Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy
- Conditions
- Transfusion Dependent Beta ThalassemiaSickle Cell Disease
- Registration Number
- NCT06664541
- Lead Sponsor
- Georgetown University
- Brief Summary
Determine knowledge, attitudes, and beliefs among adult patients, and parents of pediatric patients, with transfusion dependent beta-thalassemia and sickle cell disease toward gene therapy to treat their or their child's illness, and to assess the likely impact of gene therapy on patients' quality of life.
- Detailed Description
The overarching aim of this research is to determine knowledge, attitudes, and beliefs among adult patients, and parents of pediatric patients, with transfusion dependent beta-thalassemia toward gene therapy to treat their or their child's disease, and to assess the likely impact of gene therapy on patients' quality of life. We will compare and contrast these findings to those obtained from a sample of adult patients and parents of pediatric patients with sickle cell disease. We will also assess perceptions of the pros/cons of gene therapy, including its financial and time costs.
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 100
- adult patients (ages 18+) or parents of pediatric patients (<age 18) eligible to receive treatment for their disease with gene therapy
- Non-English-speaking
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Quality of Life Past 12 months Health related quality of life is measured by a modified patient reported outcome measure, the Transfusion-dependent Quality of Life (TranQoL) assessment.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Georgetown University Medical Center
🇺🇸Washington, District of Columbia, United States