Scientists track rare genetic disease to unlock its secrets

NCT ID NCT06014541

Terminated Knowledge-focused Sponsor: Ionis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Apr 06, 2026 · Last updated May 21, 2026 · Updated 3 times

Summary

This study looked at people with MECP2 duplication syndrome, a rare genetic condition. Researchers measured changes in biomarkers, symptoms, and quality of life over time. No treatment was given. The goal was to better understand how the disease progresses.

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Contacts and locations

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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

Conditions

Explore the condition pages connected to this study.

METHYL CPG BINDING PROTEIN 2 (MECP2) DUPLICATION SYNDROME