GENETX: Assessment for Gene Therapy Decisions

As more gene therapy treatments enter clinical trials, patients with rare genetic diseases may be asked to make decisions about these treatments. The long-term effects and efficacy of such treatments are uncertain. It may be difficult to compare the risks and benefits of new treatments with those of standard treatments. As an example, our recent research indicates that many patients with sickle cell disease do not understand gene therapy and have misconceptions.

We need more information about the communication needs of patients with rare diseases and their families who will be eligible for gene therapy. We will use this information to develop better education on the subject and understand patient preferences for care delivery.

In the GENETX study, the team will interview patients, caregivers, and health care workers of patients with rare genetic diseases. The interviews will provide information on their attitudes, beliefs, and concerns about gene therapy in both clinical and research settings. Using these insights, we will develop a platform with educational content and patient decision aids for patients and their families.

Primary Objectives

• Evaluate information needs about gene therapy
• Create a web-based platform to share information about gene therapy for patients with rare diseases and their families.

Eligibility Criteria

Inclusion criteria include:

Patient and Families:

• 18–35-year-old with a rare genetic disease, who was treated with gene therapy
• Parent or caregiver of a child with a rare genetic disease who had gene therapy
• 18–35-year-old who was offered gene therapy, but did not have it because they were not eligible or did not want it
• Parent or caregiver of a child who was offered gene therapy but did not have it because they were not eligible or did not want it

Health Care Professionals:

• Health care worker who cared for 2 or more patients who were treated with gene therapy

Exclusion criteria include:

• Unable to hold a fluent conversation in English
• No access to a computer or mobile device that supports video communications
• Condition or chronic illness that makes participation unsafe or unreasonable

Study Site

St. Jude Children’s Research Hospital, Memphis, Tennessee

Note: Interviews will be conducted remotely. Participants will not be required to travel.

About this study

GENETX will help us understand the information that patients and families need about gene therapy for rare genetic diseases, in order 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 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 what information you need
• Share your concerns about taking part in a gene therapy clinical trial

Purpose of this clinical trial

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

Eligibility overview

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

• 18–35-year-old with a rare genetic disease who had gene therapy
• Parent or caregiver of a child with a rare genetic disease who had gene therapy
• 18–35-year-old who was offered gene therapy, but did not have it because they were not eligible or did not want it
• Parent or caregiver of a child who was offered gene therapy, but did not have it because they were not eligible or did not want it
• Health care worker who cared for 2 or more patients who had gene therapy