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

February 23, 2024 updated by: St. Jude Children's Research Hospital

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

Recruiting

Conditions

Intervention / Treatment

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 (Estimated)

150

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • Recruiting
        • St. Jude Children's Research Hospital
        • Contact:
        • Principal Investigator:
          • Nidhi Bhatt, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

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
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.
Day 1, or at a future visit (up to approximately 1 year)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

St. Jude Children's Research Hospital

Investigators

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 18, 2022

Primary Completion (Estimated)

December 1, 2024

Study Completion (Estimated)

January 1, 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)

February 26, 2024

Last Update Submitted That Met QC Criteria

February 23, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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