12 completed, 4 active not recruiting, 4 recruiting, 4 terminated. 29 Phase 2, 11 Phase 1, 4 Phase 3.
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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 |
| SPI-1005 for Prevention and Treatment of Chemotherapy Induced Hearing Loss SPI-1005 Low Dose, SPI-1005 Middle Dose, SPI-1005 High Dose - Lung Cancer, Head and Neck Cancer | Phase 2 | Withdrawn | Sound Pharmaceuticals, Incorporated | 2026-06-01 | 2026-06-03 | |
| 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 |
| 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 |
| 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 |
| 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 |
| OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION) OMT-28 - Primary Mitochondrial Disease | Phase 2 | Completed | Omeicos Therapeutics GmbH | 28 | 2023-05-22 | 2026-01-22 |
| 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 |
| Efficacy of Sodium Thiosulfate and Mannitol in Reducing Ototoxicity in Adult Patients Receiving Cisplatin Chemotherapy Sodium Thiosulfate + Mannitol - Ototoxic Hearing Loss | Phase 2 | Recruiting | Sunnybrook Health Sciences Centre | 92 | 2023-02-15 | 2023-04-14 |
| Efficacy and Safety of Liuwei Dihuang Pill Versus Placebo in Presbycusis With Shen (Kidney)-Yin Deficiency Liuwei Dihuang Pill (marketed product in China), Liuwei Dihuang Pill（placebo ） - Presbycusis, Age-related Hearing Loss | Phase 2 | Completed | Shanghai Jiao Tong University School of Medicine | 120 | 2022-01-25 | 2026-01-07 |
| 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 |
| 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 |
Showing 20 of 29. Sign in to see all of them.
Usher Syndromes (12) · Retinitis Pigmentosa (10) · Hearing Loss (8) · Hearing Loss, Sensorineural (6) · Retinal Diseases (6) · Vision Disorders (6) · Eye Abnormalities (5) · Eye Diseases, Hereditary (5) · Wolfram Syndrome (5) · Ear Diseases (4) · Eye Diseases (4) · Hearing Disorders (3) · Diabetes Mellitus (2) · Ototoxicity (2)
Nervous System Diseases (29) · Neurologic Manifestations (29) · Sensation Disorders (29) · Otorhinolaryngologic Diseases (28) · Hearing Disorders (26) · Ear Diseases (25) · Pathological Conditions, Signs and Symptoms (25) · Signs and Symptoms (25) · Hearing Loss (21) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (18) · Abnormalities, Multiple (17) · Blindness (17) · Congenital Abnormalities (17) · Deaf-Blind Disorders (17)
Intravitreal Injections (4) · Acetylcysteine (2) · Valproic Acid (2) · ebselen (2) · 4-phenylbutyric acid (1) · Ancrod (1) · Blood Specimen Collection (1) · Ciliary Neurotrophic Factor (1) · Dantrolene (1) · Electroconvulsive Therapy (1) · Liuwei Dihuang Decoction (1) · Mannitol (1) · Saline Solution (1) · Tirzepatide (1) · salicylhydroxamic acid (1)
Laboratoires Thea (5) · Sanofi (2) · Sound Pharmaceuticals, Incorporated (2) · Sunnybrook Health Sciences Centre (2) · AAVantgarde Bio Srl (1) · Amylyx Pharmaceuticals Inc. (1) · BlueRock Therapeutics (1) · Centre d'Etude des Cellules Souches (1) · Contineum Therapeutics (1) · Nacuity Pharmaceuticals, Inc. (1) · Neurotech Pharmaceuticals (1) · Nordmark Arzneimittel GmbH & Co. KG (1) · Omeicos Therapeutics GmbH (1) · Ospedale San Raffaele (1) · ProQR Therapeutics (1)
United States (15) · France (9) · Canada (5) · United Kingdom (5) · Germany (4) · Italy (4) · Netherlands (3) · Australia (2) · Spain (2) · Belgium (1) · Brazil (1) · China (1) · Czechia (1) · Denmark (1) · Poland (1)
Jonathan Kil, MD (2) · Benoit Mongourdin, PH (1) · Camille Bedrosian, MD (1) · Colleen Le Prell, PhD (1) · Fumihiko Urano, MD (1) · Hongsheng Tan, Doctor (1) · Ismail Kocyigit, MD (1) · Jacque Duncan, MD (1) · Jianning Zhang, Doctor (1) · Jianrong Shi, Doctor (1) · Jose-Alain Sahel, MD, PhD (1) · Lee Anderson, MD (1)
PMID 24705452, PMID 24975234, PMID 25073872, PMID 28716314, PMID 31215818, PMID 34185708, PMID 37418643, PMID 40010822, PMID 40339601
2018 (1), 2023 (1), R21DK113487 (1)