Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Leber Hereditary Optic Neuropathy: later-stage trial registry
2 of 3 indexed Leber Hereditary Optic Neuropathy trials are currently recruiting, with more weight in Phase 3-4.
Corpus placement: #5,209 of 28,707 conditions by registered trial count.
3 US clinical trials · 2 currently recruiting · #5,209 of 28,707 conditions by registered trial count
Leber Hereditary Optic Neuropathy: 3 US clinical trials tracked, 2 recruiting.
Leber Hereditary Optic Neuropathy is the subject of 3 registered US clinical trials on ClinicalTrials.gov, 2 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 0 in Phase 1-2 (earlier-stage). The most active sponsor is GenSight Biologics, running 1 of these trials.
Enrollment posture brief
Registry condition leber-hereditary-optic-neuropathy
OPEN 2 | RECR 67% | LATE 1 | EARLY 0 | RANK #5209 | SPON 1
Volume-matched condition peers
Nearest open-share peer: Acute Hypoxemic Respiratory Failure (67% recruiting · 6 trials)
Leber Hereditary Optic Neuropathy lists 2 open slots (67% 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 Leber Hereditary Optic Neuropathy 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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Leber Hereditary Optic Neuropathy is recruiting above its size-band peers.
2 of 3 indexed trials (66.7%) 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
Stem Cell Ophthalmology Treatment Study II
MD Stem Cells
NCT03011541
New Non-invasive Modalities for Assessing Retinal Structure and Function
Randy Kardon
NCT03475173
RESCUE and REVERSE Long-term Follow-up
GenSight Biologics
NCT03406104
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 3 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Leber Hereditary Optic Neuropathy records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Leber Hereditary Optic Neuropathy 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 Leber Hereditary Optic Neuropathy page
3 registered trials, 2 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 2 Leber Hereditary Optic Neuropathy trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- GenSight Biologics sponsors the most Leber Hereditary Optic Neuropathy trials on record, review their full trial history before evaluating a specific study. See GenSight Biologics'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 Leber Hereditary Optic Neuropathy condition card totals 3 registered trials; most-linked intervention Sham Intravitreal Injection; top US site state Pennsylvania. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.