Rare disease ethics: what do patients and doctors really think about personalized trials?

NCT ID NCT07314736

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study interviews patients, families, healthcare providers, and researchers to learn their views on the ethical challenges of small-scale, personalized treatment trials for rare neurological diseases. About 385 participants will share their perspectives through semi-structured interviews. The goal is to develop a best-practice framework for conducting these trials ethically, focusing on informed consent, risk-benefit assessments, and fairness.

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 study could create a best-practice framework for conducting ethical personalized treatment trials for children with rare neurological diseases.

What could go wrong

This is an observational interview study, not a treatment trial. It will not directly test any therapy or improve any patient's condition.

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.

nervous system disorder Rare Diseases

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: •••••@•••••

    Contact