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

NCT06617429 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 129

Last updated 2026-05-06

No results posted yet for this study

Summary

The primary objective of this study is to evaluate the effect of GTX-102 in cognitive function in participants with deletion-type Angelman Syndrome (AS).

Conditions

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

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceuticals Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
4 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-12-03
Primary Completion
2026-07-31
Completion
2027-11-30
FDA Drug
Yes

Countries

  • United States
  • Canada
  • Germany
  • Japan
  • Poland
  • Spain

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 NCT06617429 on ClinicalTrials.gov