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

NCT ID: NCT05044845

Last Updated: 2025-11-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

150 participants

Study Classification

OBSERVATIONAL

Study Start Date

2022-01-18

Study Completion Date

2025-10-14

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Gene Therapy Hemophilia B

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Hemophilia B patients

Patients ≥12 years of age with a diagnosis of moderate (FIX ≥1% and ≤2%) or severe (\<1%) hemophilia B

Interview

Intervention Type BEHAVIORAL

Semi structured interview will be conducted virtually using an online video-conferencing platform, phone or in person.

Parents or Caregivers

Parents or caregivers to patients with hemophilia 12-17 years of age

Interview

Intervention Type BEHAVIORAL

Semi structured interview will be conducted virtually using an online video-conferencing platform, phone or in person.

Healthcare Workers

Doctors, nurses, social workers, pharmacists and educators who participate in the care of hemophilia B patients

Interview

Intervention Type BEHAVIORAL

Semi structured interview will be conducted virtually using an online video-conferencing platform, phone or in person.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Interview

Semi structured interview will be conducted virtually using an online video-conferencing platform, phone or in person.

Intervention Type BEHAVIORAL

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Questions

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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


\- 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


* Health care workers who do not participate in the care of hemophilia B patients
* Healthcare worker who is conducting the interviews
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

St. Jude Children's Research Hospital

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Nidhi Bhatt, MD

Role: PRINCIPAL_INVESTIGATOR

St. Jude Children's Research Hospital

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status

Civil Service Hospital

Kathmandu, , Nepal

Site Status

Hospital Dos De Mayo

Lima, , Peru

Site Status

National Hospital of Sri Lanka

Colombo, , Sri Lanka

Site Status

Ramathibodi Hospital

Bangkok, , Thailand

Site Status

National Institute of Hematology and Blood Transfusion

Hanoi, , Vietnam

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Nepal Peru Sri Lanka Thailand Vietnam

Related Links

Access external resources that provide additional context or updates about the study.

http://www.stjude.org

St. Jude Children's Research Hospital

http://www.stjude.org/protocols

ClinicalTrials Open at St. Jude

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

BINFORMED

Identifier Type: -

Identifier Source: org_study_id