Gene therapy trial aims to help girls with rett syndrome gain new skills
NCT ID NCT05898620
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a gene therapy called NGN-401 in 33 girls with Rett syndrome, a rare genetic disorder that affects development and movement. The therapy delivers a working copy of the MECP2 gene to try to improve skills and daily function. Researchers will measure success by whether participants show any improvement and gain at least one new milestone from a list of 28 skills.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
NGN-401 (a gene therapy using a harmless virus to deliver a working copy of the MECP2 gene)
What this could lead to
If successful, this could become a treatment that helps girls with Rett syndrome gain new skills and improve their daily lives.
What could go wrong
This is an early-stage, small trial (33 people) with no placebo group, so results may not be definitive. Gene therapies can have side effects like immune reactions or liver issues.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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Manchester University NHS Foundation Trust
Manchester, M13 9WL, United Kingdom
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Montefiore Medical Center
New York, New York, 10467, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Nicklaus Children's Hospital Research Institute
Miami, Florida, 33155, United States
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Royal Hospital for Children and Young People
Edinburgh, EH16 4TJ, United Kingdom
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Rush University Medical Center
Chicago, Illinois, 60612, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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The Children's Hospital at Westmead
Sydney, New South Wales, Australia
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
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UNC at Chapel Hill
Chapel Hill, North Carolina, 27514, United States
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University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
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Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States