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

NCT06664541 | Enrolling By Invitation

• 1 other identifier: STUDY00007772
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 1

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.

Conditions

Transfusion Dependent Beta Thalassemia; Sickle Cell Disease

Keywords

quality of life

Outcome Measures

Primary Outcomes (1)

• Quality of Life

  Health related quality of life is measured by a modified patient reported outcome measure, the Transfusion-dependent Quality of Life (TranQoL) assessment.

  Past 12 months

Study Arms (1)

Group 1

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.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adult patients (ages 18+) or parents of pediatric patients (\<age 18) diagnosed with transfusion-dependent beta thalassemia or sickle cell disease and eligible to receive treatment for their disease with gene therapy.

You may qualify if:

• adult patients (ages 18+) or parents of pediatric patients (\<age 18) eligible to receive treatment for their disease with gene therapy

You may not qualify if:

• Non-English-speaking

Sponsors & Collaborators

• Georgetown University: lead

Study Sites (1)

Georgetown University Medical Center

Washington D.C., District of Columbia, 20007, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 28, 2024
• First Posted: October 29, 2024
• Study Start: October 1, 2024
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: October 29, 2024

Record last verified: 2024-10

Locations

US (1)