MedTrace Logo

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

NCT04259281 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 74

Last updated 2026-01-09

No results posted yet for this study

Summary

The primary objective of the study is to evaluate the safety and tolerability of multiple-ascending doses of GTX-102 administered by intrathecal (IT) injection to participants with Angelman Syndrome (AS).

Conditions

Interventions

DRUG

GTX-102

antisense oligonucleotide

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

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

Timeline & Regulatory

Start
2020-02-24
Primary Completion
2025-01-08
Completion
2025-01-08
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 NCT04259281 on ClinicalTrials.gov