Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
GNE Myopathy: early-phase trial registry
1 of 3 indexed GNE Myopathy trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #5,209 of 28,707 conditions by registered trial count.
3 US clinical trials · 1 currently recruiting · #5,209 of 28,707 conditions by registered trial count
GNE Myopathy: 3 US clinical trials tracked, 1 recruiting.
GNE Myopathy is the subject of 3 registered US clinical trials on ClinicalTrials.gov, 1 of them currently open to new participants. 0 are in Phase 3-4 (later-stage) and 2 in Phase 1-2 (earlier-stage). The most active sponsor is Leadiant Biosciences, running 1 of these trials.
Enrollment posture brief
Registry condition gne-myopathy
OPEN 1 | RECR 33% | LATE 0 | EARLY 2 | RANK #5209 | SPON 1
Volume-matched condition peers
Nearest open-share peer: Adherence, Treatment (33% recruiting · 6 trials)
GNE Myopathy lists 1 open slots (33% of 3 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for GNE Myopathy on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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GNE Myopathy is recruiting below its size-band peers.
1 of 3 indexed trials (33.3%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
Open and recent trials
3 total, page 1 of 1
A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases
National Human Genome Research Institute (NHGRI)
NCT01417533
Multi-Center Study of ManNAc for GNE Myopathy
Leadiant Biosciences
NCT04231266
A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
Ultragenyx Pharmaceutical
NCT01517880
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 2 | 2 |
Top Sponsors
Named interventions
Most-linked intervention names on GNE Myopathy records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on GNE Myopathy trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this GNE Myopathy page
3 registered trials, 1 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 1 GNE Myopathy trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Leadiant Biosciences sponsors the most GNE Myopathy trials on record, review their full trial history before evaluating a specific study. See Leadiant Biosciences's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This GNE Myopathy condition card totals 3 registered trials; most-linked intervention Sialic Acid Extended Release (SA-ER); top US site state New York. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.