Usher Syndromes clinical trials
The registry lists 33 studies for Usher Syndromes across more than 10 countries. 13 are recruiting or about to start. 27 of them are mainly about Usher Syndromes (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-10.
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Who runs them
Laboratoires Thea (5) · Nacuity Pharmaceuticals, Inc. (3) · National Eye Institute (NEI) (3) · Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts (3) · Sanofi (2) · National Center for Research Resources (NCRR) (2) · Sensor Technology for Deafblind (2) · National Institute on Deafness and Other Communication Disorders (NIDCD) (1)
What is being tested here
Drugs under test in Usher Syndromes 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 |
|---|---|---|---|
| NPI-001 | 1 | 1 | 2 |
| N-Acetylcysteinamide | 1 | 1 | 1 |
| Acetylcysteine | 0 | 1 | 1 |
| Ciliary Neurotrophic Factor | 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 2
- Mar 2027Observational Study to Investigate the Short-term Effects of Transcorneal Electrical Stimulation on Visual Performance · Okuvision GmbH
- Jul 2027Stem Cell Ophthalmology Treatment Study II · MD Stem Cells
Who these studies take
Of the 10 Usher Syndromes studies recruiting now, 7 (70%) set no upper age limit and 2 (20%) stop at 75 or younger. 4 (40%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 2 (20%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 4 (40%) | 4 (40%) |
| 18 | 9 (90%) | 9 (90%) |
| 30 | 10 (100%) | 10 (100%) |
| 40 | 10 (100%) | 10 (100%) |
| 50 | 10 (100%) | 10 (100%) |
| 65 | 9 (90%) | 9 (90%) |
| 75 | 9 (90%) | 9 (90%) |
| 85 | 8 (80%) | 8 (80%) |
Across all 33 studies, recruiting or not: 24% stop at 75 or younger and 18% at 65; 58% take children; 0% are for women only and 0% for men only; 15% 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.
Started recruiting1
Studies
Recruiting first, then most recently updated. Showing 33 of 33. search them.
- A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher SyndromeRecruitingNCT07710196Phase 31 country
- A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)RecruitingNCT06789445Phase 1/21 country
- 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
- 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher SyndromeNot yet recruitingNCT07290530Phase 3
- Stem Cell Ophthalmology Treatment Study IIRecruitingNCT03011541Not applicable3 countries
- Inherited Retinal Degenerative Disease RegistryRecruitingNCT024359401 country
- Adaptive Optics Imaging of Outer Retinal DiseasesRecruitingNCT053554151 country
- Observational Study to Investigate the Short-term Effects of Transcorneal Electrical Stimulation on Visual PerformanceRecruitingNCT075489441 country
- Natural History of Photoreceptor Degeneration in USH1B: Clinical Parameters and Validation of Functional Vision Tests in MYO7ARecruitingNCT072788431 country
- Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis PigmentosaRecruitingNCT06591793Phase 1/22 countries
- BF844 Safety and Pharmacokinetic Study in Healthy VolunteersNot yet recruitingNCT06592131Phase 11 country
- Natural History Study of Usher Syndrome ( Light4Deaf )RecruitingNCT046657261 country
- Phenotype Correlates Genotype of Inherited Retina Dystrophies, Retinitis Pigmentosa, Con>Rod Dystrophies.RecruitingNCT039907271 country
- Genetic Analyses of Nonsyndromic and Syndromic Deafness in PakistanCompletedNCT003418741 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
- Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher SyndromeCompletedNCT04355689Phase 1/21 country
- Oral N-acetylcysteine for Retinitis PigmentosaActive, not recruitingNCT05537220Phase 37 countries
- A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1BActive, not recruitingNCT02065011Phase 22 countries
- Auditory Neural Function in Implanted Patients With Usher SyndromeCompletedNCT04906135Not applicable1 country
- 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
- An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis PigmentosaTerminatedNCT05085964Phase 23 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
- Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis PigmentosaCompletedNCT01530659Phase 21 country
- Prospective Open Clinical and Genetic Study of Patients With Retinitis PigmentosaCompletedNCT039013911 country
- Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1BTerminatedNCT01505062Phase 1/22 countries
- 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
- Natural History and Genetic Studies of Usher SyndromeCompletedNCT001067431 country
- Clinical and Genetic Testing of Patients With Usher SyndromeCompletedNCT033195241 country
- Molecular Genetics of Retinal DegenerationsCompletedNCT002310104 countries
- Clinical and Genetic Examination of Usher Syndrome Patients' Cohort in EuropeStatus unknownNCT019549534 countries
- Study of Usher Syndromes, Type 1 and Type 2CompletedNCT000013471 country
- A Genetic Analysis of Usher Syndrome in Ashkenazi JewsCompletedNCT000164711 country
- Study of Dietary N-3 Fatty Acids in Patients With Retinitis Pigmentosa and Usher SyndromeTerminatedNCT000043451 country