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Clinical trial

A Phase 1/2, First-in-human, Double-blind, Placebo-controlled Study to Assess Dose, Safety, and Efficacy of UX016 (Sialic Acid-C16 Prodrug) in Adults With GNE Myopathy

NCT07511556 is registered in ClinicalTrials.gov with Ultragenyx Pharmaceutical Inc as lead sponsor.

NCT NCT07511556
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1/2, First-in-human, Double-blind, Placebo-controlled Study to Assess Dose, Safety, and Efficacy of UX016 (Sialic Acid-C16 Prodrug) in Adults With GNE Myopathy
Lead sponsor
Ultragenyx Pharmaceutical Inc
Phase
Phase 1/2
Overall status
Recruiting
Start date
October 31, 2026
Completion date
December 31, 2028
Enrollment
24 (estimated)
Sites
2

Conditions

As the registry lists them; a condition with its own page is linked.

gne myopathy