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

NCT05044845 | Completed

• 1 other identifier: BINFORMED
• Study Type: observational
• Target: 150
• Locations: 6 countries
• Sites: 6

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. 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. 2.To explore healthcare workers beliefs and attitudes about gene therapy
3. 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.

Conditions

Gene Therapy; Hemophilia B

Outcome Measures

Primary Outcomes (1)

• 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)

Study Arms (3)

Hemophilia B patients

Patients ≥12 years of age with a diagnosis of moderate (FIX ≥1% and ≤2%) or severe (\<1%) hemophilia B

Behavioral: Interview

Parents or Caregivers

Parents or caregivers to patients with hemophilia 12-17 years of age

Behavioral: Interview

Healthcare Workers

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

Behavioral: Interview

Interventions

Interview BEHAVIORAL

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

Also known as: Questions

Healthcare Workers; Hemophilia B patients; Parents or Caregivers

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants who meet the eligibility criteria

You may qualify if:

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

You may not qualify if:

• Diagnosis of Hemophilia A
• Diagnosis of other non-Hemophilia B bleeding disorders
• Health care workers who do not participate in the care of hemophilia B patients
• Healthcare worker who is conducting the interviews

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (6)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Civil Service Hospital

Kathmandu, Nepal

Location

Hospital Dos De Mayo

Lima, Peru

Location

National Hospital of Sri Lanka

Colombo, 01000, Sri Lanka

Location

Ramathibodi Hospital

Bangkok, 10400, Thailand

Location

National Institute of Hematology and Blood Transfusion

Hanoi, Vietnam

Location

Related Links

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

MeSH Terms

Conditions

Hemophilia B

Interventions

Interviews as Topic

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; Genetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Nidhi Bhatt, MD

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: September 9, 2021
• First Posted: September 16, 2021
• Study Start: January 18, 2022
• Primary Completion: October 14, 2025
• Study Completion: October 14, 2025
• Last Updated: November 14, 2025

Record last verified: 2025-11

Locations

VN (1); TH (1); SR (1); PE (1); US (1); NE (1)