Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy
Launched by GEORGETOWN UNIVERSITY · Oct 28, 2024
Trial Information
Current as of August 29, 2025
Enrolling by invitation
Keywords
ClinConnect Summary
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.
Gender
ALL
Eligibility criteria
- Inclusion Criteria:
- • adult patients (ages 18+) or parents of pediatric patients (\<age 18) eligible to receive treatment for their disease with gene therapy
- Exclusion Criteria:
- • Non-English-speaking
About Georgetown University
Georgetown University is a prestigious academic institution renowned for its commitment to advancing medical research and education. As a clinical trial sponsor, Georgetown University leverages its extensive resources and expertise to conduct innovative research that addresses critical health challenges. The university fosters collaboration among multidisciplinary teams, emphasizing ethical practices and adherence to regulatory standards. With a focus on translating scientific discoveries into clinical applications, Georgetown University plays a vital role in enhancing patient care and contributing to the broader medical community.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Washington, District Of Columbia, United States
Patients applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported