Meckel syndrome, type 4
Meckel syndrome, type 4
Explore linked clinical trials. Standard reference: MONDO:00126263 results ยท Page 1 of 1
Clinical trials
Swipe the table sideways for more columns.
| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Leber congenital amaurosis, Meckel syndrome, type 4, congenital stationary night blindness autosomal dominant 1, enhanced S-cone syndrome, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosa | Active, not recruiting | Phase 1 / Phase 2 | 2026-07-31 | NCT05203939 | |
| Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Abnormalities, Eye Diseases, Hereditary, Leber congenital amaurosis, Leber congenital amaurosis 10, Leber hereditary optic neuropathy, Meckel syndrome, type 4, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, blindness (disorder), cranial nerve neuropathy, eye disorder, hereditary disease, hereditary optic atrophy, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, optic atrophy, optic nerve disorder, retinal disorder, vision disorder | Recruiting | Phase 3 | 2026-06-25 | NCT06891443 | |
| A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Abnormalities, Eye Diseases, Hereditary, Leber congenital amaurosis, Leber congenital amaurosis 10, Leber hereditary optic neuropathy, Meckel syndrome, type 4, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, blindness (disorder), cranial nerve neuropathy, eye disorder, hereditary disease, hereditary optic atrophy, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, optic atrophy, optic nerve disorder, retinal disorder, vision disorder | Active, not recruiting | Phase 2 / Phase 3 | 2022-03-17 | NCT03913143 |