GAS-Hem Feasibility Study

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