2 unknown, 1 active not recruiting, 1 completed, 1 recruiting. 4 Phase 2, 3 Phase 3, 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Unknown 2, Active not recruiting 1, Terminated 1 |
| Phase 3 | 3 | Active not recruiting 1, Recruiting 1, Unknown 1 |
| Phase 1 | 2 | Terminated 1, Unknown 1 |
Follow Leber Congenital Amaurosis 10: new trials by email Literature Review of Leber Congenital Amaurosis 10 trials Research Report on Leber Congenital Amaurosis 10
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 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 |
| 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 |
| 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 (6) · Leber Congenital Amaurosis (6) · Meckel Syndrome, Type 4 (6) · Retinal Diseases (6) · Vision Disorders (6) · Blindness (5) · Eye Diseases (5) · Eye Diseases, Hereditary (5) · Neurologic Manifestations (4) · Sensation Disorders (4) · Optic Atrophy, Hereditary, Leber (3) · Retinal Degeneration (3) · Congenital Abnormalities (2) · Genetic Diseases, Inborn (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Nervous System Diseases (6) · Pathological Conditions, Signs and Symptoms (6) · Signs and Symptoms (6) · Congenital Abnormalities (4) · Cranial Nerve Diseases (3) · Genetic Diseases, Inborn (3) · Heredodegenerative Disorders, Nervous System (3) · Metabolic Diseases (3) · Mitochondrial Diseases (3) · Neurodegenerative Diseases (3) · Nutritional and Metabolic Diseases (3) · Optic Atrophies, Hereditary (3) · Optic Atrophy (3) · Optic Nerve Diseases (3)
Editas Medicine, Inc. (2) · Laboratoires Thea (2) · ProQR Therapeutics (2)
United States (5) · Belgium (4) · Germany (4) · Netherlands (4) · Brazil (3) · Canada (3) · France (3) · United Kingdom (3) · Italy (2) · Australia (1) · Spain (1)
PMID 33497524, PMID 34629400, PMID 38709228
2018 (2)