New hope for angelman syndrome: experimental drug enters mid-stage trial
NCT ID NCT07157254
First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 28 times
Summary
This study tests an experimental drug called GTX-102 in 60 children and adults with Angelman syndrome, a rare genetic disorder that causes severe developmental delays. The goal is to see if the drug is safe and can improve thinking, communication, and movement. Participants will be grouped by age and genetic type, and the study lasts about a year.
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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: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Akron Children's Hospital
RECRUITINGAkron, Ohio, 44308, United States
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Azienda Ospedaliera Universitaria Meyer IRCCS
RECRUITINGFlorence, Italy
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Carum Research Inc.
RECRUITINGDallas, Texas, 75243, United States
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Cedars Sinai Medical Center
RECRUITINGLos Angeles, California, 90048, United States
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Clinical Trial Site
NOT_YET_RECRUITINGBaltimore, Maryland, 21205, United States
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Clinical Trial Site
NOT_YET_RECRUITINGKansas City, Missouri, 64108, United States
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Clinical Trial Site
NOT_YET_RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Clinical Trial Site
NOT_YET_RECRUITINGPilar, Buenos Aires, Argentina
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Clinical Trial Site
NOT_YET_RECRUITINGCuritiba, Paraná, Brazil
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Clinical Trial Site
NOT_YET_RECRUITINGSanta Cecília, Porto Alegre, Brazil
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Clinical Trial Site
NOT_YET_RECRUITINGMarseille, France
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Clinical Trial Site
NOT_YET_RECRUITINGParis, France
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Clinical Trial Site
NOT_YET_RECRUITINGRamat Gan, Israel
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Clinical Trial Site
NOT_YET_RECRUITINGRome, Italy
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Clinical Trial Site
NOT_YET_RECRUITINGLondon, United Kingdom
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Clinical Trial Site
NOT_YET_RECRUITINGOxford, United Kingdom
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Fondazione IRCCS Istituto Neurologico C. Besta
RECRUITINGMilan, Italy
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Hospital Santa Joao
RECRUITINGPorto, Portugal
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Hospital de Santa Maria
RECRUITINGLisbon, Portugal
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Rare Disease Research
RECRUITINGHillsborough, North Carolina, 27278, United States
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Rush University Medical Center
RECRUITINGChicago, Illinois, 60612, United States
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UT Health Austin
RECRUITINGAustin, Texas, 78723, United States
Conditions
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