Condition (MeSH)
Leber Congenital Amaurosis
ClinicalTrials.gov lists 32 trials for Leber Congenital Amaurosis or a narrower condition beneath it. Spark Therapeutics, Inc. has the most among industry and government lead sponsors, with 4.
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Trials per phase
32 of the 32 trials are tagged with Leber Congenital Amaurosis itself; the rest are tagged with a narrower condition beneath it.
When did Leber Congenital Amaurosis trials start? (16)
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 2/3
- Phase 3
- Not applicable
- Observational
Top of scale: 5
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) | 1 |
| 2025 | 2 |
| 2023 | 2 |
| 2022 | 1 |
| 2021 | 1 |
| 2019 | 5 |
| 2018 | 1 |
| 2017 | 1 |
| 2016 | 3 |
| 2015 | 2 |
Showing 10 of 16 earlier years.
No forecast is made for this condition: it averages 1.2 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 Leber Congenital Amaurosis trials in? (6)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 7 |
| Phase 1/2 | 11 |
| Phase 2/3 | 2 |
| Phase 3 | 2 |
| Not applicable | 1 |
| Observational | 9 |
How many Leber Congenital Amaurosis trials are recruiting? (6)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 1 |
| Recruiting | 4 |
| Active, not recruiting | 8 |
| Completed | 15 |
| Terminated | 1 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Leber Congenital Amaurosis trials? (12)
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 |
|---|---|
| Spark Therapeutics, Inc. | 4 |
| MeiraGTx UK II Ltd | 3 |
| QLT Inc. | 3 |
| Editas Medicine, Inc. | 2 |
| Laboratoires Thea | 2 |
Showing 5 of 12 sponsors.
Which drugs are being tested for Leber Congenital Amaurosis? (3)
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 |
|---|---|
| sepofarsen | 4 |
| voretigene neparvovec-rzyl | 3 |
| QLT091001 | 2 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| HG004 | 2 |
| AAV2-hRPE65v2,voretigene neparvovec-rzyl | 2 |
| ATSN-101 Diluent Solution | 1 |
| 1% Prednisolone | 1 |
| Control (placebo) group | 1 |
Showing 5 of 12 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 Leber Congenital Amaurosis trials report? (8)
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 |
|---|---|---|
| Uveitis | 3 | 1 |
| Adverse Drug Reaction | 2 | 1 |
| Convulsion | 1 | 1 |
| Endopthalmitis | 1 | 1 |
| Lumbar vertebral fracture | 1 | 1 |
Showing 5 of 8 adverse events.
Which publications cover Leber Congenital Amaurosis? (24)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial. | Nature medicine | 2022 | Yes |
| Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report. | Nature medicine | 2021 | Yes |
| Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect. | Nature medicine | 2019 | Yes |
| Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy. | Human gene therapy | 2018 | Yes |
| A Cross-Sectional and Longitudinal Study of Retinal Sensitivity in RPE65-Associated Leber Congenital Amaurosis. | Investigative ophthalmology & visual science | 2018 | Yes |
Showing 5 of 24 publications.
Prevalence (12)
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 congenital amaurosis | Point prevalence | 1-9 / 100 000 | United States | US | Exact |
| Leber congenital amaurosis | Point prevalence | 1-9 / 100 000 | United States | US | Unspecified |
| Leber congenital amaurosis | Point prevalence | 1-9 / 100 000 | United States | US | Exact |
| Leber congenital amaurosis | Prevalence at birth | 1-9 / 100 000 | Worldwide | Not stated | Exact |
| Leber congenital amaurosis | Point prevalence | 1-9 / 100 000 | Europe | Not stated | Unspecified |
Showing 5 of 12 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Leber congenital amaurosis | 65 | Exact | Orphadata Science knowledge files, Product 1 |
| Leber congenital amaurosis | 65 | 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.