Gene therapy trial offers hope for girls with rett syndrome
NCT ID NCT05898620
First seen Nov 10, 2025 · Last updated May 19, 2026 · Updated 22 times
Summary
This study tests a gene therapy called NGN-401 in girls with Rett syndrome, a rare genetic disorder that affects development and movement. The goal is to see if the treatment can help improve skills and daily function. About 33 girls aged 3 and older will take part in this Phase 3 trial.
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This is a summary of
the original study
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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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
Contact Email: •••••@•••••
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Children's Hospital of Philadelphia
NOT_YET_RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Kennedy Krieger Institute
RECRUITINGBaltimore, Maryland, 21205, United States
Contact Email: •••••@•••••
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Manchester University NHS Foundation Trust
TERMINATEDManchester, M13 9WL, United Kingdom
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Montefiore Medical Center
RECRUITINGNew York, New York, 10467, United States
Contact Email: •••••@•••••
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
Contact Email: •••••@•••••
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Nicklaus Children's Hospital Research Institute
RECRUITINGMiami, Florida, 33155, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Royal Hospital for Children and Young People
TERMINATEDEdinburgh, EH16 4TJ, United Kingdom
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Rush University Medical Center
RECRUITINGChicago, Illinois, 60612, United States
Contact Email: •••••@•••••
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Texas Children's Hospital
RECRUITINGHouston, Texas, 77030, United States
Contact Email: •••••@•••••
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The Children's Hospital at Westmead
ACTIVE_NOT_RECRUITINGSydney, New South Wales, Australia
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UCSF Benioff Children's Hospital Oakland
RECRUITINGOakland, California, 94609, United States
Contact Email: •••••@•••••
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UNC at Chapel Hill
RECRUITINGChapel Hill, North Carolina, 27514, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Alabama at Birmingham
RECRUITINGBirmingham, Alabama, 35233, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Vanderbilt University Medical Center
RECRUITINGNashville, Tennessee, 37232, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.