NCT04259281

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

A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 74 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 4 Years – 17 Years
• Healthy volunteers: Not accepted

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).

Detailed description

This is a Phase 1/2, open-label, multiple-dose, study to evaluate the safety, tolerability, and plasma and CSF concentrations of GTX-102 in pediatric participants with AS. The study includes a Loading phase followed by a Maintenance phase. Participants may continue on GTX-102 during the Maintenance phase of the study until GTX-102 is commercially available, intolerable toxicity occurs, the parent/legal guardian withdraws consent, the participant enrolls in another experimental study, or this study is terminated. This study was previously posted by GeneTX Biotherapeutics, LLC and was transferred to Ultragenyx in July 2022.

Conditions

• Angelman Syndrome

Interventions

Type	Name	Description
DRUG	GTX-102	antisense oligonucleotide

Timeline

• Start date: 2020-02-24
• Primary completion: 2025-01-08
• Completion: 2025-01-08
• First posted: 2020-02-06
• Last updated: 2026-01-09

Locations

25 sites across 8 countries: United States, Australia, Canada, France, Germany, Israel, Spain, United Kingdom

Regulatory

• FDA-regulated drug study