3 terminated, 2 active not recruiting, 2 completed, 2 unknown. 9 Phase 2, 5 Phase 3, 3 Phase 1.
Follow Eye Disorders Congenital: new trials by email Literature Review of Eye Disorders Congenital trials Research Report on Eye Disorders Congenital
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 |
| Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) sepofarsen, Placebo IVT - Leber Congenital Amaurosis 10, Blindness | Phase 3 | Recruiting | Laboratoires Thea | 32 | 2025-06-04 | 2026-10-21 |
| 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 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 |
| 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 |
| Single Ascending Dose Study in Participants With LCA10 EDIT-101 - Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies | Phase 1 / Phase 2 | Unknown | Editas Medicine, Inc. | 34 | 2019-09-26 | 2022-12-05 |
| 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 |
| Natural History Study of CEP290-Related Retinal Degeneration Blindness, Leber Congenital Amaurosis 10 | Completed | Editas Medicine, Inc. | 26 | 2017-12-17 | 2022-05-19 |
Eye Abnormalities (11) · Retinal Diseases (11) · Vision Disorders (11) · Eye Diseases, Hereditary (10) · Eye Diseases (8) · Leber Congenital Amaurosis (6) · Leber Congenital Amaurosis 10 (6) · Meckel Syndrome, Type 4 (6) · Blindness (5) · Retinitis Pigmentosa (5) · Usher Syndromes (5) · Neurologic Manifestations (4) · Sensation Disorders (4) · Optic Atrophy, Hereditary, Leber (3) · Retinal Degeneration (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Nervous System Diseases (11) · Pathological Conditions, Signs and Symptoms (11) · Signs and Symptoms (11) · Congenital Abnormalities (9) · Genetic Diseases, Inborn (8) · Neurologic Manifestations (7) · Sensation Disorders (7) · Abnormalities, Multiple (5) · Blindness (5) · Deaf-Blind Disorders (5) · Deafness (5) · Ear Diseases (5) · Hearing Disorders (5) · Hearing Loss (5)
Intravitreal Injections (3) · salicylhydroxamic acid (1)
Laboratoires Thea (6) · ProQR Therapeutics (3) · Editas Medicine, Inc. (2)
United States (9) · Germany (6) · Netherlands (6) · United Kingdom (6) · Belgium (5) · Canada (5) · France (5) · Brazil (4) · Italy (3) · Australia (1) · Denmark (1) · Spain (1)
PMID 31215818, PMID 33497524, PMID 34629400, PMID 37418643, PMID 38709228, PMID 40339601
2018 (3)