5 completed. 5 Phase 2, 3 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 5 |
| Phase 1 | 3 | Completed 3 |
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 |
|---|---|---|---|---|---|---|
| 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 |
| Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome Sodium Valproate 200Mg E/C Tablet, Sodium Valproate matched placebo - Wolfram Syndrome | Phase 2 | Completed | University of Birmingham | 63 | 2018-12-28 | 2026-05-05 |
| 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 |
| Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa NT-501, Sham - Retinitis Pigmentosa, Usher Syndrome Type 2 | Phase 2 | Completed | Neurotech Pharmaceuticals | 22 | 2012-01-01 | 2023-02-14 |
Usher Syndromes (3) · Retinitis Pigmentosa (2) · Wolfram Syndrome (2) · Ataxia (1) · Diabetes Mellitus (1) · Eye Abnormalities (1) · Eye Diseases (1) · Eye Diseases, Hereditary (1) · Optic Atrophy (1) · Retinal Diseases (1) · Vision Disorders (1)
Abnormalities, Multiple (5) · Blindness (5) · Congenital Abnormalities (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Deafness (5) · Ear Diseases (5) · Genetic Diseases, Inborn (5) · Hearing Disorders (5) · Hearing Loss (5) · Nervous System Diseases (5) · Neurologic Manifestations (5) · Otorhinolaryngologic Diseases (5) · Sensation Disorders (5) · Eye Diseases (4)
Ciliary Neurotrophic Factor (1) · Dantrolene (1) · Electroconvulsive Therapy (1) · Intravitreal Injections (1) · Valproic Acid (1) · salicylhydroxamic acid (1)
Nacuity Pharmaceuticals, Inc. (1) · Neurotech Pharmaceuticals (1) · ProQR Therapeutics (1) · University of Birmingham (1) · Washington University School of Medicine (1)
United States (3) · France (2) · Australia (1) · Canada (1) · Poland (1) · Spain (1) · United Kingdom (1)
Fumihiko Urano, MD (1) · Jacque Duncan, MD (1) · Lee Anderson, MD (1) · Timothy Barrett, PhD, MB, BS (1)
PMID 31215818, PMID 34185708, PMID 37418643, PMID 40010822
2018 (1), R21DK113487 (1)