2 terminated, 1 active not recruiting, 1 not yet recruiting, 1 recruiting. 5 Phase 3, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 5 | Terminated 2, Active not recruiting 1, Not yet recruiting 1, Recruiting 1 |
| Phase 2 | 2 | Terminated 2 |
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 |
|---|---|---|---|---|---|---|
| 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome N-acetylcysteine amide, Placebo - Retinitis Pigmentosa (RP), Usher Syndrome | Phase 3 | Not yet recruiting | Nacuity Pharmaceuticals, Inc. | 80 | 2026-12-01 | 2026-07-01 |
| A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome Placebo, NPI-001 - Retinitis Pigmentosa (RP), Usher Syndrome | Phase 3 | Recruiting | Nacuity Pharmaceuticals, Inc. | 80 | 2026-10-01 | 2026-10-13 |
| Oral N-acetylcysteine for Retinitis Pigmentosa N-acetylcysteine, Placebo - Retinitis Pigmentosa | Phase 3 | Active not recruiting | Johns Hopkins University | 485 | 2023-10-11 | 2026-06-15 |
| 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) QR-421a, Sham-procedure - Retinitis Pigmentosa, Usher Syndrome Type 2 | Phase 2 / Phase 3 | Terminated | Laboratoires Thea | 5 | 2021-12-15 | 2024-08-07 |
| 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) Ultevursen, Sham-procedure - Retinitis Pigmentosa, Usher Syndrome Type 2 | Phase 2 / Phase 3 | Terminated | Laboratoires Thea | 7 | 2021-12-08 | 2024-07-12 |
Retinitis Pigmentosa (5) · Usher Syndromes (5) · Eye Abnormalities (2) · Eye Diseases (2) · Eye Diseases, Hereditary (2) · Retinal Diseases (2) · Vision Disorders (2)
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) · Hearing Loss, Sensorineural (5) · Nervous System Diseases (5) · Neurologic Manifestations (5) · Otorhinolaryngologic Diseases (5) · Pathological Conditions, Signs and Symptoms (5)
Intravitreal Injections (2) · Acetylcysteine (1) · N-Acetylcysteinamide (1)
Laboratoires Thea (2) · Nacuity Pharmaceuticals, Inc. (2) · Johns Hopkins University (1)
United States (4) · United Kingdom (3) · Germany (2) · Netherlands (2) · Austria (1) · Canada (1) · Switzerland (1)
Peter A Campochiaro, MD (1) · Xiangrong Kong (1)
1UG1EY033293 (1)