Clinical trial NCT02674997: GAS-Hem Feasibility 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Keywords: Goal setting
Feasibility
Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem)
Patient reported outcome (PRO)
Goal attainment scaling (GAS)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2016-02-05
Enrollment: 44
Sex: ALL
Minimum age (years): 5
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with moderate to severe hemophilia A in the United States or Canada.

Brief 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.

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.

Eligibility criteria: Inclusion Criteria:

1. Participant has a documented diagnosis of hemophilia A
2. Participant has documented clotting factor levels of 5% or less
3. 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"
4. Participant is willing and able to comply with the requirements of the protocol
5. Participant is proficient in the English language to allow for use of the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) tool.

Exclusion Criteria:

1. 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
2. Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease)
3. 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
4. Participant is a family member or employee of the investigator

Primary outcomes:
measure: Change from baseline of the response to the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) outcomes tool
timeFrame: Baseline; weeks 6 and 12
measure: Change from baseline of the measurement of the responsiveness (sensitivity to change) of GAS-Hem compared with the SF-36
timeFrame: Baseline; weeks 6 and 12
measure: Change from baseline of the measurement of the responsiveness (sensitivity to change) of GAS-Hem compared with the PedQL
timeFrame: Baseline; weeks 6 and 12
Secondary outcomes:
measure: Change from baseline of Quality of life (QoL) outcomes: Medical Health Outcomes Study Health Survey Short Form-36 (SF-36)
timeFrame: Baseline; weeks 6 and 12
measure: Change from baseline of Quality of life (QoL) outcomes: Pediatric Quality of Life Inventory Version 4.0 Generic Core Scale (PedsQL)
timeFrame: Baseline; weeks 6 and 12
measure: Participant perception of the GAS-Hem as a useful means of identifying and monitoring change in relevant goal areas
timeFrame: Week 12
measure: Clinician perception of the GAS-Hem as a useful means of monitoring clinical progress in patient identified goal areas
timeFrame: Week 12
measure: Number and Location of Bleeds
timeFrame: Within six months prior starting study through week 12 in the study
Other outcomes:
Locations:
city: Peoria
country: United States
facility: Bleeding and Clotting Disorders Institute
state: Illinois
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Vancouver
country: Canada
facility: St. Paul's Hospital
state: British Columbia
city: Halifax
country: Canada
facility: QEII Health Sciences Centre
state: Nova Scotia
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: