New spinal injection therapy tested in children with rare genetic disorder
NCT ID NCT04259281
Summary
This early-stage clinical trial tested the safety and tolerability of an experimental drug called GTX-102 in children with Angelman syndrome. The drug was given as multiple injections into the spinal fluid. The main goal was to see how well children handled the treatment and to measure how much of the drug reached their system.
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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
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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