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

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ClinicalTrials.gov Identifier: NCT05044845
Recruitment Status : Recruiting
First Posted : September 16, 2021
Last Update Posted : February 26, 2024
Sponsor:
Information provided by (Responsible Party):
St. Jude Children's Research Hospital

Tracking Information
First Submitted Date September 9, 2021
First Posted Date September 16, 2021
Last Update Posted Date February 26, 2024
Actual Study Start Date January 18, 2022
Estimated Primary Completion Date December 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: August 18, 2023)		 Use of semi-structured interviews to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B [ Time Frame: 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.
Original Primary Outcome Measures
 (submitted: September 9, 2021)		 Use of semi-structured interviews to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B [ Time Frame: 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
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
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 Globally
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 Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Participants who meet the eligibility criteria
Condition
  • Gene Therapy
  • Hemophilia B
Intervention Behavioral: Interview
Semi structured interview will be conducted virtually using an online video-conferencing platform, phone or in person.
Other Name: Questions
Study Groups/Cohorts
  • Hemophilia B patients
    Patients ≥12 years of age with a diagnosis of moderate (FIX ≥1% and ≤2%) or severe (<1%) hemophilia B
    Intervention: Behavioral: Interview
  • Parents or Caregivers
    Parents or caregivers to patients with hemophilia 12-17 years of age
    Intervention: Behavioral: Interview
  • Healthcare Workers
    Doctors, nurses, social workers, pharmacists and educators who participate in the care of hemophilia B patients
    Intervention: Behavioral: Interview
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: February 23, 2024)		 150
Original Estimated Enrollment
 (submitted: September 9, 2021)		 60
Estimated Study Completion Date January 2025
Estimated Primary Completion Date December 2024   (Final data collection date for primary outcome measure)
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, 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
Sex/Gender
Sexes Eligible for Study: All
Ages 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers Not Provided
Contacts
Contact: Nidhi Bhatt, MD 901-426-6982 binformedstudy@stjude.org
Contact: Gail Fortner, BSN, RN 901-595- 5702 binformedstudy@stjude.org
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT05044845
Other Study ID Numbers BINFORMED
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Current Responsible Party St. Jude Children's Research Hospital
Original Responsible Party Same as current
Current Study Sponsor St. Jude Children's Research Hospital
Original Study Sponsor Same as current
Collaborators Not Provided
Investigators
Principal Investigator: Nidhi Bhatt, MD St. Jude Children's Research Hospital
PRS Account St. Jude Children's Research Hospital
Verification Date February 2024