Hope for angelman syndrome: major trial tests drug to unlock speech and learning
NCT ID NCT06914609
First seen Nov 01, 2025 · Last updated May 12, 2026 · Updated 25 times
Summary
This study tests a new medicine called ION582 in 158 children and adults with Angelman syndrome, a genetic disorder causing severe developmental delays. The goal is to see if the drug can improve communication and thinking skills. Participants will receive the drug or a placebo, and their progress will be measured over time.
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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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Associazione La Nostra Famiglia - IRCCS Eugenio Medea
NOT_YET_RECRUITINGConegliano, 31015, Italy
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Azienda Ospedaliero Universitaria Pisana
NOT_YET_RECRUITINGPisa, 56126, Italy
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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British Columbia Children's Hospital
RECRUITINGVancouver, V6H 3V4, Canada
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Cedars-Sinai Medical Center
RECRUITINGLos Angeles, California, 90048, United States
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Children's Healthcare of Atlanta
RECRUITINGAtlanta, Georgia, 30329, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Children's Mercy
RECRUITINGKansas City, Missouri, 64108, United States
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Children's National Hospital
RECRUITINGWashington D.C., District of Columbia, 20011, United States
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Colorado Children's Hospital Research Institute
RECRUITINGAurora, Colorado, 80045, United States
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Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
RECRUITINGSabadell, 08208, Spain
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Fondazione IRCCS Istituto Neurologico Carlo Besta
NOT_YET_RECRUITINGMilan, 20133, Italy
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Great Ormond Street Hospital for Children - NHS Foundation Trust
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Hospital Sant Joan de Deu
RECRUITINGBarcelona, 08950, Spain
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Ichan School of Medicine at Mount Sinai
RECRUITINGNew York, New York, 10029, United States
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John Radcliffe Hospital
RECRUITINGOxford, OX3 9DU, United Kingdom
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KK Women's and Children's Hospital
RECRUITINGSingapore, 229899, Singapore
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Klinikum der Ludwig-Maximilians-Universitaet Muenchen
RECRUITINGMünchen, 81377, Germany
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London Health Science Centre - Children's Hospital
RECRUITINGLondon, Ontario, N6A 5W9, Canada
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McGill University Health Centre
RECRUITINGMontreal, Quebec, H4A 3J1, Canada
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National Center of Neurology and Psychiatry
RECRUITINGKodaira, Tokyo, 187-8551, Japan
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
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Nicklaus Children's Hospital
RECRUITINGMiami, Florida, 33155, United States
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Osaka Women's and Children's Hospital
RECRUITINGIzumi, Osaka, 594-1101, Japan
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Ospedale Pediatrico Bambino Gesù
NOT_YET_RECRUITINGRoma, 00165, Italy
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Perth Children's Hospital
RECRUITINGNedlands, 6009, Australia
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Queensland Children's Hospital
RECRUITINGSouth Brisbane, Queensland, 4101, Australia
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Rady Children's Hospital
RECRUITINGSan Diego, California, 92123, United States
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Rush University Medical Center
RECRUITINGChicago, Illinois, 60612, United States
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Samsung Medical Center
RECRUITINGSeoul, 06351, South Korea
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Seoul National University Hospital
RECRUITINGSeoul, 03080, South Korea
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Sheba Medical Center
RECRUITINGRamat Gan, 5265601, Israel
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Sydney Children's Hospital
RECRUITINGRandwick, 2031, Australia
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Texas Children's Hospital
RECRUITINGHouston, Texas, 77030, United States
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University of Alberta Hospital
RECRUITINGEdmonton, T6G 2B7, Canada
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University of North Carolina at Chapel Hill School of Medicine
RECRUITINGCarrboro, North Carolina, 27510, United States
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Uniwersyteckie Centrum Kliniczne
RECRUITINGGdansk, 80-952, Poland
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Vanderbilt Clinical Research Center
RECRUITINGNashville, Tennessee, 37232, United States
Conditions
Explore the condition pages connected to this study.