Experimental injection tested in children with rare genetic disorder
NCT ID NCT04259281
Summary
This study tested the safety of an experimental drug called GTX-102 in children with Angelman syndrome, a rare genetic disorder that affects development. Researchers gave the drug through spinal injections to 74 children to see how well they tolerated it and to measure drug levels in their bodies. The study aimed to determine safe dosing before testing whether the treatment actually improves symptoms.
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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
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