GAS-Hem Feasibility Study

Hemophilia A Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02674997
• Other study ID #: 001501
• Status: Completed
• Start date: August 5, 2016
• Est. completion date: November 2, 2016

Study information

• Verified date: March 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 44
• Est. completion date: November 2, 2016
• Est. primary completion date: November 2, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• Factor VIII
Licensed Factor VIII products. Study is brand agnostic

Locations

Country	Name	City	State
Canada	QEII Health Sciences Centre	Halifax	Nova Scotia
Canada	St. Paul's Hospital	Vancouver	British Columbia
United States	Bleeding and Clotting Disorders Institute	Peoria	Illinois
United States	Oregon Health & Science University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Baxalta now part of Shire

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline of the response to the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) outcomes tool	The GAS-Hem is an online instrument for setting and tracking personalized goals related to hemophilia. The GAS-Hem uses a 5-point scale to record the degree of attainment in relation to individually defined goal areas between an established baseline (scaled as -1) and follow-up time points (here, 6 weeks and 12 weeks). The scale range is +2 (much better outcome) to -2 (much worse outcome), where 0 represents the desired outcome. The process includes a facilitator to ensure that the outcome descriptions for the scale levels are reasonably attainable (not too hard or too easy) and represent roughly equivalent change between levels (ie scores). The overall scoring formula results in a summary score of 50 when all goals are attained (individual goal attainment = 0). Scores >50 indicate generally better than expected outcomes in goal attainment. Scores <50 indicate that, overall, goals were not achieved. The overall total scores range is dependent on the average number of goals.	Baseline; weeks 6 and 12
Primary	Change from baseline of the measurement of the responsiveness (sensitivity to change) of GAS-Hem compared with the SF-36	Comparison of outcomes on the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) with the Medical Health Outcomes Study Health Survey Short Form-36 (SF-36) measures. The analysis will compare effect sizes calculated for the GAS-Hem and the SF-36 using Cohen's d and standardized response means.	Baseline; weeks 6 and 12
Primary	Change from baseline of the measurement of the responsiveness (sensitivity to change) of GAS-Hem compared with the PedQL	Comparison of outcomes on the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) with the Pediatric Quality of Life Inventory (PedsQL) measures. The analysis will compare effect sizes calculated for the GAS-Hem and the PedsQL using Cohen's d and standardized response means.	Baseline; weeks 6 and 12
Secondary	Change from baseline of Quality of life (QoL) outcomes: Medical Health Outcomes Study Health Survey Short Form-36 (SF-36)	The SF-36 is a 36 item survey of physical functioning, role limitations (both physical and emotional), bodily pain, general health vitality, social functioning and mental health administered to adults aged 19 and older. The instrument measure health related quality of life on a scale of 0 (lowest level) to 100 (highest level).	Baseline; weeks 6 and 12
Secondary	Change from baseline of Quality of life (QoL) outcomes: Pediatric Quality of Life Inventory Version 4.0 Generic Core Scale (PedsQL)	The PedsQL is a 23 item survey that assesses physical functioning, emotional functioning, social functioning, and school functioning in children and adolescents aged 5 to 18. The PedsQL measures health related quality of life on a scale of 0 (lowest quality of life) to 100 (highest quality of life).	Baseline; weeks 6 and 12
Secondary	Participant perception of the GAS-Hem as a useful means of identifying and monitoring change in relevant goal areas	Measured using a questionnaire to test acceptability and usefulness of Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) to patients, and where applicable to their parents or guardians.	Week 12
Secondary	Clinician perception of the GAS-Hem as a useful means of monitoring clinical progress in patient identified goal areas	Measured using a questionnaire for clinicians designed to test their perception of the usefulness of Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) for monitoring clinical progress.	Week 12
Secondary	Number and Location of Bleeds	History of bleeding episodes in the six months prior to signing informed consent will be collected. Participants will record details of each subsequent bleeding episode in the participant diary during the study.	Within six months prior starting study through week 12 in the study

External Links

•   To obtain more information on the study, click here/on this link.