First-in-human Study of UX016 in GNEM

Phase 1/2 Not yet recruiting 24 enrolled Ultragenyx Pharmaceutical Inc

RandomizedSequentialQuadruple-blindTreatment

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).

Timeline

Start: 2026-10
Primary completion: 2028-12
Completion: 2028-12

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	UX016	Unknown	—	Oral

First-in-human Study of UX016 in GNEM

Summary

Timeline

Drugs

Indications