GAS-Hem Feasibility Study

Brief Title

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

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.

Study Type

Observational

Primary Outcome

Change from baseline of the response to the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) outcomes tool

Secondary Outcome

 Change from baseline of Quality of life (QoL) outcomes: Medical Health Outcomes Study Health Survey Short Form-36 (SF-36)

Condition

Hemophilia A

Intervention

Factor VIII

Study Arms / Comparison Groups

 Study participants

Description:  Participants with Hemophila A

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Biological

Estimated Enrollment

Start Date

August 5, 2016

Completion Date

November 2, 2016

Primary Completion Date

November 2, 2016

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

Gender

All

Ages

5 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

Study Director, ,

Location Countries

Canada

Location Countries

Canada

Administrative Informations

NCT ID

NCT02674997

Organization ID

Responsible Party

Sponsor

Study Sponsor

Baxalta now part of Shire

Study Sponsor

Study Director, Study Director, Shire

Verification Date

April 2019