NCT02674997

GAS-Hem Feasibility Study

A New Patient-centric Outcome Measure for Patients With Hemophilia: Testing the Feasibility of GAS-Hem in Pediatric, Adolescent and Adult Hemophilia A Patients

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 44 (actual)
• Sponsor: Baxalta now part of Shire · Industry
• Sex: All
• Age: 5 Years – 65 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
BIOLOGICAL	Factor VIII	Licensed Factor VIII products. Study is brand agnostic

Timeline

• Start date: 2016-08-05
• Primary completion: 2016-11-02
• Completion: 2016-11-02
• First posted: 2016-02-05
• Last updated: 2021-03-17

Locations

4 sites across 2 countries: United States, Canada