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

Completed

Conditions studied: Gene Therapy, Hemophilia B

In brief

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.

Key facts

Study ID

NCT05044845

Run by

St. Jude Children's Research Hospital

People needed

150

Starts

2022-01-18

Expected to finish

2025-10-14

Last updated by the study team

2025-11-14

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• St. Jude Children's Research Hospital — Memphis, Tennessee, United States
• Civil Service Hospital — Kathmandu, Nepal
• Hospital Dos De Mayo — Lima, Peru
• National Hospital of Sri Lanka — Colombo, Sri Lanka
• Ramathibodi Hospital — Bangkok, Thailand
• National Institute of Hematology and Blood Transfusion — Hanoi, Vietnam

Full record on ClinicalTrials.gov

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