Scientists launch Largest-Ever study of Ultra-Rare genetic syndrome

NCT ID NCT05528744

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study aims to better understand Chopra-Amiel-Gordon Syndrome (CAGS), a rare genetic disorder. Researchers will track symptoms, brain images, and learning challenges in 125 people with confirmed or suspected CAGS over time. No treatments are being tested—the goal is to collect data to help future research.

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 will provide a detailed understanding of CAGS symptoms and progression, which could guide future care and research.

What could go wrong

This is an observational study, not a treatment trial. It will not test any therapy, so direct benefits for participants are unlikely.

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.

Chopra-Amiel-Gordon syndrome hereditary disease Rare Diseases syndromic 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: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

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

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