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NCT07511556 · ClinicalTrials.gov registry record · Phase 1
First-in-human Study of UX016 in GNEM
A Phase 1 study, sponsored by Ultragenyx Pharmaceutical.
- Not yet
- Registry status
- Phase 1
- Development phase
- 24
- Enrollment target
NCT07511556: Clinical Trial Phase 1 study, sponsored by Ultragenyx Pharmaceutical.
NCT07511556 is a Phase 1 study that has not yet begun recruiting, run by Ultragenyx Pharmaceutical. The registered enrollment target is 24 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (60% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07511556, a Phase 1 study, has not yet begun recruiting, sponsored by Ultragenyx Pharmaceutical.
- NOT YET RECRUITING
- Registry status
- Phase 1
- Development phase
- 24 participants
- Enrollment target
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).
Interventions
- OTHER Placebo
- DRUG UX016
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2026-10 |
| Est. Completion | 2028-12 |
| Phase | Phase 1 |
What is known before NCT07511556 opens enrollment
NCT07511556 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 24 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (60% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT07511556 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT07511556 about?
NCT07511556 is a clinical study titled "First-in-human Study of UX016 in GNEM". 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).
What is the current status of trial NCT07511556?
This trial is currently not yet recruiting. It is a Phase 1 study. The enrollment target is 24 participants. The study started on 2026-10. Estimated completion is 2028-12.
What interventions are being tested in trial NCT07511556?
The interventions under investigation include: Placebo (OTHER), UX016 (DRUG).
Who is sponsoring clinical trial NCT07511556?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT07511556's enrollment target sits among peer trials
24 1520th of 2000 higher than 405 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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