19 completed, 13 recruiting, 7 unknown, 5 not yet recruiting. 17 Phase 2, 7 Phase 1, 6 Not applicable, 5 Phase 3, 1 Early Phase 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 |
|---|---|---|---|---|---|---|
| 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 |
| SENSE-Brain Health Pro for Older Adults With Sensory Loss Brain Health Pro - Vision and Hearing Loss, Vision Impairment | Not applicable | Not yet recruiting | Université de Montréal | 300 | 2026-10-15 | 2026-10-05 |
| 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 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 |
| Observational Study to Investigate the Short-term Effects of Transcorneal Electrical Stimulation on Visual Performance Transcorneal Electrical Stimulation using the OkuStim System - Retinitis Pigmentosa (RP), Usher Syndrome | Recruiting | Okuvision GmbH | 30 | 2026-04-20 | 2026-05-01 | |
| Characterization and Contribution of Genome-wide DNA Methylation (DNA Methylation Episignatures) in Rare Diseases With Prenatal Onset Methylation analysis - Rare Fetal Genetic Diseases, Congenital Malformation | Recruiting | Assistance Publique - Hôpitaux de Paris | 63 | 2026-02-26 | 2026-03-27 | |
| Does Recessive Optic Atrophy Due to WFS1 Exist? analyse study - Wolfram Syndrome 1, Optic Atrophies, Hereditary | Not yet recruiting | Hôpital Necker-Enfants Malades | 45 | 2026-02-01 | 2026-01-13 | |
| Mechanisms of Fecal Incontinence in Wolfram Syndrome Retrospective case-control analysis - Wolfram Syndrome, Fecal Incontinence | Not yet recruiting | Hôpital Necker-Enfants Malades | 20 | 2025-12-20 | 2025-12-31 | |
| A Strategic Approach to Safe and Effective Cochlear Implantation in Patients With CHARGE Syndrome No intervention (observational study) - CHARGE Syndrome | Completed | Seoul National University Bundang Hospital | 12 | 2025-11-21 | 2026-01-06 | |
| Natural History of Photoreceptor Degeneration in USH1B: Clinical Parameters and Validation of Functional Vision Tests in MYO7A Vision tests, Retinal imaging, Questionnaires - Usher Syndrome | Recruiting | Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts | 60 | 2025-10-13 | 2026-02-10 | |
| Palliative Care Needs of Children With Rare Diseases and Their Families Family Centered pediatric palliative care for family caregivers of children with rare diseases. - Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia | Not applicable | Enrolling by invitation | Children's National Research Institute | 480 | 2025-10-02 | 2026-01-28 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Health Determinants in Older Adults Living At Home Health promoting assessment of older adults living at home - Frailty, Vision and Hearing Loss | Completed | University of Stavanger | 40 | 2023-03-01 | 2024-12-12 | |
| Validating a Self-fitting Hearing Aid Fitting algorithm - Hearing and Vision Loss | Not applicable | Completed | University of Minnesota | 41 | 2022-03-17 | 2023-04-25 |
Showing 20 of 55. Sign in to see all of them.
Usher Syndromes (33) · Retinitis Pigmentosa (26) · Wolfram Syndrome (12) · Retinal Diseases (9) · Vision Disorders (8) · Diabetes Mellitus (6) · Eye Diseases (6) · Eye Diseases, Hereditary (6) · CHARGE Syndrome (5) · Cone-Rod Dystrophies (5) · Deafness (5) · Eye Abnormalities (5) · Cone Dystrophy (4) · Optic Atrophy (4)
Abnormalities, Multiple (55) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (55) · Ear Diseases (55) · Nervous System Diseases (55) · Neurologic Manifestations (55) · Otorhinolaryngologic Diseases (55) · Sensation Disorders (55) · Congenital Abnormalities (54) · Hearing Disorders (54) · Hearing Loss (53) · Blindness (52) · Deafness (50) · Eye Diseases (49) · Genetic Diseases, Inborn (48)
Intravitreal Injections (4) · Valproic Acid (2) · 4-phenylbutyric acid (1) · Acetylcysteine (1) · Blood Specimen Collection (1) · Ciliary Neurotrophic Factor (1) · Dantrolene (1) · Electroconvulsive Therapy (1) · Exenatide (1) · Exome (1) · Genotype (1) · Hearing (1) · N-Acetylcysteinamide (1) · Observation (1) · Optical Imaging (1)
Laboratoires Thea (5) · Washington University School of Medicine (4) · Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts (3) · Nacuity Pharmaceuticals, Inc. (3) · National Eye Institute (NEI) (3) · Hôpital Necker-Enfants Malades (2) · National Center for Research Resources (NCRR) (2) · Sanofi (2) · Sensor Technology for Deafblind (2) · AAVantgarde Bio Srl (1) · Amylyx Pharmaceuticals Inc. (1) · Assistance Publique - Hôpitaux de Paris (1) · BlueRock Therapeutics (1) · Centre d'Etude des Cellules Souches (1) · Children's National Research Institute (1)
United States (28) · France (12) · Canada (6) · United Kingdom (6) · Germany (5) · Netherlands (5) · Italy (4) · Australia (2) · Austria (2) · Denmark (2) · Pakistan (2) · Russia (2) · Spain (2) · Belgium (1) · Brazil (1)
Benedict Tan, MBBS, FRACP (1) · Camille Bedrosian, MD (1) · Daniel X Hammer, Ph.D. (1) · Dmitry S. Atarshchikov, MD, PhD (1) · Finn M. Radtke, Professor (1) · Fumihiko Urano, MD (1) · Gil Leibowitz, MD (1) · Giuseppe Zampino, medicine (1) · Grethe Eilertsen, PhD (1) · Isabelle Audo, Pr (1)
PMID 24705452, PMID 26025012, PMID 26199618, PMID 26604914, PMID 26742931, PMID 27459537, PMID 27857759, PMID 27904503, PMID 29270420, PMID 30050918, PMID 30358468, PMID 31119146, PMID 31215818, PMID 31620478, PMID 32039263, PMID 32231088, PMID 32573764, PMID 33546345, PMID 34185708, PMID 37418643, PMID 38635319, PMID 38972091, PMID 40010822, PMID 40339601, PMID 41282901, PMID 42635483
1UG1EY033293 (1), 2018 (1), R01DK132090 (1), R01HD070855 (1), R01HD117137 (1), R21DC019458 (1), R21DK113487 (1)