15 completed, 8 active not recruiting, 4 recruiting, 4 unknown. 16 Phase 1, 13 Phase 2, 4 Phase 3, 2 Early Phase 1, 1 Not applicable.
Follow Leber Congenital Amaurosis: new trials by email Literature Review of Leber Congenital Amaurosis trials Research Report on Leber Congenital Amaurosis
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 |
|---|---|---|---|---|---|---|
| Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD) OPGx-RDH12 - Leber Congenital Amaurosis, Leber Congenital Amaurosis (LCA) | Phase 1 / Phase 2 | Recruiting | Opus Genetics, Inc | 10 | 2026-10-01 | 2026-10-30 |
| Psychotherapy Group for Parents of Children With LCA Psychotherapy group for parents, Control Group - Leber Congenital Amaurosis (LCA) | Not applicable | Active not recruiting | Instituto de Genética Ocular | 40 | 2025-08-01 | 2026-06-09 |
| 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 |
| Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR) HG004 - Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations | Phase 1 / Phase 2 | Unknown | HuidaGene Therapeutics Co., Ltd. | 20 | 2023-10-31 | 2024-09-19 |
| Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT) HG004 - Leber Congenital Amaurosis | Early Phase 1 | Active not recruiting | Xinhua Hospital, Shanghai Jiao Tong University School of Medicine | 9 | 2023-01-10 | 2024-09-19 |
| Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis OCU400 Low Dose, OCU400 Med Dose, OCU400 High Dose - Retinitis Pigmentosa, Leber Congenital Amaurosis | Phase 1 / Phase 2 | Active not recruiting | Ocugen | 22 | 2022-01-24 | 2026-07-31 |
| 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 |
| Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D ATSN-101, ATSN-101 Diluent Solution, Prednisone - Leber Congenital Amaurosis, LCA | Phase 1 / Phase 2 | Active not recruiting | Atsena Therapeutics Inc. | 15 | 2019-10-22 | 2026-08-03 |
| 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 |
| A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US AAV2-hRPE65v2,voretigene neparvovec-rzyl - Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy | Completed | Spark Therapeutics, Inc. | 87 | 2019-01-10 | 2025-09-22 | |
| Treatment of RP and LCA by Primary RPE Transplantation Human primary Retinal Pigment Epithelial (HuRPE) cells - Leber Congenital Amaurosis, Retinitis Pigmentosa | Early Phase 1 | Unknown | Eyecure Therapeutics Inc. | 30 | 2018-08-01 | 2018-08-09 |
| Natural History Study of CEP290-Related Retinal Degeneration Blindness, Leber Congenital Amaurosis 10 | Completed | Editas Medicine, Inc. | 26 | 2017-12-17 | 2022-05-19 | |
| Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65) AAV OPTIRPE65 - Leber Congenital Amaurosis (LCA), Eye Diseases | Completed | MeiraGTx UK II Ltd | 15 | 2016-11-01 | 2024-04-11 | |
| Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65 Leber Congenital Amaurosis | Completed | MeiraGTx UK II Ltd | 37 | 2016-04-01 | 2026-09-21 | |
| Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) AAV RPE65 - Leber Congenital Amaurosis | Phase 1 / Phase 2 | Completed | MeiraGTx UK II Ltd | 15 | 2016-04-01 | 2021-07-12 |
| Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT No treatment: retrospective chart review - Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP) | Completed | QLT Inc. | 59 | 2015-12-01 | 2016-04-29 | |
| Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2) AAV2-hRPE65v2 - Inherited Retinal Dystrophy Due to RPE65 Mutations | Active not recruiting | Genentech, Inc. | 41 | 2015-06-01 | 2026-08-19 | |
| Inherited Retinal Degenerative Disease Registry Eye Diseases Hereditary, Retinal Disease | Recruiting | Foundation Fighting Blindness | 20000 | 2014-06-01 | 2026-05-19 |
Showing 20 of 32. Sign in to see all of them.
Retinitis Pigmentosa (10) · Retinal Diseases (9) · Eye Diseases, Hereditary (7) · Meckel Syndrome, Type 4 (7) · Eye Abnormalities (6) · Eye Diseases (6) · Leber Congenital Amaurosis 10 (6) · Vision Disorders (6) · Blindness (5) · Neurologic Manifestations (4) · Retinal Degeneration (4) · Sensation Disorders (4) · Optic Atrophy, Hereditary, Leber (3) · Congenital Abnormalities (2)
Eye Diseases (26) · Eye Diseases, Hereditary (25) · Retinal Diseases (23) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (17) · Genetic Diseases, Inborn (14) · Retinal Degeneration (10) · Retinal Dystrophies (10) · Nervous System Diseases (8) · Pathological Conditions, Signs and Symptoms (8) · Signs and Symptoms (8) · Congenital Abnormalities (6) · Cranial Nerve Diseases (5) · Heredodegenerative Disorders, Nervous System (5) · Metabolic Diseases (5) · Neurodegenerative Diseases (5)
retinol acetate (2) · Cell Count (1) · Control Groups (1) · Polymyxin B (1) · Prednisolone (1) · Prednisone (1) · Psychotherapy, Group (1) · Trimethoprim (1) · salicylhydroxamic acid (1)
Spark Therapeutics, Inc. (4) · MeiraGTx UK II Ltd (3) · QLT Inc. (3) · Editas Medicine, Inc. (2) · Laboratoires Thea (2) · ProQR Therapeutics (2) · Assistance Publique - Hôpitaux de Paris (1) · Atsena Therapeutics Inc. (1) · Beacon Therapeutics (1) · Eyecure Therapeutics Inc. (1) · Foundation Fighting Blindness (1) · Genentech, Inc. (1) · Hadassah Medical Organization (1) · HuidaGene Therapeutics Co., Ltd. (1) · Instituto de Genética Ocular (1)
United States (23) · United Kingdom (10) · Germany (7) · Netherlands (7) · Canada (6) · France (5) · Belgium (4) · Brazil (4) · China (3) · Italy (2) · Australia (1) · Denmark (1) · Israel (1) · Spain (1) · Switzerland (1)
Sushanta Mallick (2) · Albert M Maguire, MD (1) · Caio Marques, PhD studant (1) · David Saperstein, MD (1) · Eyal Banin, MD, PhD (1) · J Timothy Stout, MD, PhD, MBA (1) · Jennifer Henrick, RN, MS (1) · Michel Michealides, Prof (1)
PMID 38254722, PMID 19953081, PMID 21606598, PMID 22323828, PMID 18441370, PMID 18441371, PMID 18774912, PMID 18809924, PMID 19583479, PMID 19675341, PMID 19854499, PMID 21911650, PMID 22812667, PMID 25030840, PMID 25537204, PMID 25936984, PMID 26448901, PMID 26605849, PMID 26656277, PMID 27375040, PMID 28712537, PMID 29033008, PMID 29869534, PMID 30025081, PMID 32347917, PMID 33497524, PMID 34629400, PMID 35765055, PMID 38709228, PMID 38888288
2018 (2), 2016 (1)