Leber hereditary optic neuropathy
Leber hereditary optic neuropathy
Explore linked clinical trials. Standard reference: MONDO:001078815 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| New Non-invasive Modalities for Assessing Retinal Structure and Function | Arterial Occlusive Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Hemianopsia, Leber hereditary optic neuropathy, Neurologic Manifestations, Nutritional and Metabolic Diseases, Optic Neuropathy, Ischemic, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, acute zonal occult outer retinopathy, blindness (disorder), branch retinal artery occlusion, cardiovascular 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 artery occlusion, retinal disorder, vascular disorder, vision disorder | Recruiting | Not applicable | 2026-09-08 | NCT03475173 | |
| Global Registry and Natural History Study for Mitochondrial Disorders | Abnormalities, Multiple, Barth syndrome, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cardiovascular Abnormalities, Cerebral Small Vessel Diseases, Chronic Disease, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Kearns-Sayre syndrome, Leber hereditary optic neuropathy, Leigh syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, MELAS syndrome, MERRF syndrome, NARP syndrome, Neurologic Manifestations, Nutritional and Metabolic Diseases, Ocular Motility Disorders, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Pearson syndrome, Pyruvate Metabolism, Inborn Errors, Signs and Symptoms, X-linked disease, ataxia neuropathy spectrum, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, coenzyme Q10 deficiency, congenital heart disease, cranial nerve neuropathy, epilepsy, eye disorder, heart disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, inherited retinal dystrophy, maternally-inherited diabetes and deafness, metabolic disease, mitochondrial encephalomyopathy, mitochondrial neurogastrointestinal encephalomyopathy, muscle tissue disorder, musculoskeletal system disorder, myelodysplastic syndrome, nervous system disorder, neuromuscular disease, ophthalmoplegia, palsy, progressive external ophthalmoplegia, progressive myoclonus epilepsy, retinal degeneration, retinal disorder, retinitis pigmentosa, spinocerebellar ataxia with epilepsy, vascular disorder, very long chain acyl-CoA dehydrogenase deficiency | Recruiting | Not stated | 2026-08-07 | NCT05554835 | |
| Stem Cell Ophthalmology Treatment Study II | Abnormalities, Multiple, CNS demyelinating autoimmune disease, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cranial Nerve Injuries, Craniocerebral Trauma, Deaf-Blind Disorders, Deafness, Doyne honeycomb retinal dystrophy, Ear Diseases, Eye Diseases, Hereditary, Geographic Atrophy, Hearing Loss, Sensorineural, Leber hereditary optic neuropathy, Neurologic Manifestations, Nutritional and Metabolic Diseases, Optic Atrophy, Autosomal Dominant, Optic Neuropathy, Ischemic, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, Stargardt disease, Usher syndrome, Vision, Low, acute transverse myelitis, age-related macular degeneration, autoimmune disease, autoimmune disorder of the nervous system, blindness (disorder), cardiovascular disorder, cone dystrophy, cone-rod dystrophy, cranial nerve neuropathy, demyelinating disease, eye disorder, glaucoma, hearing disorder, hearing loss disorder, hereditary disease, hereditary optic atrophy, immune system disorder, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, inherited retinal dystrophy, injury, macular degeneration, metabolic disease, nervous system disorder, nervous system injury, neurodegenerative disease, neuromyelitis optica, nutritional deficiency disease, nutritional disorder, ocular hypertension, optic atrophy, optic nerve disorder, optic neuritis, otorhinolaryngologic disease, retinal degeneration, retinal disorder, retinitis pigmentosa, traumatic optic neuropathy, vascular disorder, vision disorder, vitamin A deficiency, vitamin deficiency disorder, wet macular degeneration | Recruiting | Not applicable | 2026-06-29 | NCT03011541 | |
| 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 | |
| Genetic Diagnosis in Inborn Errors of Metabolism | Leber hereditary optic neuropathy, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, epilepsy, inborn mitochondrial metabolism disorder, metabolic disease, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease | Enrolling by invitation | Not stated | 2026-05-28 | NCT06376279 | |
| Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Leber hereditary optic neuropathy, Nutritional and Metabolic Diseases, 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 | Recruiting | Phase 2 | 2026-03-02 | NCT07303296 | |
| A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Leber hereditary optic neuropathy, Nutritional and Metabolic Diseases, 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 | Active, not recruiting | Phase 3 | 2026-02-12 | NCT07406854 | |
| North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC) | Abnormalities, Multiple, Acid-Base Imbalance, Barth syndrome, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cardiovascular Abnormalities, Cerebral Small Vessel Diseases, Chronic Disease, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Deafness, Disease Attributes, Ear Diseases, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Eye Diseases, Hereditary, Kearns-Sayre syndrome, Leber hereditary optic neuropathy, Leigh syndrome, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, MELAS syndrome, MERRF syndrome, NARP syndrome, Neurologic Manifestations, Nutritional and Metabolic Diseases, Ocular Motility Disorders, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Pyruvate Metabolism, Inborn Errors, Sensation Disorders, Signs and Symptoms, Visceral myopathy familial external ophthalmoplegia, X-linked disease, acidosis disorder, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, congenital heart disease, cranial nerve neuropathy, diabetes mellitus, disease, endocrine system disorder, epilepsy, eye disorder, glucose metabolism disease, hearing disorder, hearing loss disorder, heart disorder, hereditary disease, hereditary optic atrophy, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, inherited neurodegenerative disorder, inherited retinal dystrophy, lactic acidosis, maternally-inherited Leigh syndrome, metabolic disease, mitochondrial complex I deficiency, mitochondrial complex IV deficiency, nuclear-type, mitochondrial encephalomyopathy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, ophthalmoplegia, optic atrophy, optic nerve disorder, otorhinolaryngologic disease, palsy, progressive external ophthalmoplegia, progressive myoclonus epilepsy, retinal degeneration, retinal disorder, retinitis pigmentosa, stroke disorder, syndromic disease, vascular disorder | Recruiting | Not stated | 2026-02-04 | NCT01694940 | |
| Tissue Sample Study for Mitochondrial Disorders | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Leber hereditary optic neuropathy, Leigh syndrome, MELAS syndrome, Nutritional and Metabolic Diseases, Pyruvate Metabolism, Inborn Errors, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, metabolic disease, mitochondrial encephalomyopathy, mitochondrial neurogastrointestinal encephalomyopathy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular disease, vascular disorder | Enrolling by invitation | Not stated | 2026-01-07 | NCT01803906 | |
| Pilot Study of the Efficacy of Nicotinamide (Vitamin B3) in Leber's Hereditary Optic Neuropathy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Leber hereditary optic neuropathy, Nutritional and Metabolic Diseases, 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 | Not yet recruiting | Phase 1 | 2025-12-18 | NCT07258667 | |
| Metabolomics Analysis According to the Retinal Nerve Fiber Layer in Patients With NOHL Mutations (MétabOCT) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Leber hereditary optic neuropathy, Nutritional and Metabolic Diseases, 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 | Recruiting | Not applicable | 2025-11-18 | NCT06682819 | |
| Optic Nerve Head Evaluation Through Multimodal Blood Flow Analysis: a Prospective Observational, Multi-center Study | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Leber hereditary optic neuropathy, Nutritional and Metabolic Diseases, Optic Neuropathy, Ischemic, anterior ischemic optic neuropathy, cardiovascular disorder, cranial nerve neuropathy, eye disorder, glaucoma, hereditary disease, hereditary optic atrophy, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, low tension glaucoma, metabolic disease, nervous system disorder, neurodegenerative disease, ocular hypertension, optic atrophy, optic disk drusen, optic nerve disorder, vascular disorder | Not yet recruiting | Not stated | 2025-07-20 | NCT07075887 | |
| Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Leber hereditary optic neuropathy, Nutritional and Metabolic Diseases, 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 | Active, not recruiting | Phase 1 / Phase 2 | 2024-09-04 | NCT05293626 | |
| Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Leber hereditary optic neuropathy, Nutritional and Metabolic Diseases, 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 | Recruiting | Phase 2 / Phase 3 | 2022-11-14 | NCT04912843 | |
| 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 |