Hemophilia ANCT02674997
GAS-Hem Feasibility Study
An observational study of Hemophilia A, run by Baxalta now part of Shire at 4 sites in 2 countries.
Trial summary
The purpose of this study is to investigate the feasibility and acceptability of the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) as a patient reported outcome (PRO) measure to monitor clinical progress in participant-identified goal areas in individuals with hemophilia A. Read more about this study
- Who is it for?
- Ages 5 to 65 years
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Factor VIII
- How many people
- 44 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 5 Aug 2016to 2 Nov 2016
- 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 5 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with moderate to severe hemophilia A in the United States or Canada.
Who may be able to join
- Participant has a documented diagnosis of hemophilia A
- Participant has documented clotting factor levels of 5% or less
- Participant is on a prescribed regimen of continuous prophylaxis. Continuous prophylaxis is defined by the World Federation of Hemophilia as "… the intent of treating for 52 weeks a year and receiving a minimum of an a priori defined frequency of infusions for at least 45 weeks (85%) of the year under consideration"
- Participant is willing and able to comply with the requirements of the protocol
- Participant is proficient in the English language to allow for use of the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) tool.
Who cannot join
- Participants with an active Factor VIII (FVIII) inhibitory antibody (≥ 0.4 Bethesda units (BU) using the Nijmegen modification of the Bethesda assay or ≥ 0.6 BU using the Bethesda assay) at any time prior to screening
- Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease)
- Participant has participated in a clinical study involving a medicinal product or device within 30 days prior to enrollment or is scheduled to participate in a clinical study involving a medical product or device during the course of this study
- Participant is a family member or employee of the investigator
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
United States2 sites
Bleeding and Clotting Disorders Institute
Peoria, Illinois, 61615
Oregon Health & Science University
Portland, Oregon, 97239
Canada2 sites
QEII Health Sciences Centre
Halifax, Nova Scotia, B3H 3G1
St. Paul's Hospital
Vancouver, British Columbia, V6Z 1Y6
About this study
Official title: A New Patient-centric Outcome Measure for Patients With Hemophilia: Testing the Feasibility of GAS-Hem in Pediatric, Adolescent and Adult Hemophilia A Patients
The purpose of this study is to investigate the feasibility and acceptability of the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) as a patient reported outcome (PRO) measure to monitor clinical progress in participant-identified goal areas in individuals with hemophilia A.
Read the detailed description
Hemophilia A is an X-linked recessive, congenital bleeding disorder caused by deficient or defective coagulation factor VIII (FVIII). The absence of FVIII leads to 'spontaneous' bleeding episodes (occurring primarily in joints, muscles and, less commonly, in soft tissues) and to excessive bleeding following trauma or injury. With proper treatment (FVIII) and self-care, most hemophilia patients can maintain an active, productive lifestyle. With a growing appreciation that a low bleed rate sometimes is only achieved, in part, by patients limiting their activities, there has been growing interest in understanding how hemophilia may still limit life despite absence of major bleeds. The current study will introduce and test a standardized method for individualized outcome measurement based on Goal Attainment Scaling (GAS), a method that allows participants to set goals to address challenges that are meaningful to them and monitor attainment in those areas over time. The Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) is an online instrument for setting and tracking personalized goals related to hemophilia. The goal menu captures common challenges faced by people with hemophilia, accompanied by an inventory of descriptors that can be personalized. The tool can be used to track the health of individuals across a wide spectrum of ages, life experiences and health states. This is a 12-week, multicenter, prospective, observational, feasibility study of up to 60 people (aged 5 and 65) with moderate to severe hemophilia A to investigate the feasibility and acceptability of GAS-Hem as a patient reported outcome (PRO) measure to monitor clinical progress in participant-identified goal areas. Outcomes, including GAS-Hem (primary), health-related quality of life, bleeding episodes (location, cause, time) and treatment (time, dose and reason for infusion) will be assessed at screen/baseline, with follow-up at 6 and 12 weeks. Endpoints of interest include participant GAS-Hem outcomes at 6 and 12 weeks, participant quality of life (QoL) outcomes at 6 and 12 weeks, as well as participant and clinician perception of the GAS-Hem as a useful means of identifying and monitoring progress in relevant goal areas.
- Conditions
- Hemophilia A
- Being tested
- Factor VIII
- Keywords
- Goal setting, Feasibility, Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem), Patient reported outcome (PRO), Goal attainment scaling (GAS)
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Factor VIIIBiological
Also called FVIII
Licensed Factor VIII products. Study is brand agnostic
| Study group | Role | What they receive |
|---|---|---|
| Study participants | Participants with Hemophila A Receives: Biological: Factor VIII |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline of the response to the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) outcomes tool
The GAS-Hem is an online instrument for setting and tracking personalized goals related to hemophilia. The GAS-Hem uses a 5-point scale to record the degree of attainment in relation to individually defined goal areas between an established baseline (scaled as -1) and follow-up time points (here, 6 weeks and 12 weeks). The scale range is +2 (much better outcome) to -2 (much worse outcome), where 0 represents the desired outcome. The process includes a facilitator to ensure that the outcome descriptions for the scale levels are reasonably attainable (not too hard or too easy) and represent roughly equivalent change between levels (ie scores). The overall scoring formula results in a summary score of 50 when all goals are attained (individual goal attainment = 0). Scores >50 indicate generally better than expected outcomes in goal attainment. Scores <50 indicate that, overall, goals were not achieved. The overall total scores range is dependent on the average number of goals.
Measured: Baseline; weeks 6 and 12
Change from baseline of the measurement of the responsiveness (sensitivity to change) of GAS-Hem compared with the SF-36
Comparison of outcomes on the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) with the Medical Health Outcomes Study Health Survey Short Form-36 (SF-36) measures. The analysis will compare effect sizes calculated for the GAS-Hem and the SF-36 using Cohen's d and standardized response means.
Measured: Baseline; weeks 6 and 12
Change from baseline of the measurement of the responsiveness (sensitivity to change) of GAS-Hem compared with the PedQL
Comparison of outcomes on the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) with the Pediatric Quality of Life Inventory (PedsQL) measures. The analysis will compare effect sizes calculated for the GAS-Hem and the PedsQL using Cohen's d and standardized response means.
Measured: Baseline; weeks 6 and 12
Secondary outcomes (5)
Change from baseline of Quality of life (QoL) outcomes: Medical Health Outcomes Study Health Survey Short Form-36 (SF-36)
The SF-36 is a 36 item survey of physical functioning, role limitations (both physical and emotional), bodily pain, general health vitality, social functioning and mental health administered to adults aged 19 and older. The instrument measure health related quality of life on a scale of 0 (lowest level) to 100 (highest level).
Measured: Baseline; weeks 6 and 12
Change from baseline of Quality of life (QoL) outcomes: Pediatric Quality of Life Inventory Version 4.0 Generic Core Scale (PedsQL)
The PedsQL is a 23 item survey that assesses physical functioning, emotional functioning, social functioning, and school functioning in children and adolescents aged 5 to 18. The PedsQL measures health related quality of life on a scale of 0 (lowest quality of life) to 100 (highest quality of life).
Measured: Baseline; weeks 6 and 12
Participant perception of the GAS-Hem as a useful means of identifying and monitoring change in relevant goal areas
Measured using a questionnaire to test acceptability and usefulness of Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) to patients, and where applicable to their parents or guardians.
Measured: Week 12
Clinician perception of the GAS-Hem as a useful means of monitoring clinical progress in patient identified goal areas
Measured using a questionnaire for clinicians designed to test their perception of the usefulness of Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) for monitoring clinical progress.
Measured: Week 12
Number and Location of Bleeds
History of bleeding episodes in the six months prior to signing informed consent will be collected. Participants will record details of each subsequent bleeding episode in the participant diary during the study.
Measured: Within six months prior starting study through week 12 in the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2016 (estimated)
- Study start5 Aug 2016
- Primary completion2 Nov 2016
- Study completion2 Nov 2016
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
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02674997
- Sponsor's protocol ID
- 001501
- Status last verified
- Mar 2021
- First submitted
- 24 Nov 2015
- Last updated
- 17 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A New Patient-centric Outcome Measure for Patients With Hemophilia: Testing the Feasibility of GAS-Hem in Pediatric, Adolescent and Adult Hemophilia A Patients
- ClinicalTrials.gov ID
- NCT02674997
- Acronym / sponsor ID
- 001501
- Conditions
- Hemophilia A
- Keywords
- Goal setting, Feasibility, Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem), Patient reported outcome (PRO), Goal attainment scaling (GAS)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 5 Aug 2016 (actual)
- Primary completion
- 2 Nov 2016 (actual)
- Study completion
- 2 Nov 2016 (actual)
- First posted
- 5 Feb 2016 (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 purpose of this study is to investigate the feasibility and acceptability of the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) as a patient reported outcome (PRO) measure to monitor clinical progress in participant-identified goal areas in individuals with hemophilia A.
Detailed description
Hemophilia A is an X-linked recessive, congenital bleeding disorder caused by deficient or defective coagulation factor VIII (FVIII). The absence of FVIII leads to 'spontaneous' bleeding episodes (occurring primarily in joints, muscles and, less commonly, in soft tissues) and to excessive bleeding following trauma or injury. With proper treatment (FVIII) and self-care, most hemophilia patients can maintain an active, productive lifestyle. With a growing appreciation that a low bleed rate sometimes is only achieved, in part, by patients limiting their activities, there has been growing interest in understanding how hemophilia may still limit life despite absence of major bleeds. The current study will introduce and test a standardized method for individualized outcome measurement based on Goal Attainment Scaling (GAS), a method that allows participants to set goals to address challenges that are meaningful to them and monitor attainment in those areas over time. The Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) is an online instrument for setting and tracking personalized goals related to hemophilia. The goal menu captures common challenges faced by people with hemophilia, accompanied by an inventory of descriptors that can be personalized. The tool can be used to track the health of individuals across a wide spectrum of ages, life experiences and health states. This is a 12-week, multicenter, prospective, observational, feasibility study of up to 60 people (aged 5 and 65) with moderate to severe hemophilia A to investigate the feasibility and acceptability of GAS-Hem as a patient reported outcome (PRO) measure to monitor clinical progress in participant-identified goal areas. Outcomes, including GAS-Hem (primary), health-related quality of life, bleeding episodes (location, cause, time) and treatment (time, dose and reason for infusion) will be assessed at screen/baseline, with follow-up at 6 and 12 weeks. Endpoints of interest include participant GAS-Hem outcomes at 6 and 12 weeks, participant quality of life (QoL) outcomes at 6 and 12 weeks, as well as participant and clinician perception of the GAS-Hem as a useful means of identifying and monitoring progress in relevant goal areas.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Study participants | – | Biological: Factor VIIIParticipants with Hemophila A |
Who may qualify
- Ages
- 5 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with moderate to severe hemophilia A in the United States or Canada.
Key inclusion criteria (5 listed)
- Participant has a documented diagnosis of hemophilia A
- Participant has documented clotting factor levels of 5% or less
- Participant is on a prescribed regimen of continuous prophylaxis. Continuous prophylaxis is defined by the World Federation of Hemophilia as "… the intent of treating for 52 weeks a year and receiving a minimum of an a priori defined frequency of infusions for at least 45 weeks (85%) of the year under consideration"
- Participant is willing and able to comply with the requirements of the protocol
- Participant is proficient in the English language to allow for use of the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) tool.
Key exclusion criteria (4 listed)
- Participants with an active Factor VIII (FVIII) inhibitory antibody (≥ 0.4 Bethesda units (BU) using the Nijmegen modification of the Bethesda assay or ≥ 0.6 BU using the Bethesda assay) at any time prior to screening
- Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease)
- Participant has participated in a clinical study involving a medicinal product or device within 30 days prior to enrollment or is scheduled to participate in a clinical study involving a medical product or device during the course of this study
- Participant is a family member or employee of the investigator
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 |
|---|---|---|
| Factor VIIIAlso: FVIIILicensed Factor VIII products. Study is brand agnostic | Biological | Study participants |
Primary outcome
Change from baseline of the response to the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) outcomes tool
Time frame: Baseline; weeks 6 and 12
The GAS-Hem is an online instrument for setting and tracking personalized goals related to hemophilia. The GAS-Hem uses a 5-point scale to record the degree of attainment in relation to individually defined goal areas between an established baseline (scaled as -1) and follow-up time points (here, 6 weeks and 12 weeks). The scale range is +2 (much better outcome) to -2 (much worse outcome), where 0 represents the desired outcome. The process includes a facilitator to ensure that the outcome descriptions for the scale levels are reasonably attainable (not too hard or too easy) and represent roughly equivalent change between levels (ie scores). The overall scoring formula results in a summary score of 50 when all goals are attained (individual goal attainment = 0). Scores >50 indicate generally better than expected outcomes in goal attainment. Scores <50 indicate that, overall, goals were not achieved. The overall total scores range is dependent on the average number of goals.
Change from baseline of the measurement of the responsiveness (sensitivity to change) of GAS-Hem compared with the SF-36
Time frame: Baseline; weeks 6 and 12
Comparison of outcomes on the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) with the Medical Health Outcomes Study Health Survey Short Form-36 (SF-36) measures. The analysis will compare effect sizes calculated for the GAS-Hem and the SF-36 using Cohen's d and standardized response means.
Change from baseline of the measurement of the responsiveness (sensitivity to change) of GAS-Hem compared with the PedQL
Time frame: Baseline; weeks 6 and 12
Comparison of outcomes on the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) with the Pediatric Quality of Life Inventory (PedsQL) measures. The analysis will compare effect sizes calculated for the GAS-Hem and the PedsQL using Cohen's d and standardized response means.
5 secondary outcomes
Change from baseline of Quality of life (QoL) outcomes: Medical Health Outcomes Study Health Survey Short Form-36 (SF-36)
Time frame: Baseline; weeks 6 and 12
Change from baseline of Quality of life (QoL) outcomes: Pediatric Quality of Life Inventory Version 4.0 Generic Core Scale (PedsQL)
Time frame: Baseline; weeks 6 and 12
Participant perception of the GAS-Hem as a useful means of identifying and monitoring change in relevant goal areas
Time frame: Week 12
Clinician perception of the GAS-Hem as a useful means of monitoring clinical progress in patient identified goal areas
Time frame: Week 12
Number and Location of Bleeds
Time frame: Within six months prior starting study through week 12 in the study
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2021.
- To obtain more information on the study, click here/on this link.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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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.
Study Director study director
Takeda
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Baxalta now part of Shire at 4 sites in 2 countries.
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 5 inclusion and 4 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: finished, as of the sponsor's last update on 17 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Canada. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Change from baseline of the response to the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) outcomes tool (time frame: Baseline; weeks 6 and 12). The study lists 3 primary outcomes in all. 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 17 Mar 2021.