Potential breakthrough treatment tested for rare genetic disorder
NCT ID NCT06617429
Summary
This study is testing whether an investigational drug called GTX-102 can improve thinking skills and other symptoms in children with Angelman syndrome, a rare genetic disorder that affects development. Researchers are comparing the drug against a sham (placebo-like) procedure in 129 children to see if it helps with learning, communication, and behavior. The goal is to see if this treatment can meaningfully reduce the burden of the condition.
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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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British Columbia Children's Hospital
Vancouver, V6H 3V4, Canada
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Carum Research Inc
Dallas, Texas, 75243, United States
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Cedars Sinai
Los Angeles, California, 90048, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Mercy
Kansas City, Missouri, 64108, United States
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Columbia University Medical Center
New York, New York, 10032, United States
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Haunersche Kinderklinik
Munich, 80336, Germany
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Hokkaido University Hospital
Sapporo, 060-8648, Japan
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Hospital Sant Joan de Deu
Barcelona, 08950, Spain
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Hospital Universitario Parc Tauli
Barcelona, 08208, Spain
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Hospital Universitario Puerta de Hierro
Madrid, 28222, Spain
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Hospital Universitario Virgen del Rocio
Seville, 41013, Spain
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McGill University Health Centre
Montreal, Quebec, H4A3J1, Canada
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Medical University of Gdańsk
Gdansk, 80211, Poland
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Nagoya City University Graduate School of Medical Sciences
Nagoya, Aichi-ken, 467-0001, Japan
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Nicklaus Children's Hospital
Miami, Florida, 33155, United States
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Osaka City General Hospital
Osaka, 543-0021, Japan
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Polish Mothers Memorial Institute
Lodz, 93-338, Poland
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Rare Disease Research
Atlanta, Georgia, 30329, United States
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Rare Disease Research
Hillsborough, North Carolina, 27278, United States
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Rush University
Chicago, Illinois, 60612, United States
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The University of Texas
Austin, Texas, 78723, United States
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UCSD, Rady Children's Hospital
San Diego, California, 92123, United States
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UCSF
San Francisco, California, 94143, United States
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UNC Chapel Hill Pediatrics
Chapel Hill, North Carolina, 27599, United States
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Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20251, Germany
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University of Leipzig
Leipzig, 04155, Germany
Conditions
Explore the condition pages connected to this study.