Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

129

Participants

Timeline

Start Date

December 3, 2024

Primary Completion Date

July 31, 2026

Study Completion Date

November 30, 2027

Conditions
Angelman Syndrome
Interventions
DRUG

GTX-102

antisense oligonucleotide

PROCEDURE

Sham-LP

Small needle prick on the lower back at the location where the LP injection is normally made

Trial Locations (28)

10032

Columbia University Medical Center, New York

20251

Universitaetsklinikum Hamburg-Eppendorf, Hamburg

27278

Rare Disease Research, Hillsborough

27599

UNC Chapel Hill Pediatrics, Chapel Hill

28222

Hospital Universitario Puerta de Hierro, Madrid

30329

Rare Disease Research, Atlanta

33155

Nicklaus Children's Hospital, Miami

41013

Hospital Universitario Virgen del Rocio, Seville

60612

Rush University, Chicago

64108

Children's Mercy, Kansas City

75243

Carum Research Inc, Dallas

78723

The University of Texas, Austin

80045

Children's Hospital Colorado, Aurora

80211

Medical University of Gdańsk, Gdansk

80336

Haunersche Kinderklinik, Munich

90048

Cedars Sinai, Los Angeles

92123

UCSD, Rady Children's Hospital, San Diego

94143

UCSF, San Francisco

02115

Boston Children's Hospital, Boston

H4A3J1

McGill University Health Centre, Montreal

V6H 3V4

British Columbia Children's Hospital, Vancouver

04155

University of Leipzig, Leipzig

467-0001

Nagoya City University Graduate School of Medical Sciences, Nagoya

543-0021

Osaka City General Hospital, Osaka

060-8648

Hokkaido University Hospital, Sapporo

93-338

Polish Mothers Memorial Institute, Lodz

08208

Hospital Universitario Parc Tauli, Barcelona

08950

Hospital Sant Joan de Deu, Barcelona

All Listed Sponsors
lead

Ultragenyx Pharmaceutical Inc

INDUSTRY

NCT06617429 - Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) | Biotech Hunter | Biotech Hunter