Brain drug tested for rare genetic disorder in kids
NCT ID NCT05630066
Summary
This study tested a drug called alogabat in children and teenagers with Angelman syndrome, a rare genetic disorder. The main goals were to see how the drug moves through the body, if it is safe, and if it changes brain wave patterns. Forty-eight participants aged 5 to 17 took the drug daily for 12 weeks while researchers monitored them.
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Contacts and locations
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Locations
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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CHRU de Brest
Brest, 29609, France
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CHU Dijon Bourgogne Hôpital François Mitterand
Dijon, 21000, France
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Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine
Carrboro, North Carolina, 27510, United States
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Columbia University Medical Center
New York, New York, 10032, United States
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Corporacio Sanitaria Parc Tauli
Sabadell, Barcelona, 08208, Spain
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Dr. Von Haunersches Kinderspital
München, 80337, Germany
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Groupe Hospitalier Necker Enfants Malades
Paris, 75015, France
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Hopital la Timone Enfants
Marseille, 13005, France
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Hospital Sant Joan de Deu
Esplugues de Llobregat · Barcelona, Barcelona, 08950, Spain
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Hospital Universitario Puerta De Hierro Majadahonda
Madrid, 28222, Spain
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Hospital Universitario de Navarra;Unidad de Neuropediatría
Pamploa, Navarre, 31008, Spain
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IRCCS Eugenio Medea
Conegliano Veneto (TV), Veneto, 31015, Italy
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IRCCS Istituto G. Gaslini
Genoa, Liguria, 16147, Italy
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Multicare Institute for Research and Innovation
Tacoma, Washington, 98405, United States
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Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 00165, Italy
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Queensland Children?s Hospital
South Brisbane, Queensland, 4101, Australia
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Rush Medical Center
Chicago, Illinois, 60612, United States
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Vanderbilt Children's Hospital
Nashville, Tennessee, 37232-9119, United States
Conditions
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