GAS-Hem Feasibility Study

NCT02674997 | Completed

• 1 other identifier: 001501
• Study Type: observational
• Target: 44
• Locations: 2 countries
• Sites: 4

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.

Conditions

Hemophilia A

Keywords

Goal setting; Feasibility; Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem); Patient reported outcome (PRO); Goal attainment scaling (GAS)

Outcome Measures

Primary Outcomes (3)

• 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

• 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

• 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 Outcomes (5)

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

  Baseline; weeks 6 and 12

• Change from baseline of Quality of life (QoL) outcomes: Pediatric Quality of Life Inventory Version 4.0 Generic Core Scale (PedsQL)

  Baseline; weeks 6 and 12

• Participant perception of the GAS-Hem as a useful means of identifying and monitoring change in relevant goal areas

  Week 12

• Clinician perception of the GAS-Hem as a useful means of monitoring clinical progress in patient identified goal areas

  Week 12

• Number and Location of Bleeds

  Within six months prior starting study through week 12 in the study

Study Arms (1)

Study participants

Participants with Hemophila A

Biological: Factor VIII

Interventions

Factor VIII BIOLOGICAL

Licensed Factor VIII products. Study is brand agnostic

Also known as: FVIII

Study participants

Eligibility Criteria

• Age: 5 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with moderate to severe hemophilia A in the United States or Canada.

You may qualify if:

• Participant has a documented diagnosis of hemophilia A
• Participant has documented clotting factor levels of 5% or less
• 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"
• Participant is willing and able to comply with the requirements of the protocol
• Participant is proficient in the English language to allow for use of the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) tool.

You may not qualify if:

• 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
• Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease)
• 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
• Participant is a family member or employee of the investigator

Sponsors & Collaborators

• Baxalta now part of Shire: lead

Study Sites (4)

Bleeding and Clotting Disorders Institute

Peoria, Illinois, 61615, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

St. Paul's Hospital

Vancouver, British Columbia, V6Z 1Y6, Canada

Location

QEII Health Sciences Centre

Halifax, Nova Scotia, B3H 3G1, Canada

Location

Related Links

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

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2015
• First Posted: February 5, 2016
• Study Start: August 5, 2016
• Primary Completion: November 2, 2016
• Study Completion: November 2, 2016
• Last Updated: March 17, 2021

Record last verified: 2021-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

CA (2); US (2)