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First-in-human Study of UX016 in GNEM

NCT07511556 · Status: NOT_YET_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2026-04-06

No results posted yet for this study

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).

Conditions

  • GNE Myopathy

Interventions

DRUG

UX016

Tablets for oral use

OTHER

Placebo

Tablets for oral use. Tablets will match the UX016 tablets, but contain no active ingredients

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-10-31
Primary Completion
2028-12-31
Completion
2028-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07511556 on ClinicalTrials.gov