New drug trial aims to tame angelman syndrome in kids
NCT ID NCT04259281
First seen Jan 11, 2026 · Last updated May 14, 2026 · Updated 15 times
Summary
This study tested a new medicine called GTX-102 in 74 children with Angelman Syndrome, a rare genetic disorder causing developmental delays and seizures. The main goal was to see if the drug is safe and tolerable when given as multiple injections into the spine. Researchers monitored side effects and how the drug moves through the body.
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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.
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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Locations
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AP-HP Hopital Necker-Enfants Malades
Paris, 75015, France
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Austin Health
Heidelberg, Victoria, 3084, Australia
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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British Columbia Children's Hospital
Vancouver, British Columbia, V6H3V4, Canada
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Cambridge University Hospitals
Cambridge, United Kingdom
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Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
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Children's Hospital of Western Ontario
London, Ontario, Canada
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Great Ormond Street Hospital for Children
London, United Kingdom
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Hopital de la Timone
Marseille, France
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Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, Spain
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Hospital Universitari Parc Tauli
Sabadell, Barcelona, Spain
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Hospital Universitario Puerta de Hierro
Majadahonda, Madrid, Spain
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MAGIC Clinic Ltd
Calgary, Alberta, T2E 7Z4, Canada
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McGill University Health Centre
Montreal, Quebec, Canada
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Oxford University Hospitals NHS Foundation Trust
Oxford, OX3 7LE, United Kingdom
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Queensland Children's Hospital
South Brisbane, QLD 4101, Australia
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Rady Children's Hospital
San Diego, California, 92123, United States
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Rare Disease Research
Atlanta, Georgia, 30318, United States
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Rush University Medical Center
Chicago, Illinois, 60612, United States
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The Edmond and Lily Safra Children's Hospital
Ramat Gan, Israel
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The Royal Children's Hospital
Parkville, Victoria, 3052, Australia
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UCLA Medical Center
Los Angeles, California, 90095, United States
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Universitatsklinikum Hamburg-Eppendorf
Hamburg, 20246, Germany
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Universitatsklinikum Leipzig
Leipzig, Saxony, 04103, Germany
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Weill Cornell Medicine
New York, New York, 10065, United States
Conditions
Explore the condition pages connected to this study.