New hope for angelman syndrome: Long-Term drug safety trial begins
NCT ID NCT06415344
First seen Nov 01, 2025 · Last updated May 20, 2026 · Updated 28 times
Summary
This study looks at the long-term safety of a drug called GTX-102 for people with Angelman Syndrome, a genetic disorder that causes developmental delays and seizures. About 255 participants who were in earlier GTX-102 trials will continue taking the drug to see if it remains safe over time. The goal is to manage symptoms, not to cure the condition.
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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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Locations
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AP-HM - Hopital de la Timone
Marseille, 13385, France
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AP-HP Hopital Necker-Enfants Malades
Paris, 75015, France
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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, V6H 3V4, Canada
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Childrens Hospital London Health Sciences Centre
London, Ontario, N6A 5W9, Canada
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Childrens Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
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Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
Sabadell, 08208, Spain
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Great Ormond Street Hospital for Children
London, WC1N 3JH, United Kingdom
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Hospital Universitario Puerta de Hierro
Majadahonda, 28222, Spain
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MAGIC Clinic Ltd
Calgary, Alberta, T2E 7Z4, Canada
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Oxford University Hospitals NHS Foundation Trust
Oxford, OX3 9DU, United Kingdom
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Queensland Children's Hospital
South Brisbane, Queensland, 4101, Australia
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Rare Disease Research, LLC
Atlanta, Georgia, 30329, United States
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Rush University Medical Center
Chicago, Illinois, 60612, United States
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Sheba Medical Center
Ramat Gan, 5265601, Israel
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The Research Institute of the McGill University Health Centre
Montreal, Quebec, H3G 1A4, Canada
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The Royal Children's Hospital
Melbourne, Victoria, 3052, Australia
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Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20246, Germany
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Universitaetsklinikum Leipzig
Leipzig, 04103, Germany
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University of California, Los Angeles (UCLA)
Los Angeles, California, 90095, United States
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University of California, San Diego - Rady Children's Hospital
San Diego, California, 92123, United States
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University of Cambridge
Cambridge, CB2 0QQ, United Kingdom
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Weill Cornell Medicine
New York, New York, 10021, United States
Conditions
Explore the condition pages connected to this study.