NCT05044845

Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy

Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy Globally

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 150 (actual)
• Sponsor: St. Jude Children's Research Hospital · Academic / Other
• Sex: All
• Age: 12 Years
• Healthy volunteers: —

Summary

Gene therapy is a paradigm-shifting treatment for hemophilia B patients, particularly in resource-limited countries where factor availability remains low. Transparent and culturally sensitive communication around gene therapy is vital to the success of a high-quality consenting process. Current literature on knowledge, beliefs and attitudes about gene therapy in resource-limited countries is inadequate. In addition, few educational resources to explain basic gene therapy concepts exist in languages other than English. This study aims to address these gaps in knowledge and aid for the development of educational resources to assist the informed consent processes for gene therapy in resource-limited countries. Primary Objective: To assess baseline knowledge, beliefs, and attitudes about gene therapy held by hemophilia B patients globally Secondary Objectives: 1. To explore healthcare workers' (i.e., physicians, nurses, social workers, educators/academic coordinators) perspectives regarding the education needs of hemophilia B patients globally 2. To explore healthcare workers beliefs and attitudes about gene therapy 3. To identify preferences of patients with hemophilia B and their healthcare workers on how/by what method or pathway educational content should be provided.

Detailed description

This prospective cohort study will use qualitative methods, specifically semi structured interviews to evaluate knowledge, beliefs, and attitude about gene therapy in patients 12 years and older with a diagnosis of moderate and severe hemophilia B (≤2% FIX activity only), caregivers/parents and healthcare workers who care for them at St. Jude and other global countries. Participants will be interviewed virtually using an online video-conferencing platform, phone or in person. Interviews will be performed by experienced qualitative interviewers in the official language of each participating site. Interviews will last approximately 45-60 minutes and will be performed only once. All interviews will be audio recorded and obtained information will be analyzed using semantic content analysis to identify common themes.

Conditions

• Gene Therapy
• Hemophilia B

Interventions

Type	Name	Description
BEHAVIORAL	Interview	Semi structured interview will be conducted virtually using an online video-conferencing platform, phone or in person.

Timeline

• Start date: 2022-01-18
• Primary completion: 2025-10-14
• Completion: 2025-10-14
• First posted: 2021-09-16
• Last updated: 2025-11-14

Locations

6 sites across 6 countries: United States, Nepal, Peru, Sri Lanka, Thailand, Vietnam