Scientists hunt for clues in rare genetic brain disorder

NCT ID NCT06014541

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

Summary

This study aimed to observe how MECP2 duplication syndrome progresses over time by measuring biological markers in spinal fluid and blood, and by tracking changes in development, behavior, and seizures. It enrolled 29 males aged 1 month to 65 years with a confirmed genetic diagnosis. The study was terminated early, so results may be limited.

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 help researchers better understand how MECP2 duplication syndrome progresses and identify biological markers to use in future treatment trials.

What could go wrong

This is an observational study that was terminated early, so it may not collect enough data to draw firm conclusions. It does not test any treatment, so there is no direct benefit to participants.

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.

Rett syndrome syndromic X-linked intellectual disability Lubs type

As listed by the trial registrant

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

Contacts and locations

Locations

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Gillette Children's Specialty Healthcare

    Saint Paul, Minnesota, 55101, United States

  • UCSD - Rady Children's Hospital

    San Diego, California, 92123, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37203, United States