Long-term Extension of GTX-102 in Angelman Syndrome

PHASE3Enrolling by invitationINTERVENTIONAL
Enrollment

255

Participants

Timeline

Start Date

July 31, 2024

Primary Completion Date

February 28, 2029

Study Completion Date

February 28, 2029

Conditions
Angelman Syndrome
Interventions
DRUG

GTX-102

Antisense Oligonucleotide

Trial Locations (23)

3052

The Royal Children's Hospital, Melbourne

4101

Queensland Children's Hospital, South Brisbane

10021

Weill Cornell Medicine, New York

13385

AP-HM - Hopital de la Timone, Marseille

20246

Universitaetsklinikum Hamburg-Eppendorf, Hamburg

28222

Hospital Universitario Puerta de Hierro, Majadahonda

30329

Rare Disease Research, LLC, Atlanta

60612

Rush University Medical Center, Chicago

75015

AP-HP Hopital Necker-Enfants Malades, Paris

90095

University of California, Los Angeles (UCLA), Los Angeles

92123

University of California, San Diego - Rady Children's Hospital, San Diego

5265601

Sheba Medical Center, Ramat Gan

02115

Boston Children's Hospital, Boston

T2E 7Z4

MAGIC Clinic Ltd, Calgary

V6H 3V4

British Columbia Children's Hospital, Vancouver

N6A 5W9

Childrens Hospital London Health Sciences Centre, London

K1H 8L1

Childrens Hospital of Eastern Ontario, Ottawa

H3G 1A4

The Research Institute of the McGill University Health Centre, Montreal

04103

Universitaetsklinikum Leipzig, Leipzig

08208

Corporacio Sanitaria Parc Tauli - Hospital de Sabadell, Sabadell

CB2 0QQ

University of Cambridge, Cambridge

WC1N 3JH

Great Ormond Street Hospital for Children, London

OX3 9DU

Oxford University Hospitals NHS Foundation Trust, Oxford

All Listed Sponsors
lead

Ultragenyx Pharmaceutical Inc

INDUSTRY