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

Angelman Syndrome Clinical Trial

Official title:
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)

Clinical Trial 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).

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04259281
Study type Interventional
Source Ultragenyx Pharmaceutical Inc
Contact
Status Active, not recruiting
Phase Phase 1/Phase 2
Start date February 24, 2020
Completion date December 2025
View Details
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