inclusion criteria (4 listed)
- 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 (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rFVIIIFcAlso: antihemophilic factor (recombinant) Fc fusion protein, Eloctate, recombinant coagulation factor VIII Fc fusion protein, efmoroctocog alfaAs described in the treatment arm | Biological | non-Fc (fusion protein) replacement products for hemophilia A, rFVIIIFc for hemophilia A |
| rFIXFcAlso: coagulation factor IX (recombinant) Fc fusion protein, Alprolix, BIIB029As described in the treatment arm | Biological | non-Fc factor replacement products for hemophilia B, rFIXFc for hemophilia B |
| non-Fc FVIII replacement productsAlso: rFVIII, pdFVIIIStandard plasma-derived (pd) or recombinant (r) FVIII concentrate and other traditional treatment options | Drug | non-Fc (fusion protein) replacement products for hemophilia A |
| non-Fc FIX replacement productsAlso: rFIX, pdFIXStandard plasma-derived (pd) or recombinant (r) FIX concentrate and other traditional treatment options | Drug | non-Fc factor replacement products for hemophilia B |
Primary outcome
Annualized number of injections for prophylactic treatment with a Factor VIII or Factor IX replacement product
Time frame: Month 2 to month 14
9 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)
Time frame: Baseline to 14 months
Change in Hemophilia Activities List (HAL)
Time frame: Baseline to 14 months
Change in Validated Hemophilia Regimen Treatment Adherence Scale-Prophylaxis (VERITAS-Pro)
Time frame: Baseline to 14 months
Change in Wong-Baker Faces Pain Rating Scale
Time frame: 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
Change in Missed planned activity and productivity
Time frame: Baseline to 14 months
Change in Caregiver burden
Time frame: Baseline to 14 months
Number of inpatient and outpatient healthcare visits
Time frame: Baseline to 14 months
Results and safety
The study was stopped early (reason given: Study terminated due to recruitment challenges.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Dec 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Bioverativ Therapeutics Inc.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A and Hemophilia B, run by Bioverativ Therapeutics Inc. at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 19 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Study terminated due to recruitment challenges.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized number of injections for prophylactic treatment with a Factor VIII or Factor IX replacement product (time frame: Month 2 to month 14). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Dec 2020.