New angelman drug shows promise in early safety trial
NCT ID NCT04259281
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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 a spinal injection. Researchers monitored side effects and how the drug moves through the body.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can early parent coaching help infants with rare genetic disorders thrive?
- Newborn screening study aims to catch rare diseases at birth
- New hope for angelman syndrome: drug trial targets brain function
- New registry aims to shed light on angelman syndrome
- New study tracks angelman syndrome progression in kids and adults
- 2000-Patient study aims to uncover hidden metabolic risks in rare genetic disorders