Condition (MeSH)
Stargardt Disease
ClinicalTrials.gov lists 66 trials for Stargardt Disease or a narrower condition beneath it. Astellas Institute for Regenerative Medicine has the most among industry and government lead sponsors, with 5.
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Trials per phase
66 of the 66 trials are tagged with Stargardt Disease itself; the rest are tagged with a narrower condition beneath it.
When did Stargardt Disease trials start? (18)
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
- Not applicable
- Observational
Top of scale: 7
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) | 11 |
| 2025 | 6 |
| 2024 | 7 |
| 2023 | 3 |
| 2022 | 4 |
| 2021 | 2 |
| 2020 | 2 |
| 2019 | 3 |
| 2018 | 5 |
| 2017 | 2 |
Showing 10 of 18 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 13 trials starting in 2026 and about 9 in 2027, the average of the 24 months through September, counted as registered and carried forward. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.
Prediction, not a registry value: median of 36 months from start to primary completion across 15 trials still running at the 2026-10-09 snapshot; registered median of 41 months across 24 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Stargardt Disease trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 10 |
| Phase 1/2 | 13 |
| Phase 2 | 7 |
| Phase 2/3 | 2 |
| Phase 3 | 3 |
| Not applicable | 8 |
| Observational | 23 |
How many Stargardt Disease 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 | 3 |
| Recruiting | 22 |
| Active, not recruiting | 10 |
| Completed | 23 |
| Terminated | 3 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Stargardt Disease trials? (11)
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 |
|---|---|
| Astellas Institute for Regenerative Medicine | 5 |
| Alkeus Pharmaceuticals, Inc. | 4 |
| Nanoscope Therapeutics Inc. | 3 |
| National Eye Institute (NEI) | 3 |
| Ray Therapeutics, Inc. | 3 |
Showing 5 of 11 sponsors.
Which drugs are being tested for Stargardt Disease? (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 |
|---|---|
| ALK-001 | 4 |
| Tinlarebant | 3 |
| Emixustat | 2 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| MA09-hRPE | 3 |
| NaCl 0.9% NS | 1 |
| OCU410ST | 1 |
| Gene Therapy-vMCO-010 | 1 |
| Docosahexaenoic Acid (DHA) Dietary Supplement | 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 Stargardt Disease 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 |
|---|---|---|
| neoplasm | 3 | 1 |
| Infections and infestations | 2 | 1 |
| agina pectoris | 2 | 1 |
| Chorioretinopathy | 1 | 1 |
| Endophthalmitis | 1 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Stargardt Disease? (25)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Retinal light sensitivity as outcome measure in recessive Stargardt disease. | The British journal of ophthalmology | 2021 | Yes |
| Long-term safety and tolerability of subretinal transplantation of embryonic stem cell-derived retinal pigment epithelium in Asian Stargardt disease patients. | The British journal of ophthalmology | 2021 | Yes |
| Antioxidant Saffron and Central Retinal Function in ABCA4-Related Stargardt Macular Dystrophy. | Nutrients | 2019 | Yes |
| Perceptual learning in patients with Stargardt disease. | Canadian journal of ophthalmology. Journal canadien d'ophtalmologie | 2019 | Yes |
| Randomised study evaluating the pharmacodynamics of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease. | The British journal of ophthalmology | 2022 | No |
Showing 5 of 25 publications.
Prevalence (3)
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 |
|---|---|---|---|---|---|
| Stargardt disease | Point prevalence | 1-5 / 10 000 | Europe | Not stated | Exact |
| Stargardt disease | Point prevalence | 1-5 / 10 000 | Europe | Not stated | Unspecified |
| Stargardt disease | Point prevalence | 1-5 / 10 000 | Europe | Not stated | Exact |
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Stargardt disease | 827 | Exact | Orphadata Science knowledge files, Product 1 |
| Stargardt disease | 827 | 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.