Brief Title
Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy
Official Title
Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy at a Single Center in the US and in Resource Limited Countries
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 at St. Jude Children's Research Hospital and in resource-limited countries. 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 in resource-limited countries and at St. Jude 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 resource limited 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
Primary Outcome
Use of semi-structured interviews to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B
Condition
Gene Therapy
Intervention
Interview
Study Arms / Comparison Groups
Hemophilia B patients
Description: Patients ≥12 years of age with a diagnosis of moderate (FIX ≥1% and ≤2%) or severe (<1%) hemophilia B
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Behavioral
Estimated Enrollment
60
Start Date
January 18, 2022
Completion Date
December 2022
Primary Completion Date
November 2022
Eligibility Criteria
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, 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
Gender
All
Ages
12 Years - N/A
Contacts
Nidhi Bhatt, MD, 901-426-6982, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT05044845
Organization ID
BINFORMED
Responsible Party
Sponsor
Study Sponsor
St. Jude Children's Research Hospital
Study Sponsor
Nidhi Bhatt, MD, Principal Investigator, St. Jude Children's Research Hospital
Verification Date
May 2022