3 completed, 2 active not recruiting, 2 unknown, 1 recruiting. 6 Phase 2, 4 Phase 1, 3 Phase 3.
Follow Meckel Syndrome, Type 4: new trials by email Literature Review of Meckel Syndrome, Type 4 trials Research Report on Meckel Syndrome, Type 4
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 |
| 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 |
| 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 | |
| Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene QR-110 - Leber's Congenital Amaurosis | Phase 1 / Phase 2 | Completed | Laboratoires Thea | 11 | 2017-10-16 | 2024-10-15 |
| Assessment of the Prevalence of Genes AHI1, NPHP1 and CEP290 in Joubert Syndrome Whole blood sample - Joubert Syndrome, Cerebello-oculo-renal Syndromes | Completed | Assistance Publique - Hôpitaux de Paris | 80 | 2007-03-01 | 2010-06-03 |
Leber Congenital Amaurosis (7) · Eye Abnormalities (6) · Leber Congenital Amaurosis 10 (6) · Retinal Diseases (6) · Vision Disorders (6) · Blindness (5) · Eye Diseases (5) · Eye Diseases, Hereditary (5) · Neurologic Manifestations (4) · Optic Atrophy, Hereditary, Leber (4) · Sensation Disorders (4) · Retinal Degeneration (3) · Congenital Abnormalities (2) · Genetic Diseases, Inborn (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Nervous System Diseases (7) · Genetic Diseases, Inborn (6) · Pathological Conditions, Signs and Symptoms (6) · Signs and Symptoms (6) · Congenital Abnormalities (5) · Cranial Nerve Diseases (4) · Heredodegenerative Disorders, Nervous System (4) · Metabolic Diseases (4) · Mitochondrial Diseases (4) · Neurodegenerative Diseases (4) · Nutritional and Metabolic Diseases (4) · Optic Atrophies, Hereditary (4) · Optic Atrophy (4) · Optic Nerve Diseases (4)
Laboratoires Thea (3) · Editas Medicine, Inc. (2) · ProQR Therapeutics (2) · Assistance Publique - Hôpitaux de Paris (1) · Ocugen (1)
United States (7) · Belgium (5) · France (4) · Germany (4) · Netherlands (4) · Brazil (3) · Canada (3) · United Kingdom (3) · Italy (2) · Australia (1) · Spain (1)
Jennifer Henrick, RN, MS (1) · Mohamed Genead, MD (1)
PMID 30559420, PMID 31215818, PMID 33497524, PMID 33795869, PMID 34629400, PMID 35379979, PMID 38709228
2018 (2), 2017 (1)