Talking gene therapy: new study seeks family insights

NCT ID NCT05810181

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study interviews patients and families who have experience with gene therapy for rare pediatric diseases, including sickle cell disease. Researchers want to understand their beliefs, attitudes, and what information they need. The goal is to create an online platform with educational content and decision aids to help future families. No treatment is given in this study.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

What this could lead to

If successful, this could lead to better educational tools and decision aids for families considering gene therapy for rare diseases.

What could go wrong

This is an interview study, not a treatment trial. It may not directly improve health outcomes, and the tools developed may not suit all families.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

bone marrow failure syndrome sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • St. Jude Children's Research Hospital

    RECRUITING

    Memphis, Tennessee, 38105, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••