GAS-Hem Feasibility Study
GAS-Hem Feasibility Study
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.
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.
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