Skip to Main Content
Recruiting

GENETX: Assessing Gene Therapy Communication Needs

About this study

GENETX will help us understand the information that patients and families need about gene therapy for rare genetic diseases, to make important decisions about treatment. We want to share correct, easy-to-understand information about gene therapy, so patients and families can speak with their care teams about it.

First, we need to find out what kind of information will help. At the beginning of this study, we will speak with

  • Patients with inherited genetic disorders where gene therapy is a possible treatment
    • Blood Disorders like Sickle Cell Disease and Hemophilia
    • Neuromuscular Diseases like Duchenne Muscular Dystrophy
  • Families of these patients
  • Health care workers who care for these patients.

By taking part in this study, they will help us create online resources for others.

If you enroll in this study, you will:

  • Do an online video interview
  • Share your thoughts about what information you need
  • Share your concerns about taking part in a gene therapy clinical trial

Eligibility overview

To take part in this study, you must be one of the following:

  • Parent or caregiver whose child had gene therapy OR a parent or caregiver of a child who died after receiving gene therapy at least 6 to 24 months before enrollment OR patients age 8 and above who had gene therapy.
  • Parent or caregiver of children (patients 8 and above) with a rare genetic disease who were offered a clinical trial but were not eligible or decided against receiving gene therapy.
  • Health care worker who has cared for 2 or more patients who had gene therapy

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.

Overview

Study goal:

To learn more about participants' beliefs, attitudes, and questions about gene therapy to help make web-based resources to share information. This could help patients make treatment decisions.

Age:

18 to 35 years old with rare genetic diseases

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.

 

Learn more