Breakthrough drug trial offers hope for angelman syndrome families
NCT ID NCT06914609
Summary
This study is testing whether the experimental drug ION582 can improve symptoms in children and adults with Angelman syndrome, a rare genetic disorder that affects development. About 158 participants aged 2 to 50 will receive either the drug or a placebo for about 60 weeks, followed by a longer treatment period where everyone gets the drug. Researchers will measure changes in communication skills, thinking abilities, daily living skills, and sleep problems.
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
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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 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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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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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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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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Vanderbilt Clinical Research Center
RECRUITINGNashville, Tennessee, 37232, United States
Conditions
Explore the condition pages connected to this study.