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A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

Active, not recruiting · NCT07157254 · Registry updated 2026-09-09
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Clinical trials

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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)Abnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorderActive, not recruitingPhase 22026-09-09NCT07157254

1 results · Page 1 of 1

Organizations

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OrganizationsLinked trials
Ultragenyx Pharmaceutical Inc24

18 results · Page 1 of 1

Research sites

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Research sitesCityCountryLinked trials
Akron Children's HospitalAkronUnited States38
Azienda Ospedaliera Universitaria Meyer IRCCSFlorenceItaly4
Carum Research Inc.DallasUnited States1
Casa dos RarosSanta CecíliaBrazil1
Cedars Sinai Medical CenterLos AngelesUnited States135
Fondazione IRCCS Istituto Neurologico C. BestaMilanItaly1
Hopital de la TimoneMarseilleFrance14
Hospital de Crianças César Pernetta e Hospital Pequeno PríncipeCuritibaBrazil1
Hospital de Santa MariaLisbonPortugal7
Hospital Santa JoaoPortoPortugal1
Hospital Universitario AustralPilarArgentina1
Kennedy Kreiger InstituteBaltimoreUnited States1
Necker-Enfants Malades HospitalParisFrance3
Rare Disease ResearchHillsboroughUnited States4
Rush University Medical CenterChicagoUnited States283
Sheba Medical CenterRamat GanIsrael43
University of OxfordOxfordUnited Kingdom5
UT Health AustinAustinUnited States1