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

St. Jude Children's Research Hospital

Status: Completed

Conditions: Gene Therapy; Hemophilia B

Treatments: Behavioral: Interview

Study type: Observational

Funder types: Other

Identifiers

NCT05044845

BINFORMED

Details and patient eligibility

About

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.

Full 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.

Enrollment: 150 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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

Trial design

150 participants in 3 patient groups

Hemophilia B patients

Description:

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

Treatment:

Behavioral: Interview

Parents or Caregivers

Description:

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

Treatment:

Behavioral: Interview

Healthcare Workers

Description:

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

Treatment:

Behavioral: Interview