7 completed, 4 recruiting, 2 unknown, 1 active not recruiting. 15 Phase 1, 11 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 15 | Completed 7, Recruiting 4, Unknown 2, Active not recruiting 1, Terminated 1 |
| Phase 2 | 11 | Completed 5, Recruiting 3, Active not recruiting 1, Terminated 1, Unknown 1 |
Follow Deafness: new trials by email Literature Review of Deafness trials Research Report on Deafness
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 |
|---|---|---|---|---|---|---|
| Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss SKY-GJB2 - GJB2-mediated Hearing Loss, GJB2 Gene Mutation | Phase 1 / Phase 2 | Recruiting | Skylark Bio Inc. | 10 | 2026-05-28 | 2026-08-27 |
| 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 |
| A Phase I/II Clinical Trial With SENS-501 in Children Suffering From Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations SENS-501 administration - OTOF Gene Mutation, DFNB9 | Phase 1 / Phase 2 | Active not recruiting | Sensorion | 12 | 2024-06-21 | 2026-07-08 |
| Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels N-Acetylcysteine - Mitochondrial Disease | Phase 1 | Recruiting | Michio Hirano, MD | 18 | 2023-07-06 | 2026-01-23 |
| Phase I/IIa Study Evaluating Safety and Efficacy of an Intratympanic Dose of PIPE-505 in Subjects With Hearing Loss PIPE-505, Placebo - Sensorineural Hearing Loss | Phase 1 / Phase 2 | Completed | Contineum Therapeutics | 28 | 2020-10-15 | 2025-04-01 |
| 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 |
| Intratympanic N-Acetylcysteine for Prevention of Cisplatin-induced Ototoxicity. N-Acetyl Cysteine - Ototoxic Hearing Loss | Phase 1 / Phase 2 | Unknown | Sunnybrook Health Sciences Centre | 80 | 2020-02-26 | 2023-09-06 |
| 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 |
| A Safety Study of the Auditory Brainstem Implant for Pediatric Profoundly Deaf Patients Auditory Brainstem Implant - Profound Bilateral Deafness Due to, Bilateral Cochlear Aplasia | Phase 1 | Completed | Laurie Eisenberg | 10 | 2014-03-01 | 2025-09-15 |
| Efficacy, Safety, and Tolerability of Ancrod in Patients With Sudden Hearing Loss Ancrod, Saline solution - Hearing Loss, Deafness | Phase 1 / Phase 2 | Completed | Nordmark Arzneimittel GmbH & Co. KG | 31 | 2013-05-01 | 2018-12-21 |
| 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 |
| Outcomes In Children With Developmental Delay And Deafness cochlear implantation, hearing aids - Deafness, Hearing Loss | Phase 1 | Completed | Stanford University | 303 | 2009-09-01 | 2016-11-15 |
Hearing Loss (6) · Usher Syndromes (6) · Hearing Loss, Sensorineural (5) · Retinitis Pigmentosa (4) · Ear Diseases (3) · Diabetes Mellitus (2) · Hearing Disorders (2) · Ataxia (1) · Cone-Rod Dystrophies (1) · Deafness, Autosomal Recessive 1A (1) · Deafness, Autosomal Recessive 9 (1) · Eye Abnormalities (1) · Eye Diseases (1) · Eye Diseases, Hereditary (1)
Nervous System Diseases (15) · Neurologic Manifestations (15) · Pathological Conditions, Signs and Symptoms (15) · Sensation Disorders (15) · Signs and Symptoms (15) · Otorhinolaryngologic Diseases (14) · Hearing Disorders (13) · Ear Diseases (12) · Hearing Loss (9) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Genetic Diseases, Inborn (8) · Abnormalities, Multiple (7) · Blindness (7) · Congenital Abnormalities (7) · Deaf-Blind Disorders (7)
Acetylcysteine (2) · Ancrod (1) · Auditory Brain Stem Implants (1) · Cochlear Implantation (1) · Dantrolene (1) · Hearing Aids (1) · Intravitreal Injections (1) · Saline Solution (1)
AAVantgarde Bio Srl (1) · BlueRock Therapeutics (1) · Contineum Therapeutics (1) · EyeXCel Pty. Ltd. (1) · Laurie Eisenberg (1) · Michio Hirano, MD (1) · Nacuity Pharmaceuticals, Inc. (1) · Nordmark Arzneimittel GmbH & Co. KG (1) · ProQR Therapeutics (1) · Sanofi (1) · Sensorion (1) · Skylark Bio Inc. (1) · Stanford University (1) · Sunnybrook Health Sciences Centre (1) · Washington University School of Medicine (1)
United States (9) · Australia (3) · France (3) · Canada (2) · Czechia (1) · Germany (1) · Italy (1) · United Kingdom (1)
Benedict Tan, MBBS, FRACP (1) · Eric Wilkinson, MD (1) · Fumihiko Urano, MD (1) · John Oghalai (1) · Jose-Alain Sahel, MD, PhD (1) · Laurie S. Eisenberg, PhD (1) · Lee Anderson, MD (1) · Martin Canis, MD PhD (1) · Michio Hirano, MD (1) · Richard Weleber, MD (1) · Stephen Huhn, MD (1) · Trung N Le (1)
PMID 24705452, PMID 26366332, PMID 27898605, PMID 29714549, PMID 29718280, PMID 31215818, PMID 34185708, PMID 35607935, PMID 37418643
2018 (1), 2023 (1), R01DC010075 (1), R21DK113487 (1)