MedTrace Logo

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

NCT07157254 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 60

Last updated 2026-04-24

No results posted yet for this study

Summary

The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.

Conditions

Interventions

OTHER

No intervention

During the no treatment period participants do not receive any study drug

DRUG

GTX-102

antisense oligonucleotide

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
1 Year
Max Age
64 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-10-13
Primary Completion
2030-01-31
Completion
2030-01-31
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Brazil
  • France
  • Israel
  • Italy
  • Portugal
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07157254 on ClinicalTrials.gov