Assessing Knowledge and Attitudes About Gene Therapy in Hemophilia B Patients
Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy Globally
This study is trying to understand what people with hemophilia B and their caregivers know and think about gene therapy to help create better educational resources.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 150 (estimated) |
| Ages | 12 Years and up |
| Sex | All |
| Sponsor | St. Jude Children's Research Hospital Academic / other |
| Locations | 6 sites (Memphis, Tennessee and 5 other locations) |
| Trial ID | NCT05044845 on ClinicalTrials.gov |
What this trial studies
This observational study aims to evaluate the knowledge, beliefs, and attitudes of patients with hemophilia B regarding gene therapy. It will involve semi-structured interviews with patients aged 12 and older, their caregivers, and healthcare workers across various global locations, including St. Jude Children's Research Hospital. The study seeks to identify gaps in understanding and develop educational resources to facilitate informed consent for gene therapy, particularly in resource-limited settings. Interviews will be conducted virtually or in person, lasting approximately 45-60 minutes each.
Who should consider this trial
Good fit: Ideal candidates include patients aged 12 years and older with a diagnosis of moderate to severe hemophilia B.
Not a fit: Patients with hemophilia A or other non-hemophilia B bleeding disorders will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance patient understanding and acceptance of gene therapy, leading to improved treatment outcomes for hemophilia B.
How similar studies have performed: While this study addresses a specific gap in knowledge and education regarding gene therapy in hemophilia B, similar qualitative approaches have shown promise in other areas of patient education.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Memphis, Tennessee and 5 other locations
- St. Jude Children's Research Hospital — Memphis, Tennessee, United States (Recruiting)
- Civil Service Hospital — Kathmandu, Nepal (Recruiting)
- Hospital Dos De Mayo — Lima 15003, Peru (Recruiting)
- National Hospital of Sri Lanka — Colombo 10, Sri Lanka (Recruiting)
- Ramathibodi Hospital — Bangkok, Thailand (Recruiting)
- National Institute of Hematology and Blood Transfusion — Hanoi, Vietnam (Recruiting)
Study contacts
- Principal investigator: Nidhi Bhatt, MD — St. Jude Children's Research Hospital
- Study coordinator: Nidhi Bhatt, MD
- Email: binformedstudy@stjude.org
- Phone: 901-426-6982
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.