Condition (MeSH)
Optic Atrophy, Hereditary, Leber
ClinicalTrials.gov lists 44 trials for Optic Atrophy, Hereditary, Leber or a narrower condition beneath it.
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Trials per phase
1 more section with rows
44 of the 44 trials are tagged with Optic Atrophy, Hereditary, Leber itself; the rest are tagged with a narrower condition beneath it.
When did Optic Atrophy, Hereditary, Leber trials start? (17)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Not applicable
- Observational
Top of scale: 8
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 2 |
| 2025 | 2 |
| 2024 | 1 |
| 2023 | 3 |
| 2022 | 1 |
| 2021 | 1 |
| 2019 | 3 |
| 2018 | 4 |
| 2017 | 2 |
| 2016 | 8 |
Showing 10 of 17 earlier years.
No forecast is made for this condition: it averages 1.8 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 3 trials still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Optic Atrophy, Hereditary, Leber trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 3 |
| Phase 1/2 | 6 |
| Phase 2 | 6 |
| Phase 2/3 | 3 |
| Phase 3 | 8 |
| Phase 4 | 1 |
| Not applicable | 5 |
| Observational | 9 |
| Expanded access | 3 |
How many Optic Atrophy, Hereditary, Leber trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 2 |
| Recruiting | 7 |
| Active, not recruiting | 3 |
| Completed | 21 |
| Terminated | 5 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Optic Atrophy, Hereditary, Leber trials? (6)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| GenSight Biologics | 8 |
| Santhera Pharmaceuticals | 8 |
| Laboratoires Thea | 3 |
| Amgen | 2 |
| University Hospital, Angers | 2 |
Showing 5 of 6 sponsors.
Which drugs are being tested for Optic Atrophy, Hereditary, Leber? (4)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| GS010 | 6 |
| Idebenone | 5 |
| sepofarsen | 4 |
| (Cystagon) Cysteamine Bitartrate | 2 |
Interventions as named in the registry (11)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| rAAV2-ND4 | 3 |
| NR082 Injection | 2 |
| Nicotinamide treatment | 1 |
| NFS-02 Injection | 1 |
| Cyclosporine 0.05% | 1 |
Showing 5 of 11 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Optic Atrophy, Hereditary, Leber trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Total | 34 | 1 |
| Cataract | 6 | 1 |
| Vomiting | 5 | 2 |
| Convulsion | 4 | 2 |
| Dehydration | 3 | 2 |
Showing 5 of 10 adverse events.
Which publications cover Optic Atrophy, Hereditary, Leber? (50)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy. | JAMA ophthalmology | 2025 | Yes |
| Leber Hereditary Optic Neuropathy Gene Therapy: Longitudinal Relationships Among Visual Function and Anatomical Measures. | American journal of ophthalmology | 2024 | Yes |
| Leber hereditary optic neuropathy gene therapy. | Current opinion in ophthalmology | 2024 | Yes |
| Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial. | Cell reports. Medicine | 2024 | Yes |
| Induced Pluripotent Stem Cells for Inherited Optic Neuropathies-Disease Modeling and Therapeutic Development. | Journal of neuro-ophthalmology : the official journal of the North American Neuro-Ophthalmology Society | 2022 | Yes |
Showing 5 of 50 publications.
Prevalence (21)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Leber hereditary optic neuropathy | Point prevalence | 1-9 / 100 000 | United Kingdom | GB | Exact |
| Leber hereditary optic neuropathy | Point prevalence | 1-9 / 100 000 | United Kingdom | GB | Unspecified |
| Leber hereditary optic neuropathy | Point prevalence | 1-9 / 100 000 | United Kingdom | GB | Exact |
| Leber hereditary optic neuropathy | Point prevalence | 1-9 / 100 000 | Worldwide | Not stated | Exact |
| Leber hereditary optic neuropathy | Point prevalence | 1-9 / 100 000 | Denmark | Not stated | Exact |
Showing 5 of 21 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Leber hereditary optic neuropathy | 104 | Exact | Orphadata Science knowledge files, Product 1 |
| Leber hereditary optic neuropathy | 104 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.