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Choroideremia clinical trials
Every US clinical trial registered for Choroideremia — phase mix, recruiting status, and the sponsors running them, straight from the NIH ClinicalTrials.gov registry.
8 US clinical trials · 3 currently recruiting
The research picture
Choroideremia has 8 registered US clinical trials, 3 of them open to new participants right now — about 38% of the total.
- 3
- recruiting participants now
- 38%
- of trials open to enrollment
- 0
- in Phase 3–4 (later-stage)
- 2
- top sponsor: Ray Therapeutics
Counts reflect the public ClinicalTrials.gov registry as last mirrored by PlainTrial. Status and phase are reported by each study's sponsor. This is reference information, not medical advice.
Active & Recent Trials
Inherited Retinal Degenerative Disease Registry
Foundation Fighting Blindness
NCT02435940
High Resolution Retinal Imaging
University of Pennsylvania
NCT01866371
Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration
Ray Therapeutics
NCT06375239
Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia
4D Molecular Therapeutics
NCT04483440
Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa or Choroideremia Patients (ENVISION)
Ray Therapeutics
NCT06460844
Natural Disease Progression in Participants With Choroideremia
Biogen
NCT04795206
Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations
Spark Therapeutics
NCT02341807
Choroideremia Gene Therapy Clinical Trial
Byron Lam
NCT02553135
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 3 |
| Phase 2 | 1 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Choroideremia Trial Landscape
ClinicalTrials.gov lists 8 US studies indexed under Choroideremia, and 3 of those are currently open to recruitment — roughly 38% of the total volume on the registry. That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
The phase distribution for Choroideremia shows 0 late-stage studies (Phase 3 and Phase 4 combined) alongside 4 earlier-phase entries (Phase 1 through Phase 2). Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
Top sponsor activity for Choroideremia is led by Ray Therapeutics with 2 indexed trials, alongside 6 other organizations in the top contributor list. The list on this page surfaces up to 8 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation — eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Choroideremia?
PlainTrial tracks 8 US clinical trials for Choroideremia, of which 3 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Choroideremia?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology — how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.