NCT06664541

Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy

• Status: Enrolling By Invitation
• Phase: —
• Study type: Observational
• Enrollment: 100 (estimated)
• Sponsor: Georgetown University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Transfusion Dependent Beta Thalassemia
• Sickle Cell Disease

Timeline

• Start date: 2024-10-01
• Primary completion: 2026-10-01
• Completion: 2026-12-01
• First posted: 2024-10-29
• Last updated: 2024-10-29

Locations

1 site across 1 country: United States