5 completed, 4 terminated, 3 active not recruiting, 2 recruiting. 17 Phase 2, 6 Phase 1, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 17 | Completed 5, Terminated 4, Active not recruiting 3, Recruiting 2, Unknown 2, Not yet recruiting 1 |
| Phase 1 | 6 | Completed 3, Recruiting 2, Terminated 1 |
| Phase 3 | 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 |
|---|---|---|---|---|---|---|
| Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene No intervention, will not receive any active study intervention, Intravitreal Injection of Ultevursen - Retinitis Pigmentosa (RP), Usher Syndrome Type 2 | Phase 2 | Not yet recruiting | Laboratoires Thea | 81 | 2026-10-05 | 2026-09-01 |
| 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 |
| Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene Intravitreal Injection of Ultevursen, No intervention, will not receive any active study intervention - Retinitis Pigmentosa (RP), Usher Syndrome Type 2 | Phase 2 | Active not recruiting | Laboratoires Thea | 81 | 2024-12-11 | 2026-07-09 |
| 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 |
| Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1 Tirzepatide - Wolfram Syndrome | Phase 2 | Unknown | Ospedale San Raffaele | 10 | 2024-01-01 | 2024-02-22 |
| AMX0035 in Adult Patients With Wolfram Syndrome AMX0035 - Wolfram Syndrome | Phase 2 | Active not recruiting | Amylyx Pharmaceuticals Inc. | 12 | 2023-03-03 | 2026-04-14 |
| 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 |
| Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome Depakine - Wolfram Syndrome | Phase 2 | Unknown | Centre d'Etude des Cellules Souches | 23 | 2021-11-26 | 2023-11-28 |
| An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa RNA antisense oligonucleotide for intravitreal injection - Retinitis Pigmentosa, Usher Syndrome Type 2 | Phase 2 | Terminated | Laboratoires Thea | 21 | 2021-09-16 | 2024-08-07 |
| 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 |
| A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B Blood draw for the laboratory assessment - Usher's Syndrome | Phase 2 | Active not recruiting | Sanofi | 9 | 2013-09-12 | 2026-01-13 |
| 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 |
| 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 (12) · Retinitis Pigmentosa (10) · Retinal Diseases (6) · Vision Disorders (6) · Eye Abnormalities (5) · Eye Diseases, Hereditary (5) · Wolfram Syndrome (5) · Eye Diseases (4) · Diabetes Mellitus (2) · Ataxia (1) · Cone-Rod Dystrophies (1) · Genetic Diseases, Inborn (1) · Night Blindness (1) · Optic Atrophy (1) · Retinal Degeneration (1)
Abnormalities, Multiple (17) · Blindness (17) · Congenital Abnormalities (17) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (17) · Deafness (17) · Ear Diseases (17) · Hearing Disorders (17) · Hearing Loss (17) · Nervous System Diseases (17) · Neurologic Manifestations (17) · Otorhinolaryngologic Diseases (17) · Sensation Disorders (17) · Genetic Diseases, Inborn (16) · Eye Diseases (13)
Intravitreal Injections (4) · Valproic Acid (2) · 4-phenylbutyric acid (1) · Blood Specimen Collection (1) · Ciliary Neurotrophic Factor (1) · Dantrolene (1) · Electroconvulsive Therapy (1) · Tirzepatide (1) · salicylhydroxamic acid (1) · sodium phenylbutyrate and taurursodiol (1)
Laboratoires Thea (5) · Sanofi (2) · AAVantgarde Bio Srl (1) · Amylyx Pharmaceuticals Inc. (1) · BlueRock Therapeutics (1) · Centre d'Etude des Cellules Souches (1) · Nacuity Pharmaceuticals, Inc. (1) · Neurotech Pharmaceuticals (1) · Ospedale San Raffaele (1) · ProQR Therapeutics (1) · University of Birmingham (1) · Washington University School of Medicine (1)
United States (11) · France (7) · United Kingdom (5) · Canada (3) · Italy (3) · Germany (2) · Netherlands (2) · Spain (2) · Australia (1) · Belgium (1) · Brazil (1) · Denmark (1) · Poland (1)
Camille Bedrosian, MD (1) · Fumihiko Urano, MD (1) · Jacque Duncan, MD (1) · Jose-Alain Sahel, MD, PhD (1) · Lee Anderson, MD (1) · Lorenzo Piemonti, MD (1) · Richard Weleber, MD (1) · Timothy Barrett, PhD, MB, BS (1)
PMID 24705452, PMID 31215818, PMID 34185708, PMID 37418643, PMID 40010822, PMID 40339601
2018 (1), R21DK113487 (1)