Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

Glycogen Storage Disease Type III Clinical Trial

Official title:
A Phase 1/2 First-in-human, Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses and Repeat Doses of UX053 in Patients With GSD III

Status Terminated

Clinical Trial Summary

The primary objective of the study is to evaluate the safety of UX053 in adults with Glycogen Storage Disease Type III (GSD III).

Clinical Trial Description

This study is a phase 1/2 first-in-human (FIH), study to evaluate the safety, tolerability, and pharmacokinetic (PK) of a single ascending dose (SAD) and repeat doses (RD) of UX053 in patients with GSD III. The SAD cohorts will be open-label (OL). There will be two types of RD cohorts, an open-label (OL-RD) and a randomized, double-blind (DB), and placebo-controlled (DB-RD). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04990388
Study type	Interventional
Source	Ultragenyx Pharmaceutical Inc
Contact
Status	Terminated
Phase	Phase 1/Phase 2
Start date	October 18, 2021
Completion date	March 20, 2023

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See also
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