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ClinicalTrials.gov 2 recruiting now official registry

Eye Diseases, Hereditary clinical trials

Every US clinical trial registered for Eye Diseases, Hereditary — phase mix, recruiting status, and the sponsors running them, straight from the NIH ClinicalTrials.gov registry.

9 US clinical trials · 2 currently recruiting

The research picture

Eye Diseases, Hereditary has 9 registered US clinical trials, 2 of them open to new participants right now — about 22% of the total.

2
recruiting participants now
22%
of trials open to enrollment
1
in Phase 3–4 (later-stage)
2
top sponsor: ProQR 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

RECRUITING Phase 2 81 participants

Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

Laboratoires Thea

NCT06627179

RECRUITING Phase 3 32 participants

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

Laboratoires Thea

NCT06891443

ACTIVE NOT RECRUITING 50 participants

Natural History of PRPF31 Mutation-Associated Retinal Dystrophy

PYC Therapeutics

NCT05573984

ACTIVE NOT RECRUITING 44 participants

Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F

Jaeb Center for Health Research

NCT04765345

ACTIVE NOT RECRUITING Phase 2 36 participants

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

ProQR Therapeutics

NCT03913143

ACTIVE NOT RECRUITING 18 participants

Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study

Nanoscope Therapeutics

NCT06162585

ACTIVE NOT RECRUITING Phase 1 11 participants

A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene

ProQR Therapeutics

NCT04123626

COMPLETED Phase 2 27 participants

Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]

Nanoscope Therapeutics

NCT04945772

COMPLETED 26 participants

Natural History Study of CEP290-Related Retinal Degeneration

Editas Medicine

NCT03396042

Phase Distribution

PhaseTrial count
Phase 1 1
Phase 2 3
Phase 3 1

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Eye Diseases, Hereditary Trial Landscape

ClinicalTrials.gov lists 9 US studies indexed under Eye Diseases, Hereditary, and 2 of those are currently open to recruitment — roughly 22% 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 Eye Diseases, Hereditary shows 1 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 Eye Diseases, Hereditary is led by ProQR Therapeutics with 2 indexed trials, alongside 5 other organizations in the top contributor list. The list on this page surfaces up to 9 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 Eye Diseases, Hereditary?

PlainTrial tracks 9 US clinical trials for Eye Diseases, Hereditary, of which 2 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Eye Diseases, Hereditary?

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.

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.

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