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

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.

Study Overview

• Status: Completed
• Conditions: Hemophilia B; Gene Therapy
• Intervention / Treatment: Behavioral: Interview

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 Type: Observational
• Enrollment (Actual): 150

Contacts and Locations

Study Locations

• Nepal
  • Kathmandu, Nepal
    • Civil Service Hospital
• Peru
  • Lima, Peru
    • Hospital Dos De Mayo
• Sri Lanka
  • Colombo, Sri Lanka, 01000
    • National Hospital of Sri Lanka
• Thailand
  • Bangkok, Thailand, 10400
    • Ramathibodi Hospital
• United States
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • St. Jude Children's Research Hospital
• Vietnam
  • Hanoi, Vietnam
    • National Institute of Hematology and Blood Transfusion

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

Participants who meet the eligibility criteria

Description

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 Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Hemophilia B patients; Patients ≥12 years of age with a diagnosis of moderate (FIX ≥1% and ≤2%) or severe (<1%) hemophilia B	Behavioral: Interview; Semi structured interview will be conducted virtually using an online video-conferencing platform, phone or in person.; Other Names: Questions
Parents or Caregivers; Parents or caregivers to patients with hemophilia 12-17 years of age	Behavioral: Interview; Semi structured interview will be conducted virtually using an online video-conferencing platform, phone or in person.; Other Names: Questions
Healthcare Workers; Doctors, nurses, social workers, pharmacists and educators who participate in the care of hemophilia B patients	Behavioral: Interview; Semi structured interview will be conducted virtually using an online video-conferencing platform, phone or in person.; Other Names: Questions

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Use of semi-structured interviews to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B	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.	Day 1, or at a future visit (up to approximately 1 year)

Collaborators and Investigators

• Sponsor: St. Jude Children's Research Hospital
• Investigators: Principal Investigator: Nidhi Bhatt, MD, St. Jude Children's Research Hospital

Publications and helpful links

Helpful Links

• St. Jude Children's Research Hospital
• ClinicalTrials Open at St. Jude

Study record dates

Study Major Dates

• Study Start (Actual): January 18, 2022
• Primary Completion (Actual): October 14, 2025
• Study Completion (Actual): October 14, 2025

Study Registration Dates

• First Submitted: September 9, 2021
• First Submitted That Met QC Criteria: September 9, 2021
• First Posted (Actual): September 16, 2021

Study Record Updates

• Last Update Posted (Estimated): November 14, 2025
• Last Update Submitted That Met QC Criteria: November 13, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Genetic Diseases, X-Linked; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Hemophilia B; Health Care Quality, Access, and Evaluation; Investigative Techniques; Epidemiologic Methods; Data Collection; Health Care Evaluation Mechanisms; Quality of Health Care; Public Health; Environment and Public Health; Interviews as Topic
• Other Study ID Numbers: BINFORMED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No