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NCT02377921 · ClinicalTrials.gov registry record · Phase 3
Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
A Phase 3 study, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- Phase 3
- Development phase
- 89
- Enrollment target
NCT02377921: Completed Phase 3 study, sponsored by Ultragenyx Pharmaceutical.
NCT02377921 is a Phase 3 study that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 89 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (88% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02377921, a Phase 3 study, has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 89 participants
- Enrollment target
Study Summary
The primary objective of this study is to evaluate the effect of 6 g/day aceneuramic acid extended-release (Ace-ER) treatment of participants with GNEM on upper extremity muscle strength (upper extremity composite \[UEC\] score) as measured by dynamometry.
Primary Outcome
Muscle strength based on the maximum voluntary isometric contraction (MVIC) against a dynamometer was measured bilaterally in the following upper extremity muscle groups: gross grip, shoulder abductors, elbow flexors, and elbow extensors. The UEC is derived from the sum of the average of the right and left total force values (measured in kg).
Interventions
- DRUG Placebo
- DRUG aceneuramic acid extended-release (Ace-ER)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 89 participants |
| Start Date | 2015-05-20 |
| Est. Completion | 2017-06-09 |
| Phase | Phase 3 |
What the finished NCT02377921 record still lists
NCT02377921 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 89 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (88% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT02377921 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02377921 about?
NCT02377921 is a clinical study titled "Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)". The primary objective of this study is to evaluate the effect of 6 g/day aceneuramic acid extended-release (Ace-ER) treatment of participants with GNEM on upper extremity muscle strength (upper extremity composite \[UEC\] score) as measured by dynamometry.
What is the current status of trial NCT02377921?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 89 participants. The study started on 2015-05-20. Estimated completion is 2017-06-09.
What interventions are being tested in trial NCT02377921?
The interventions under investigation include: Placebo (DRUG), aceneuramic acid extended-release (Ace-ER) (DRUG).
Who is sponsoring clinical trial NCT02377921?
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 NCT02377921's enrollment target sits among peer trials
89 1738th of 2000 higher than 262 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 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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