Recruiting

Understanding how families decide about gene therapy for rare diseases

Plain-English translation of NCT05810181 on ClinicalTrials.gov ↗ · Source last updated May 2026 · Translation generated June 2026 · How we translate trials

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What is this trial?

Researchers at St. Jude Children's Research Hospital are trying to understand what patients and families need to know when considering gene therapy — a new type of treatment that uses genetic editing to treat rare genetic diseases. This study will ask you about your experiences, concerns, and questions. Then, the research team will use what they learn to create a new website with educational materials and decision-making tools to help other families facing the same choice.

Right now, there are not enough good educational resources to help patients and families understand gene therapy options and make confident decisions about whether this treatment is right for them. This gap is becoming more urgent as new gene therapies are being tested in clinical trials.

Do you qualify?

You likely qualify if…

you are a parent or caregiver of a child (age 8 or older) with a rare genetic disease who has received gene therapy, or you are an adult (age 8+) who received gene therapy yourself
you are a parent or caregiver who was offered gene therapy but decided against it or were not eligible for a trial
you are a healthcare provider who has cared for at least two patients receiving gene therapy
you have internet access and a device (computer or mobile phone) to join a video interview
you are willing to allow researchers to verify your diagnosis with your doctor

You likely don't qualify if…

you cannot participate in a video interview using your own internet connection and device
you are unwilling to have your doctor confirm your diagnosis with the research team
if your child died from gene therapy, it has been less than 6 months or more than 24 months since their death
you are unable to provide informed consent to participate

What participation looks like

If you qualify, you will join a one-on-one video interview with a member of the research team using your own computer or phone — this is the main study activity. The researchers will ask you questions about your experience with gene therapy, your decision-making process, your concerns, and what information would have helped you. The study team will also contact your healthcare provider to verify your diagnosis. Your honest feedback will help shape a new educational platform designed to support families facing similar decisions in the future.

AI-generated summary from trial data · Jun 3, 2026 · Not medical advice

Trial locations(1 site)

United States

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