Condition (MeSH)
Optic Atrophies, Hereditary
ClinicalTrials.gov lists 63 trials for Optic Atrophies, Hereditary or a narrower condition beneath it.
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Trials per phase
1 more section with rows
5 of the 63 trials are tagged with Optic Atrophies, Hereditary itself; the rest are tagged with a narrower condition beneath it.
When did Optic Atrophies, Hereditary trials start? (19)
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) | 4 |
| 2025 | 4 |
| 2024 | 4 |
| 2023 | 4 |
| 2022 | 1 |
| 2021 | 2 |
| 2019 | 4 |
| 2018 | 6 |
| 2017 | 3 |
| 2016 | 8 |
Showing 10 of 19 earlier years.
No forecast is made for this condition: it averages 3.6 trial starts a year over the five years to September, under the 5 a year the method needs.
Prediction, not a registry value: median of 34 months from start to primary completion across 6 trials still running at the 2026-10-09 snapshot; registered median of 30 months across 33 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Optic Atrophies, Hereditary trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 4 |
| Phase 1/2 | 8 |
| Phase 2 | 10 |
| Phase 2/3 | 3 |
| Phase 3 | 8 |
| Phase 4 | 1 |
| Not applicable | 7 |
| Observational | 19 |
| Expanded access | 3 |
How many Optic Atrophies, Hereditary 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 | 4 |
| Recruiting | 12 |
| Active, not recruiting | 5 |
| Completed | 26 |
| Terminated | 6 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Optic Atrophies, Hereditary trials? (7)
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 |
| PYC Therapeutics | 3 |
| Amgen | 2 |
Showing 5 of 7 sponsors.
Which drugs are being tested for Optic Atrophies, Hereditary? (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 |
| PYC-001 | 2 |
| Retrospective case-control analysis | 1 |
| Nicotinamide treatment | 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 Atrophies, Hereditary 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 Atrophies, Hereditary? (11)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Motor and functional evaluation of patients with spastic paraplegia, optic atrophy, and neuropathy (SPOAN). | Arquivos de neuro-psiquiatria | 2010 | Yes |
| OPA3, mutated in 3-methylglutaconic aciduria type III, encodes two transcripts targeted primarily to mitochondria. | Molecular genetics and metabolism | 2010 | Yes |
| Gene therapy restores vision in a canine model of childhood blindness. | Nature genetics | 2001 | Yes |
| Do idebenone and vitamin therapy shorten the time to achieve visual recovery in Leber hereditary optic neuropathy? | Journal of neuro-ophthalmology : the official journal of the North American Neuro-Ophthalmology Society | 2000 | Yes |
| [Leber optic neuropathy in women and children]. | Der Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft | 1999 | Yes |
Showing 5 of 11 publications.
Sub-conditions in MeSH (3)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence and rare-disease identity.