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Long-term Extension of GTX-102 in Angelman Syndrome

NCT06415344 · Status: ENROLLING_BY_INVITATION · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 255

Last updated 2026-05-06

No results posted yet for this study

Summary

The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)

Conditions

Interventions

DRUG

GTX-102

Antisense Oligonucleotide

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceuticals Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-07-31
Primary Completion
2029-02-28
Completion
2029-02-28
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • France
  • Germany
  • Israel
  • Spain
  • 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 NCT06415344 on ClinicalTrials.gov