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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
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

Study Description
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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.

Condition or disease Intervention/treatment
Gene Therapy Hemophilia B Behavioral: Interview

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 Design
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Study Type : Observational
Estimated Enrollment : 150 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy Globally
Actual Study Start Date : January 18, 2022
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : January 2025

Resource links provided by the National Library of Medicine


Groups and Cohorts
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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 Name: 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 Name: 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 Name: Questions


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Sampling Method:   Non-Probability Sample
Study Population
Participants who meet the eligibility criteria
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05044845


Contacts
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Contact: Nidhi Bhatt, MD 901-426-6982 binformedstudy@stjude.org
Contact: Gail Fortner, BSN, RN 901-595- 5702 binformedstudy@stjude.org

Locations
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United States, Tennessee
St. Jude Children's Research Hospital Recruiting
Memphis, Tennessee, United States, 38105
Contact: Nidhi Bhatt, MD    866-278-5833    referralinfo@stjude.org   
Principal Investigator: Nidhi Bhatt, MD         
Sponsors and Collaborators
St. Jude Children's Research Hospital
Investigators
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Principal Investigator: Nidhi Bhatt, MD St. Jude Children's Research Hospital
More Information
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Additional Information:

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Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT05044845    
Other Study ID Numbers: BINFORMED
First Posted: September 16, 2021    Key Record Dates
Last Update Posted: February 26, 2024
Last Verified: February 2024

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked