18 completed, 6 terminated, 5 active not recruiting, 4 recruiting. 38 Phase 2, 10 Phase 1, 6 Phase 3.
Follow Blindness: new trials by email Literature Review of Blindness trials Research Report on Blindness
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 |
| Clinical Investigation of Safety and Effectiveness for the enVVe® System envve system - Chronic Venous Insufficiency, CVI | Phase 2 / Phase 3 | Not yet recruiting | Hancock Jaffe Laboratiores, Inc | 220 | 2026-07-01 | 2026-05-27 |
| Ixoberogene Soroparvovec (Ixo-vec) Contralateral Dosing Study in Participants With Neovascular Age-related Macular Degeneration Ixo-vec - Neovascular Age-Related Macular Degeneration (nAMD) Wet AMD | Phase 2 | Recruiting | Adverum Biotechnologies, Inc. | 15 | 2026-06-02 | 2026-08-05 |
| 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 |
| Safety and Efficacy of ADVM-022 in Treatment-Experienced Patients With Neovascular Age-related Macular Degeneration [LUNA] ADVM-022 - Neovascular Age-related Macular Degeneration | Phase 2 | Active not recruiting | Adverum Biotechnologies, Inc. | 69 | 2022-08-23 | 2026-10-27 |
| 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 |
| An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. sepofarsen - Leber Congenital Amaurosis 10, Blindness | Phase 2 / Phase 3 | Unknown | ProQR Therapeutics | 15 | 2021-03-23 | 2022-03-25 |
| 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 |
| ADVM-022 Intravitreal Gene Therapy for DME 6E11 vg/eye of ADVM-022, 2E11 vg/eye of ADVM-022, Aflibercept - Diabetic Macular Edema, Diabetic Retinopathy | Phase 2 | Completed | Adverum Biotechnologies, Inc. | 36 | 2020-05-28 | 2025-07-10 |
| Extension Study to Study PQ-110-001 (NCT03140969) QR-110 - Leber Congenital Amaurosis 10, Blindness | Phase 1 / Phase 2 | Terminated | Laboratoires Thea | 9 | 2019-05-13 | 2024-10-15 |
| A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) sepofarsen, Sham - Leber Congenital Amaurosis 10, Blindness | Phase 2 / Phase 3 | Active not recruiting | ProQR Therapeutics | 36 | 2019-04-04 | 2022-03-17 |
| 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 |
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Usher Syndromes (12) · Eye Diseases (10) · Retinal Diseases (10) · Retinitis Pigmentosa (10) · Vision Disorders (9) · Eye Abnormalities (8) · Eye Diseases, Hereditary (7) · Hemianopsia (5) · Stroke (5) · Wolfram Syndrome (5) · Leber Congenital Amaurosis (3) · Leber Congenital Amaurosis 10 (3) · Macular Degeneration (3) · Meckel Syndrome, Type 4 (3)
Nervous System Diseases (38) · Neurologic Manifestations (35) · Sensation Disorders (35) · Pathological Conditions, Signs and Symptoms (34) · Signs and Symptoms (34) · Vision Disorders (29) · Eye Diseases (28) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (21) · Congenital Abnormalities (20) · Ear Diseases (18) · Genetic Diseases, Inborn (18) · Hearing Disorders (18) · Otorhinolaryngologic Diseases (18) · Abnormalities, Multiple (17)
Intravitreal Injections (4) · salicylhydroxamic acid (3) · Valproic Acid (2) · 2-(1-(5-(cyclohexylcarbamoyl)-6-propylsulfanylpyridin-2-yl)-3-piperidyl)acetic acid (1) · 4-phenylbutyric acid (1) · Blood Specimen Collection (1) · Ciliary Neurotrophic Factor (1) · Control Groups (1) · Dantrolene (1) · Electroconvulsive Therapy (1) · Fluoxetine (1) · Melatonin (1) · Revacept (1) · Rituximab (1) · Sugars (1)
Laboratoires Thea (6) · University of Magdeburg (4) · Adverum Biotechnologies, Inc. (3) · ProQR Therapeutics (3) · Sanofi (2) · US Department of Veterans Affairs (2) · University of Birmingham (2) · AAVantgarde Bio Srl (1) · AdvanceCor GmbH (1) · Amylyx Pharmaceuticals Inc. (1) · BlueRock Therapeutics (1) · Bogachan Sahin (1) · Carmen Clapp (1) · Centre d'Etude des Cellules Souches (1) · Hancock Jaffe Laboratiores, Inc (1)
United States (23) · United Kingdom (10) · France (9) · Germany (9) · Canada (5) · Italy (5) · Belgium (4) · Netherlands (4) · Brazil (3) · Austria (2) · Spain (2) · Australia (1) · Denmark (1) · Mexico (1) · Poland (1)
Adam Turpcu, PhD (2) · John Fryer, Ph.D., Asst. Director (2) · Nancy Rocheleau, Program Analyst (2) · Alan Lankford, PhD (1) · Alan R. Spitzer, MD (1) · Alexandra Sinclair, MbChb PhD (1) · Barbara J. Stoll, MD (1) · Bernhard A Sabel, Ph.D. (1) · Bernhard A Sabel, Prof. Dr. (1) · Beverly S. Brozanski, MD (1) · Camille Bedrosian, MD (1)
PMID 10654946, PMID 24504128, PMID 24705452, PMID 25678831, PMID 28082176, PMID 28923789, PMID 31215818, PMID 33098644, PMID 33230035, PMID 34185708, PMID 34219679, PMID 35695006, PMID 36166771, PMID 36240084, PMID 37314061, PMID 37418643, PMID 37740611, PMID 40010822, PMID 40339601
2018 (3), M01RR000054 (1), M01RR000070 (1), R21DK113487 (1), U10HD021385 (1), U10HD021415 (1), U10HD027851 (1), U10HD027853 (1), U10HD027856 (1), U10HD027871 (1), U10HD027880 (1), U10HD027904 (1), U10HD040689 (1)