Retinal Dystrophies clinical trials
The registry lists 60 studies for Retinal Dystrophies across more than 10 countries. 20 are recruiting or about to start. 28 of them are mainly about Retinal Dystrophies (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12. Counting its 3 narrower conditions too, such as Retinitis Pigmentosa: 343 studies, 89 open.
Get an email when these change. Save the Retinal Dystrophies studies recruiting or about to start — once a week, free: studies that open, close, add sites or post results.
See an example email
Example · made-up studies
Breast cancer · Recruiting: 2 came in, 3 changed
What changed in your saved searches, Oct 31 – Nov 6:
A study of drug A after surgery for early breast cancer
Came in · now Recruiting
Drug B with hormone therapy in advanced breast cancer
Changed
· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
Exercise during chemotherapy
Now Active, not recruiting
One email a week for all your saved searches. Stop any time.
Who runs them
Nanoscope Therapeutics Inc. (5) · PYC Therapeutics (4) · Innostellar Biotherapeutics Co.,Ltd (3) · Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine (2) · SparingVision (2) · University of Michigan (2) · University College, London (2) · MD Stem Cells (2)
What is being tested here
Drugs under test in Retinal Dystrophies 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 |
|---|---|---|---|
| Disulfiram | 1 | 0 | 1 |
| VP-001 | 0 | 0 | 3 |
| Metformin | 0 | 0 | 1 |
| Ranibizumab | 0 | 0 | 1 |
| Sepofarsen | 0 | 1 | 1 |
| Brolucizumab | 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.
The 10 nearest
- Dec 2026EyeConic: Qualification for Cone-Optogenetics · Institute of Molecular and Clinical Ophthalmology Basel
- Dec 2026A Study of LX107 Gene Therapy in AIPL1-IRD Patients · Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
- Dec 2026Evaluation of Objective Perimetry Using Chromatic Multifocal Pupillometer · Sheba Medical Center
- Feb 2027Promising ROd-cone DYstrophy Gene therapY · SparingVision
- Feb 2027Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD) · Ray Therapeutics, Inc.
- Feb 2027Disulfiram for Treatment of Retinal Degeneration · University of Washington
- Mar 2027Natural History Study in Patients With PDE6A-, PDE6B- and RHO-linked Retinitis Pigmentosa · University Hospital Tuebingen
- Mar 2027Observational Study to Investigate the Short-term Effects of Transcorneal Electrical Stimulation on Visual Performance · Okuvision GmbH
- May 2027Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study · Nanoscope Therapeutics Inc.
- Jun 2027Natural History Study of Inherited Retinal Diseases · Zhongmou Therapeutics
Who these studies take
Of the 17 Retinal Dystrophies studies recruiting now, 11 (65%) set no upper age limit and 3 (18%) stop at 75 or younger. 6 (35%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 5 (29%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 6 (35%) | 6 (35%) |
| 18 | 17 (100%) | 17 (100%) |
| 30 | 17 (100%) | 17 (100%) |
| 40 | 17 (100%) | 17 (100%) |
| 50 | 16 (94%) | 16 (94%) |
| 65 | 15 (88%) | 15 (88%) |
| 75 | 14 (82%) | 14 (82%) |
| 85 | 13 (76%) | 13 (76%) |
Across all 60 studies, recruiting or not: 20% stop at 75 or younger and 15% at 65; 53% take children; 2% are for women only and 2% for men only; 22% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 50 of 60. Browse all 60 → or search them.
- Familial Mediterranean Fever and Related Disorders: Genetics and Disease CharacteristicsRecruitingNCT000013731 country
- The Effects of Disulfiram (Antabuse®) on Visual Acuity in Patients With Retinal DegenerationRecruitingNCT06319872Phase 11 country
- Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy (SPVN20) in Subjects With Rod-Cone DystrophyRecruitingNCT07807111Phase 1/23 countries
- Stem Cell Ophthalmology Treatment Study IIRecruitingNCT03011541Not applicable3 countries
- Safety and Efficacy Study of PUMCH-E111 Injection in Subjects With RLBP1 Related Inherited Retinal DystrophyRecruitingNCT07656753Early Phase 11 country
- Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome PatientsRecruitingNCT06395285Phase 13 countries
- Disulfiram for Treatment of Retinal DegenerationRecruitingNCT05626920Phase 1/21 country
- Observational Study to Investigate the Short-term Effects of Transcorneal Electrical Stimulation on Visual PerformanceRecruitingNCT075489441 country
- Real-time Acoustic Biofeedback for Enhancing Fixation Stability: A Proof-of-concept Study to Improve Ophthalmic Imaging Diagnostic QualityEnrolling by invitationNCT07529041Not applicable1 country
- Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD)RecruitingNCT075026641 country
- A Study of LX107 Gene Therapy in AIPL1-IRD PatientsRecruitingNCT07063030Early Phase 11 country
- VIsual Cerebral ConnecTivity On Functional Magnetic Resonance Imaging in Patients With Hereditary REtinal DystrophiesRecruitingNCT046481241 country
- Natural History Study of Inherited Retinal DiseasesRecruitingNCT070855331 country
- EyeConic: Qualification for Cone-OptogeneticsRecruitingNCT052949787 countries
- Functional Assessments in Vision ImpairmentNot yet recruitingNCT069081611 country
- Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal DegenerationRecruitingNCT063752391 country
- Evaluation of Objective Perimetry Using Chromatic Multifocal PupillometerRecruitingNCT020143891 country
- Coloured-light in Retinitis Pigmentosa.Not yet recruitingNCT06224114Not applicable1 country
- Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal DystrophyRecruitingNCT05858983Phase 1/21 country
- Phenotype Correlates Genotype of Inherited Retina Dystrophies, Retinitis Pigmentosa, Con>Rod Dystrophies.RecruitingNCT039907271 country
- Oral Metformin for Treatment of ABCA4 RetinopathyActive, not recruitingNCT04545736Phase 1/21 country
- SS-HH-OCT as a Novel Diagnostic Modality for Early-Onset Retinal Dystrophies (EORDs)CompletedNCT06177977Not applicable1 country
- Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal DystrophyCompletedNCT04516369Phase 31 country
- SAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy SubjectsCompletedNCT05902962Phase 11 country
- 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
- MAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (Wallaby)CompletedNCT06455826Phase 11 country
- Study of New Mutations in Cone DisordersTerminatedNCT046582511 country
- Promising ROd-cone DYstrophy Gene therapYActive, not recruitingNCT05748873Phase 1/22 countries
- Natural History Study in Patients With PDE6A-, PDE6B- and RHO-linked Retinitis PigmentosaActive, not recruitingNCT063237721 country
- Natural History of PRPF31 Mutation-Associated Retinal DystrophyActive, not recruitingNCT055739842 countries
- Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 MutationsActive, not recruitingNCT06196827Phase 11 country
- Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 MutationsActive, not recruitingNCT07054632Phase 31 country
- The Natural History of Mucolipidosis Type IVTerminatedNCT010677421 country
- Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B GeneTerminatedNCT03328130Phase 1/21 country
- Prospective Analysis of "Genotype-phenotype" Correlations Observed in a Large Cohort of Patients With Hereditary Retinal Dystrophies - GEPHIRDTerminatedNCT036623861 country
- Personalized Antisense Oligonucleotide Therapy for a Single Participant With PRPH2 Mutation Associated With Retinal DystrophyActive, not recruitingNCT07177196Phase 1/21 country
- iPS Cells of Patients for Models of Retinal DystrophiesCompletedNCT03853252Not applicable1 country
- Fellow-eye Study (FE) of LX101 in Subjects With Inherited Retinal DystrophyActive, not recruitingNCT06212297Phase 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
- Performance of Long-wavelength Autofluorescence ImagingCompletedNCT03592017Not applicable1 country
- Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]CompletedNCT04945772Phase 22 countries
- Retinal Imaging by Adaptive Optics in Healthy Eyes and During Retinal and General DiseasesCompletedNCT015461811 country
- Observational Study to Assess the Reliability and Validity of the MLYMT and MLSDTCompletedNCT058201002 countries
- Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt DiseaseCompletedNCT05417126Phase 21 country
- Natural History of the Progression of Choroideremia StudyCompletedNCT033595519 countries
- High Resolution Optical Coherence TomographyStatus unknownNCT051303851 country
- 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
- Dual Wavelength OCTCompletedNCT038438401 country
- An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.Status unknownNCT04855045Phase 2/37 countries