Clinical Trial

First-in-human Study of UX016 in GNEM

Name: First-in-human Study of UX016 in GNEM — clinical trial record (NCT07511556)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Not Yet Recruiting Phase 1/2

View on ClinicalTrials.gov →

Summary

The main goal of this study is to evaluate the safety of UX016 and to evaluate the impact of UX016 on muscle strength in adults with GNE Myopathy (GNEM).

Trial Details

NCT Number	NCT07511556
Lead Sponsor	Ultragenyx Pharmaceutical Inc
Conditions	GNE Myopathy
Enrollment	24 participants
Start Date	2026-10
Primary Completion	2028-12 (estimated)
Study Completion	2028-12 (estimated)
Updated on ClinicalTrials.gov	2026-04-06