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ClinicalTrials.gov 4 recruiting now early-phase tilt small index

Eye Diseases, Hereditary: early-phase trial registry

4 of 11 indexed Eye Diseases, Hereditary trials are currently recruiting, with more weight in Phase 1-2.

Corpus placement: #1,577 of 28,707 conditions by registered trial count.

11 US clinical trials · 4 currently recruiting · #1,577 of 28,707 conditions by registered trial count

Eye Diseases, Hereditary: 11 US clinical trials tracked, 4 recruiting.

Eye Diseases, Hereditary is the subject of 11 registered US clinical trials on ClinicalTrials.gov, 4 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 5 in Phase 1-2 (earlier-stage). The most active sponsor is ProQR Therapeutics, running 2 of these trials.

Enrollment posture brief

Registry condition eye-diseases-hereditary

OPEN 4 | RECR 36% | LATE 1 | EARLY 5 | RANK #1577 | SPON 2

Nearest open-share peer: Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive (36% recruiting · 11 trials)

Acute Pancreatitis81.8%Acute Stroke45.5%Acquired Brain Injury36.4%Eye Diseases, Hereditary (th…36%Acute Lung Injury9.1%
Open-enrollment share among volume-matched conditions near Eye Diseases, Hereditary

Eye Diseases, Hereditary lists 4 open slots (36% of 11 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →

Key findings

What ClinicalTrials.gov does not surface for Eye Diseases, Hereditary on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.

  1. Eye Diseases, Hereditary is recruiting below its size-band peers.

    4 of 11 indexed trials (36.4%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.

  2. One sponsor concentrates Eye Diseases, Hereditary research.

    ProQR Therapeutics accounts for 2 of 11 trials (18.2%). 7 distinct sponsors appear on at least one study for this condition label.

  3. Most Eye Diseases, Hereditary trials plan to enrol fewer than 100 people.

    Median target enrolment is 32 among the 11 trials that report a genuine target (sentinel values excluded). 10 of those (90.9%) plan for fewer than 100 participants.

  4. Eye Diseases, Hereditary splits between interventional and observational designs.

    6 interventional and 5 observational studies make up this index (54.5% interventional). Study type is sponsor-reported on ClinicalTrials.gov.

Phase 12Phase 23Phase 31

Open and recent trials

11 total, page 1 of 1

RECRUITING 20,000 participants

Inherited Retinal Degenerative Disease Registry

Foundation Fighting Blindness

NCT02435940

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

RECRUITING Phase 1 16 participants

A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001

PYC Therapeutics

NCT06852963

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 2
Phase 2 3
Phase 3 1

Named interventions

Most-linked intervention names on Eye Diseases, Hereditary records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.

sepofarsen Drug
2 trials
Sham Injection Procedure
1 trials
Gene Therapy Product-MCO-010 Biological
1 trials
Gene Therapy product-MCO-010 Biological
1 trials
QR-1123 Drug
1 trials
VP-001 Drug
1 trials
No intervention, will not receive any active study intervention Other
1 trials
Intravitreal Injection of Ultevursen Drug
1 trials

US sites by state

Facility locations filed on Eye Diseases, Hereditary trials. A trial with sites in several states counts in each.

Florida 8 trials
Texas 6 trials
California 6 trials
Oregon 5 trials
Michigan 4 trials
Pennsylvania 2 trials
North Dakota 2 trials
Maryland 2 trials

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

What to do with this Eye Diseases, Hereditary page

11 registered trials, 4 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.

  • 4 Eye Diseases, Hereditary trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
  • ProQR Therapeutics sponsors the most Eye Diseases, Hereditary trials on record, review their full trial history before evaluating a specific study. See ProQR Therapeutics's trials
  • Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide

Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Eye Diseases, Hereditary condition card totals 11 registered trials; most-linked intervention sepofarsen; top US site state Florida. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.