3 completed, 2 recruiting, 1 terminated, 1 unknown. 7 Phase 1, 6 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 7 | Completed 3, Recruiting 2, Terminated 1, Unknown 1 |
| Phase 2 | 6 | Completed 3, Recruiting 2, Terminated 1 |
Follow Deaf-Blind Disorders: new trials by email Literature Review of Deaf-Blind Disorders trials Research Report on Deaf-Blind Disorders
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO) OpCT-001 - Primary Photoreceptor Disease, Retinitis Pigmentosa (RP) | Phase 1 / Phase 2 | Recruiting | BlueRock Therapeutics | 54 | 2025-03-10 | 2026-10-15 |
| BF844 Safety and Pharmacokinetic Study in Healthy Volunteers BF844 - Usher Syndrome Type 3 | Phase 1 | Unknown | EyeXCel Pty. Ltd. | 76 | 2024-09-09 | 2024-09-19 |
| Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa AAVB-081 - Usher Syndrome, Type 1B | Phase 1 / Phase 2 | Recruiting | AAVantgarde Bio Srl | 15 | 2024-07-02 | 2025-05-02 |
| Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome NPI-001, Placebo - Usher Syndromes | Phase 1 / Phase 2 | Completed | Nacuity Pharmaceuticals, Inc. | 49 | 2020-09-03 | 2026-07-01 |
| Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene QR-421a, Sham-procedure (dose cohort 1&2 only) - Retinitis Pigmentosa, Usher Syndrome Type 2 | Phase 1 / Phase 2 | Completed | ProQR Therapeutics | 20 | 2019-03-06 | 2022-04-20 |
| A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome dantrolene sodium - Wolfram Syndrome, Diabetes Mellitus | Phase 1 / Phase 2 | Completed | Washington University School of Medicine | 21 | 2017-01-01 | 2024-03-13 |
| Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B SAR421869 - Usher Syndrome, Retinitis Pigmentosa | Phase 1 / Phase 2 | Terminated | Sanofi | 9 | 2012-03-26 | 2022-04-28 |
Usher Syndromes (6) · Retinitis Pigmentosa (4) · Ataxia (1) · Cone-Rod Dystrophies (1) · Diabetes Mellitus (1) · Eye Abnormalities (1) · Eye Diseases (1) · Eye Diseases, Hereditary (1) · Optic Atrophy (1) · Retinal Degeneration (1) · Retinal Diseases (1) · Usher syndrome, type 1B (1) · Vision Disorders (1) · Wolfram Syndrome (1)
Abnormalities, Multiple (7) · Blindness (7) · Congenital Abnormalities (7) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Deafness (7) · Ear Diseases (7) · Genetic Diseases, Inborn (7) · Hearing Disorders (7) · Hearing Loss (7) · Nervous System Diseases (7) · Neurologic Manifestations (7) · Otorhinolaryngologic Diseases (7) · Pathological Conditions, Signs and Symptoms (7) · Sensation Disorders (7)
Dantrolene (1) · Intravitreal Injections (1)
AAVantgarde Bio Srl (1) · BlueRock Therapeutics (1) · EyeXCel Pty. Ltd. (1) · Nacuity Pharmaceuticals, Inc. (1) · ProQR Therapeutics (1) · Sanofi (1) · Washington University School of Medicine (1)
United States (4) · Australia (2) · France (2) · Canada (1) · Italy (1) · United Kingdom (1)
Benedict Tan, MBBS, FRACP (1) · Fumihiko Urano, MD (1) · Jose-Alain Sahel, MD, PhD (1) · Lee Anderson, MD (1) · Richard Weleber, MD (1)
PMID 24705452, PMID 31215818, PMID 34185708, PMID 37418643
2018 (1), R21DK113487 (1)