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

Georgetown University

Status: Invitation-only

Conditions: Sickle Cell Disease; Transfusion Dependent Beta Thalassemia

Study type: Observational

Funder types: Other

Identifiers

NCT06664541

STUDY00007772

Details and patient eligibility

About

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.

Full 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.

Enrollment: 100 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

100 participants in 1 patient group

Group 1

Description:

Patients or parents of pediatric patients diagnosed with beta-thalassemia or sickle cell disease and eligible to receive treatment for their disease with gene therapy.