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

Recruiting

• Conditions: Hemophilia B; Gene Therapy
• Interventions: Behavioral: Interview

• Registration Number: NCT05044845
• Lead Sponsor: St. Jude Children's Research Hospital

Brief 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.

Study Timeline

• Study First Submitted: 2021-09-09
• Study First Submitted QC: 2021-09-09
• Study First Posted: 2021-09-16
• Study Start: 2022-01-18
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-07
• Last Update Posted: 2025-04-09
• Primary Completion: 2025-12
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

• Patients ≥12 years of age
• Diagnosis of moderate (FIX ≥1% and ≤2%) or severe (<1%) hemophilia B
• Parents or caregivers to patients with hemophilia 12-17 years of age

Inclusion Criteria - Healthcare worker:

• Doctors, nurses, social workers, pharmacists and educators who participate in the care of hemophilia B patients

Exclusion Criteria

• Diagnosis of Hemophilia A
• Diagnosis of other non-Hemophilia B bleeding disorders

Exclusion Criteria - Healthcare worker:

• Health care workers who do not participate in the care of hemophilia B patients
• Healthcare worker who is conducting the interviews

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Hemophilia B patients	Interview	Patients ≥12 years of age with a diagnosis of moderate (FIX ≥1% and ≤2%) or severe (\<1%) hemophilia B
Parents or Caregivers	Interview	Parents or caregivers to patients with hemophilia 12-17 years of age
Healthcare Workers	Interview	Doctors, nurses, social workers, pharmacists and educators who participate in the care of hemophilia B patients

Primary Outcome Measures

Name	Time	Method
Use of semi-structured interviews to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B	Day 1, or at a future visit (up to approximately 1 year)	Semi-structured interviews will be performed in patients with hemophilia B, their caregivers and healthcare workers to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B including concerns, expectations, and best way to receive information about gene therapy. Interviews will be audio recorded, transcribed verbatim and analyzed using semantic content analysis to identify common themes.

Trial Locations

Locations (5)

St. Jude Children's Research Hospital; 🇺🇸 Memphis, Tennessee, United States

Hospital Dos De Mayo; 🇵🇪 Lima 15003, Peru

National Hospital of Sri Lanka; 🇱🇰 Colombo 10, Sri Lanka

Ramathibodi Hospital; 🇹🇭 Bangkok, Thailand

National Institute of Hematology and Blood Transfusion; 🇻🇳 Hanoi, Vietnam