Trials / Not Yet Recruiting
Not Yet RecruitingNCT07511556
First-in-human Study of UX016 in GNEM
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
- Status
- Not Yet Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 24 (estimated)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- 18 Years – 55 Years
- Healthy volunteers
- Not accepted
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
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | UX016 | Tablets for oral use |
| OTHER | Placebo | Tablets for oral use. Tablets will match the UX016 tablets, but contain no active ingredients |
Timeline
- Start date
- 2026-10-01
- Primary completion
- 2028-12-01
- Completion
- 2028-12-01
- First posted
- 2026-04-06
- Last updated
- 2026-04-06
Locations
2 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT07511556. Inclusion in this directory is not an endorsement.