Eye Diseases, Hereditary clinical trials
The registry lists 39 studies for Eye Diseases, Hereditary across more than 10 countries. 4 are recruiting or about to start. 5 of them are mainly about Eye Diseases, Hereditary (how this is decided). Per ClinicalTrials.gov, last updated 2026-10-26. Counting its 22 narrower conditions too, such as Macular Degeneration: 3,275 studies, 569 open.
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Who runs them
GenSight Biologics (7) · Laboratoires Thea (6) · Nanoscope Therapeutics Inc. (4) · ProQR Therapeutics (3) · Sun Yat-sen University (2) · National Eye Institute (NEI) (2) · Editas Medicine, Inc. (2) · PYC Therapeutics (2)
What is being tested here
Drugs under test in Eye Diseases, Hereditary studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Sepofarsen | 1 | 2 | 3 |
| Doxycycline | 0 | 0 | 2 |
| VP-001 | 0 | 0 | 1 |
| Topotecan Sustained-Release Episcleral Plaque | 0 | 0 | 1 |
| Valproic Acid | 0 | 0 | 1 |
| Bevacizumab | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 6, phase 3 first
- Nov 2027Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) · Phase 3 · Laboratoires Thea
- Mar 2027Natural History Study in Patients With PDE6A-, PDE6B- and RHO-linked Retinitis Pigmentosa · University Hospital Tuebingen
- May 2027Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study · Nanoscope Therapeutics Inc.
- Jun 2027Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F · Jaeb Center for Health Research
- Jul 2027Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study · Nanoscope Therapeutics Inc.
- Oct 2027Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa · GenSight Biologics
Who these studies take
Of the 3 Eye Diseases, Hereditary studies recruiting now, 3 (100%) set no upper age limit and 0 (0%) stop at 75 or younger. 2 (67%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 2 (67%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 2 (67%) | 2 (67%) |
| 18 | 3 (100%) | 3 (100%) |
| 30 | 3 (100%) | 3 (100%) |
| 40 | 3 (100%) | 3 (100%) |
| 50 | 3 (100%) | 3 (100%) |
| 65 | 3 (100%) | 3 (100%) |
| 75 | 3 (100%) | 3 (100%) |
| 85 | 3 (100%) | 3 (100%) |
Across all 39 studies, recruiting or not: 10% stop at 75 or younger and 5% at 65; 69% take children; 0% are for women only and 0% for men only; 8% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 13, 2026.
Newly registered1
Studies
Recruiting first, then most recently updated. Showing 39 of 39. search them.
- Evaluation of ETHY-001 for Safety, Tolerability, and Clinical Activity in Thyroid Eye DiseaseRecruitingNCT07885605Phase 11 country
- Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)RecruitingNCT06891443Phase 310 countries
- Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A GeneNot yet recruitingNCT07796646Phase 2
- Inherited Retinal Degenerative Disease RegistryRecruitingNCT024359401 country
- Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A GeneActive, not recruitingNCT06627179Phase 210 countries
- 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 VP001Active, not recruitingNCT06852963Phase 1/21 country
- Natural History Study in Patients With PDE6A-, PDE6B- and RHO-linked Retinitis PigmentosaActive, not recruitingNCT063237721 country
- Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 YearCompletedNCT03293524Phase 37 countries
- Natural History of PRPF31 Mutation-Associated Retinal DystrophyActive, not recruitingNCT055739842 countries
- EAP_GS010_single PatientNo longer availableNCT03672968
- Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis PigmentosaActive, not recruitingNCT03326336Phase 1/23 countries
- Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)CompletedNCT02652780Phase 35 countries
- RESCUE and REVERSE Long-term Follow-upCompletedNCT03406104Phase 35 countries
- Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 MutationCompletedNCT02652767Phase 35 countries
- REALITY LHON RegistryCompletedNCT032950715 countries
- Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1FActive, not recruitingNCT047653458 countries
- Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B GeneTerminatedNCT03328130Phase 1/21 country
- Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE StudyActive, not recruitingNCT061625852 countries
- Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT StudyActive, not recruitingNCT060481851 country
- Topotecan Episcleral Plaque for Treatment of RetinoblastomaCompletedNCT04428879Phase 11 country
- Extension Study to Study PQ-110-001 (NCT03140969)TerminatedNCT03913130Phase 1/22 countries
- Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)TerminatedNCT05176717Phase 2/32 countries
- Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)TerminatedNCT05158296Phase 2/34 countries
- Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)CompletedNCT029468792 countries
- Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]CompletedNCT04945772Phase 22 countries
- Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt DiseaseCompletedNCT05417126Phase 21 country
- Single Ascending Dose Study in Participants With LCA10Status unknownNCT03872479Phase 1/21 country
- Natural History Study of CEP290-Related Retinal DegenerationCompletedNCT033960424 countries
- 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 GeneActive, not recruitingNCT04123626Phase 1/21 country
- Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A GeneCompletedNCT03780257Phase 1/23 countries
- A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)Active, not recruitingNCT03913143Phase 2/39 countries
- Doxycycline Treatment in Mild Thyroid-Associated OphthalmopathyStatus unknownNCT02203682Phase 21 country
- Safety and Efficacy of Subretinal Implants for Partial Restoration of Vision in Blind PatientsCompletedNCT01024803Not applicable3 countries
- Evaluation and Treatment of Patients With Inherited Eye DiseasesCompletedNCT000118171 country
- Screening for Studies on Inherited Eye DiseasesCompletedNCT000017321 country
- Efficacy and Safety of Oral Valproic Acid for Retinitis PigmentosaCompletedNCT01399515Phase 21 country
- Intravitreal Bevacizumab vs.Combination Therapy for CNV Due to Other Than AMDWithdrawnNCT01256580Not applicable
- Efficacy of Subantimicrobial Dose Doxycycline for Moderate to Severe and Active Graves' OrbitopathyCompletedNCT01727973Phase 1/21 country
- Fundus Changes in the Microphthalmy EyesCompletedNCT00811512