Scientists hunt for clues to rare genetic disorders

NCT ID NCT06274164

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This observational study aims to find biological markers (biomarkers) for RAI1-related disorders, including Smith-Magenis and Potocki-Lupski syndromes. Researchers will study 90 participants through clinical exams, blood tests, optional skin biopsies, and sleep studies. The goal is to better understand these conditions and develop tools to measure disease severity and treatment effects in future trials.

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 identify reliable biomarkers to track disease progression and response to future treatments for RAI1-related disorders.

What could go wrong

This is an early observational study, not a treatment trial. It may not find stable biomarkers, and results may not apply to all patients.

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.

Potocki-Lupski syndrome Smith-Magenis syndrome

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

  • Texas Children's Hospital

    RECRUITING

    Houston, Texas, 77030, United States

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