Condition (MeSH)
Choroideremia
ClinicalTrials.gov lists 34 trials for Choroideremia or a narrower condition beneath it. Biogen has the most among industry and government lead sponsors, with 4.
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Trials per phase
34 of the 34 trials are tagged with Choroideremia itself; the rest are tagged with a narrower condition beneath it.
When did Choroideremia trials start? (15)
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 3
- Not applicable
- Observational
Top of scale: 4
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 |
|---|---|
| 2024 | 3 |
| 2023 | 2 |
| 2022 | 1 |
| 2021 | 2 |
| 2020 | 3 |
| 2018 | 3 |
| 2017 | 3 |
| 2016 | 4 |
| 2015 | 4 |
| 2014 | 1 |
Showing 10 of 15 earlier years.
No forecast is made for this condition: it averages 1.6 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 Choroideremia trials in? (6)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 2 |
| Phase 1/2 | 5 |
| Phase 2 | 4 |
| Phase 3 | 2 |
| Not applicable | 10 |
| Observational | 11 |
How many Choroideremia trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 4 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 2 |
| Completed | 19 |
| Terminated | 3 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Choroideremia trials? (5)
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 |
|---|---|
| Biogen | 4 |
| 4D Molecular Therapeutics | 2 |
| Ray Therapeutics, Inc. | 2 |
| Science Corporation | 2 |
| Intelligent Medical Implants GmbH | 1 |
Which drugs are being tested for Choroideremia? (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 |
|---|---|
| Vitamin A Palmitate | 1 |
| KIO-301 | 1 |
| SIMVASTATIN | 1 |
Interventions as named in the registry (6)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| BIIB111 | 3 |
| rAAV2.REP1 | 2 |
| BIIB112 | 1 |
| 4D-110 | 1 |
| AAV2-hCHM | 1 |
Showing 5 of 6 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 Choroideremia 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 |
|---|---|---|
| Visual acuity reduced | 35 | 3 |
| Macular hole | 4 | 3 |
| Noninfective retinitis | 3 | 2 |
| Abscess limb | 1 | 1 |
| Angina pectoris | 1 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Choroideremia? (15)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| An Open-Label Phase II Study Assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia: The GEMINI Study. | Human gene therapy | 2024 | Yes |
| Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial. | Ophthalmology | 2022 | Yes |
| DANON DISEASE: A MODEL OF PHOTORECEPTOR DEGENERATION SECONDARY TO PRIMARY RETINAL PIGMENT EPITHELIUM DISEASE. | Retinal cases & brief reports | 2022 | Yes |
| Participant perspectives on a phase I/II ocular gene therapy trial (NCT02077361). | Ophthalmic genetics | 2019 | Yes |
| Measurement and Reproducibility of Preserved Ellipsoid Zone Area and Preserved Retinal Pigment Epithelium Area in Eyes With Choroideremia. | American journal of ophthalmology | 2017 | Yes |
Showing 5 of 15 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 |
|---|---|---|---|---|---|
| Choroideremia | Point prevalence | 1-9 / 100 000 | Europe | Not stated | Exact |
| Choroideremia | Point prevalence | 1-9 / 100 000 | Europe | Not stated | Exact |
| Choroideremia | Point prevalence | 1-9 / 100 000 | Europe | Not stated | Unspecified |
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Choroideremia | 180 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Choroideremia | 180 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.