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NCT02731690 · ClinicalTrials.gov registry record · Phase 2
A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment
A Phase 2 study, sponsored by Ultragenyx Pharmaceutical.
- Terminated
- Registry status
- Phase 2
- Development phase
- 42
- Enrollment target
NCT02731690: Clinical Trial Phase 2 study, sponsored by Ultragenyx Pharmaceutical.
NCT02731690 is a Phase 2 study that was terminated before completion, run by Ultragenyx Pharmaceutical. The registered enrollment target is 42 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (68% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02731690, a Phase 2 study, was terminated before completion, sponsored by Ultragenyx Pharmaceutical.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 42 participants
- Enrollment target
Study Summary
The primary objective of this Phase 2 study is to evaluate the safety of open-label 6 g/day Ace-ER in GNEM participants with severe ambulatory impairment.
Primary Outcome
An AE was defined as any untoward medical occurrence associated with the use of a drug, whether or not considered drug related. An SAE or serious suspected adverse reaction is an AE or suspected adverse reaction that at any dose, in the view of either the Investigator or Ultragenyx, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability (substantial disruptio
Interventions
- DRUG Aceneuramic Acid Extended-Release
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 42 participants |
| Start Date | 2016-04-29 |
| Est. Completion | 2018-01-10 |
| Phase | Phase 2 |
Why NCT02731690 stopped before completion
NCT02731690 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 42 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (68% lower).
The record links to 0 conditions, and to 1 intervention - of which Aceneuramic Acid Extended-Release is the first listed.
NCT02731690 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02731690 about?
NCT02731690 is a clinical study titled "A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment". The primary objective of this Phase 2 study is to evaluate the safety of open-label 6 g/day Ace-ER in GNEM participants with severe ambulatory impairment.
What is the current status of trial NCT02731690?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 42 participants. The study started on 2016-04-29. Estimated completion is 2018-01-10.
What interventions are being tested in trial NCT02731690?
The interventions under investigation include: Aceneuramic Acid Extended-Release (DRUG).
Who is sponsoring clinical trial NCT02731690?
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 NCT02731690's enrollment target sits among peer trials
42 1339th of 2000 higher than 649 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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