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NCT04231266 · ClinicalTrials.gov registry record · Phase 2
Multi-Center Study of ManNAc for GNE Myopathy
A Phase 2 study of GNE Myopathy, sponsored by Leadiant Biosciences.
- Active
- Registry status
- Phase 2
- Development phase
- 54
- Enrollment target
- 10
- Study locations
NCT04231266: Active Phase 2 study of GNE Myopathy, sponsored by Leadiant Biosciences.
NCT04231266 is a Phase 2 study of GNE Myopathy that is active but no longer recruiting, run by Leadiant Biosciences. The registered enrollment target is 54 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (59% lower). The trial reports 10 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04231266, a Phase 2 study of GNE Myopathy, is active but no longer recruiting, sponsored by Leadiant Biosciences.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 54 participants
- Enrollment target
- 10
- Study locations
Study Summary
GNE myopathy is a rare genetic muscle disease characterized by progressive muscle atrophy and weakness. The disease is caused by mutations in the gene that encodes the enzyme that initiates and regulates N-acetylneuraminic acid (Neu5Ac) biosynthesis and glycan sialylation. Currently, there is no therapy available for this disease. N-Acetylmannosamine (ManNAc), an orphan drug in development for GNE myopathy, is an uncharged monosaccharide and the first committed precursor in Neu5Ac biosynthesis. In this randomized, double-blind, placebo-controlled trial the efficacy and long-term safety of ManNAc will be evaluated in subjects with GNE myopathy.
Primary Outcome
The primary endpoint is the change in muscle strength decline under treatment compared to placebo. The primary analysis is based on the disease progression ratio (γ) comparing the rate of progression from baseline until last visit, under placebo to that under treatment.
Conditions Studied
Interventions
- OTHER Placebo
- DRUG ManNAc
Study Locations (10)
New York
- Columbia University Medical Center - New York
- University of Rochester - Rochester
California
- UCLA - Los Angeles
Iowa
- University of Iowa - Iowa City
Kansas
- University of Kansas, Medical Center - Kansas City
Maryland
- NIH Clinical Center - Bethesda
Massachusetts
- Brigham and Women's Hospital - Boston
Missouri
- Washington University - St Louis
Ohio
- Ohio State University, Wexner Medical Center - Columbus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 54 participants |
| Start Date | 2022-04-05 |
| Est. Completion | 2025-10-30 |
| Phase | Phase 2 |
What the registry record for NCT04231266 still lists
NCT04231266 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 54 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (59% lower).
The record links to 1 condition, with GNE Myopathy appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT04231266 lists 10 locations in 9 states (New York, California, Iowa).
Frequently Asked Questions
What is clinical trial NCT04231266 about?
NCT04231266 is a clinical study titled "Multi-Center Study of ManNAc for GNE Myopathy". GNE myopathy is a rare genetic muscle disease characterized by progressive muscle atrophy and weakness. The disease is caused by mutations in the gene that encodes the enzyme that initiates and regulates N-acetylneuraminic acid (Neu5Ac) biosynthesis and glycan sialylation. Currently, there is no the...
What is the current status of trial NCT04231266?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 54 participants. The study started on 2022-04-05. Estimated completion is 2025-10-30.
What conditions does trial NCT04231266 study?
This clinical trial studies the following conditions: GNE Myopathy.
What interventions are being tested in trial NCT04231266?
The interventions under investigation include: Placebo (OTHER), ManNAc (DRUG).
Who is sponsoring clinical trial NCT04231266?
This trial is sponsored by Leadiant Biosciences, which has 4 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04231266 being conducted?
This trial has 10 study locations across California, Iowa, Kansas, Maryland, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for GNE Myopathy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04231266's enrollment target sits among peer trials
54 1160th of 2000 higher than 830 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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