A Study of the Safety and Tolerability of GTX-102 in Children with Angelman Syndrome

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

74

Participants

Timeline

Start Date

February 24, 2020

Primary Completion Date

January 8, 2025

Study Completion Date

January 8, 2025

Conditions
Angelman Syndrome
Interventions
DRUG

GTX-102

antisense oligonucleotide

Trial Locations (25)

3052

The Royal Children's Hospital, Parkville

3084

Austin Health, Heidelberg

10065

Weill Cornell Medicine, New York

20246

Universitatsklinikum Hamburg-Eppendorf, Hamburg

30318

Rare Disease Research, Atlanta

60612

Rush University Medical Center, Chicago

75015

AP-HP Hopital Necker-Enfants Malades, Paris

90095

UCLA Medical Center, Los Angeles

92123

Rady Children's Hospital, San Diego

02115

Boston Children's Hospital, Boston

QLD 4101

Queensland Children's Hospital, South Brisbane

T2E 7Z4

MAGIC Clinic Ltd, Calgary

V6H3V4

British Columbia Children's Hospital, Vancouver

Unknown

Children's Hospital of Western Ontario, London

McGill University Health Centre, Montreal

Hopital de la Timone, Marseille

The Edmond and Lily Safra Children's Hospital, Ramat Gan

Hospital Sant Joan de Deu, Esplugues de Llobregat

Hospital Universitari Parc Tauli, Sabadell

Hospital Universitario Puerta de Hierro, Majadahonda

Cambridge University Hospitals, Cambridge

Great Ormond Street Hospital for Children, London

K1H 8L1

Children's Hospital of Eastern Ontario, Ottawa

04103

Universitatsklinikum Leipzig, Leipzig

OX3 7LE

Oxford University Hospitals NHS Foundation Trust, Oxford

All Listed Sponsors
lead

Ultragenyx Pharmaceutical Inc

INDUSTRY

NCT04259281 - A Study of the Safety and Tolerability of GTX-102 in Children with Angelman Syndrome | Biotech Hunter | Biotech Hunter