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

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

Start Date

October 2021

Completion Date

July 2022

Primary Completion Date

June 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

Accepts Healthy Volunteers

No

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

October 2021