{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Leber+Hereditary+Optic+Neuropathy", "query": { "condition": "Leber Hereditary Optic Neuropathy" }, "page_size": 10 }, "pagination": { "page": 1, "page_size": 10, "total_count": 27, "total_pages": 3, "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Leber+Hereditary+Optic+Neuropathy&page=2&page_size=10", "previous_page_url": null }, "source": "remote", "last_synced_at": "2026-05-22T08:13:34.450Z", "attribution": "Data derived from public ClinicalTrials.gov records. The official record at https://clinicaltrials.gov/ remains the source of truth for current availability, contacts, and full study details.", "notes": [ "This endpoint exposes only public summary fields — no participant contact emails, phone numbers, or scraped investigator contact details.", "For contact information and full protocol detail, follow each trial's `official_url` to the ClinicalTrials.gov record.", "This API is a navigational aid. It does not provide medical advice, eligibility determinations, or compensation guarantees." ], "trials": [ { "nct_id": "NCT02774005", "title": "Study to Assess the Efficacy and Safety of Raxone in LHON Patients", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE4" ], "conditions": [ "Leber's Hereditary Optic Neuropathy (LHON)" ], "interventions": [ { "name": "Idebenone", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Santhera Pharmaceuticals", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "12 Years", "maximum_age": null, "sex": "ALL", "summary": "12 Years and older" }, "enrollment_count": 199, "start_date": "2016-05", "completion_date": "2021-03-29", "has_results": true, "last_update_posted_date": "2023-04-21", "last_synced_at": "2026-05-22T08:13:34.450Z", "location_count": 9, "location_summary": "Phoenix, Arizona • Palo Alto, California • Stanford, California + 6 more", "locations": [ { "city": "Phoenix", "state": "Arizona" }, { "city": "Palo Alto", "state": "California" }, { "city": "Stanford", "state": "California" }, { "city": "Aurora", "state": "Colorado" }, { "city": "Atlanta", "state": "Georgia" } ], "official_url": "https://clinicaltrials.gov/study/NCT02774005" }, { "nct_id": "NCT02435940", "title": "Inherited Retinal Degenerative Disease Registry", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Eye Diseases Hereditary", "Retinal Disease", "Achromatopsia", "Bardet-Biedl Syndrome", "Bassen-Kornzweig Syndrome", "Batten Disease", "Best Disease", "Choroidal Dystrophy", "Choroideremia", "Cone Dystrophy", "Cone-Rod Dystrophy", "Congenital Stationary Night Blindness", "Enhanced S-Cone Syndrome", "Fundus Albipunctatus", "Goldmann-Favre Syndrome", "Gyrate Atrophy", "Juvenile Macular Degeneration", "Kearns-Sayre Syndrome", "Leber Congenital Amaurosis", "Refsum Syndrome", "Retinitis Pigmentosa", "Retinitis Punctata Albescens", "Retinoschisis", "Rod-Cone Dystrophy", "Rod Dystrophy", "Rod Monochromacy", "Stargardt Disease", "Usher Syndrome" ], "interventions": [], "intervention_types": [], "sponsor": "Foundation Fighting Blindness", "sponsor_class": "OTHER", "healthy_volunteers": true, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 20000, "start_date": "2014-06", "completion_date": "2037-06", "has_results": false, "last_update_posted_date": "2026-05-19", "last_synced_at": "2026-05-22T08:13:34.450Z", "location_count": 1, "location_summary": "Columbia, Maryland", "locations": [ { "city": "Columbia", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT02435940" }, { "nct_id": "NCT02023866", "title": "Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Inherited Mitochondrial Disease, Including Leigh Syndrome" ], "interventions": [ { "name": "Cysteamine Bitartrate", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Amgen", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "6 Years", "maximum_age": "17 Years", "sex": "ALL", "summary": "6 Years to 17 Years" }, "enrollment_count": 36, "start_date": "2014-05", "completion_date": "2016-10", "has_results": true, "last_update_posted_date": "2024-12-27", "last_synced_at": "2026-05-22T08:13:34.450Z", "location_count": 5, "location_summary": "San Diego, California • Stanford, California • Akron, Ohio + 2 more", "locations": [ { "city": "San Diego", "state": "California" }, { "city": "Stanford", "state": "California" }, { "city": "Akron", "state": "Ohio" }, { "city": "Houston", "state": "Texas" }, { "city": "Salt Lake City", "state": "Utah" } ], "official_url": "https://clinicaltrials.gov/study/NCT02023866" }, { "nct_id": "NCT01389817", "title": "Near-infrared Light-emitting Diode (NIR-LED) Therapy for Leber's Hereditary Optic Neuropathy (LHON)", "overall_status": "TERMINATED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Leber's Hereditary Optic Neuropathy (LHON)" ], "interventions": [ { "name": "Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.))", "type": "DEVICE" } ], "intervention_types": [ "DEVICE" ], "sponsor": "Medical College of Wisconsin", "sponsor_class": "OTHER", "healthy_volunteers": true, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 4, "start_date": "2011-07", "completion_date": "2013-05", "has_results": true, "last_update_posted_date": "2014-09-29", "last_synced_at": "2026-05-22T08:13:34.450Z", "location_count": 3, "location_summary": "Milwaukee, Wisconsin", "locations": [ { "city": "Milwaukee", "state": "Wisconsin" }, { "city": "Milwaukee", "state": "Wisconsin" }, { "city": "Milwaukee", "state": "Wisconsin" } ], "official_url": "https://clinicaltrials.gov/study/NCT01389817" }, { "nct_id": "NCT01064505", "title": "Safety Study of a Single IVT Injection of QPI-1007 in Chronic Optic Nerve Atrophy and Recent Onset NAION Patients", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Optic Atrophy", "Non-arteritic Anterior Ischemic Optic Neuropathy" ], "interventions": [ { "name": "QPI-1007 at various doses", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Quark Pharmaceuticals", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "50 Years", "maximum_age": null, "sex": "ALL", "summary": "50 Years and older" }, "enrollment_count": 48, "start_date": "2010-02", "completion_date": "2013-04", "has_results": false, "last_update_posted_date": "2013-05-13", "last_synced_at": "2026-05-22T08:13:34.450Z", "location_count": 22, "location_summary": "Phoenix, Arizona • Beverly Hills, California • Los Angeles, California + 19 more", "locations": [ { "city": "Phoenix", "state": "Arizona" }, { "city": "Beverly Hills", "state": "California" }, { "city": "Los Angeles", "state": "California" }, { "city": "Aurora", "state": "Colorado" }, { "city": "Miami", "state": "Florida" } ], "official_url": "https://clinicaltrials.gov/study/NCT01064505" }, { "nct_id": "NCT03140969", "title": "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", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Leber's Congenital Amaurosis" ], "interventions": [ { "name": "QR-110", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Laboratoires Thea", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "6 Years", "maximum_age": null, "sex": "ALL", "summary": "6 Years and older" }, "enrollment_count": 11, "start_date": "2017-10-16", "completion_date": "2019-10-02", "has_results": true, "last_update_posted_date": "2024-10-15", "last_synced_at": "2026-05-22T08:13:34.450Z", "location_count": 2, "location_summary": "Iowa City, Iowa • Philadelphia, Pennsylvania", "locations": [ { "city": "Iowa City", "state": "Iowa" }, { "city": "Philadelphia", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT03140969" }, { "nct_id": "NCT03655223", "title": "Early Check: Expanded Screening in Newborns", "overall_status": "ENROLLING_BY_INVITATION", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Spinal Muscular Atrophy", "Fragile X Syndrome", "Fragile X - Premutation", "Duchenne Muscular Dystrophy", "Hyperinsulinemic Hypoglycemia, Familial 1", "Diabetes Mellitus", "Adrenoleukodystrophy, Neonatal", "Medium-chain Acyl-CoA Dehydrogenase Deficiency", "Very Long Chain Acyl Coa Dehydrogenase Deficiency", "Beta-ketothiolase Deficiency", "Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency", "Primary Hyperoxaluria Type 1", "Congenital Bile Acid Synthesis Defect Type 2", "Pyridoxine-Dependent Epilepsy", "Hereditary Fructose Intolerance", "Hypophosphatasia", "Hyperargininemia", "Mucopolysaccharidosis Type 6", "Argininosuccinic Aciduria", "Citrullinemia, Type I", "Wilson Disease", "Maple Syrup Urine Disease, Type 1A", "Maple Syrup Urine Disease, Type 1B", "Biotinidase Deficiency", "Neonatal Severe Primary Hyperparathyroidism", "Intrinsic Factor Deficiency", "Usher Syndrome Type 1D/F Digenic (Diagnosis)", "Cystic Fibrosis", "Stickler Syndrome Type 2", "Stickler Syndrome Type 1", "Alport Syndrome, Autosomal Recessive", "Alport Syndrome, X-Linked", "Carbamoyl Phosphate Synthetase I Deficiency Disease", "Carnitine Palmitoyl Transferase 1A Deficiency", "Carnitine Palmitoyltransferase II Deficiency", "Cystinosis", "Chronic Granulomatous Disease", "Cerebrotendinous Xanthomatoses", "Maple Syrup Urine Disease, Type 2", "Severe Combined Immunodeficiency Due to DCLRE1C Deficiency", "Thyroid Dyshormonogenesis 6", "Thyroid Dyshormonogenesis 5", "Supravalvar Aortic Stenosis", "Factor X Deficiency", "Hemophilia A", "Hemophilia B", "Tyrosinemia, Type I", "Fructose 1,6 Bisphosphatase Deficiency", "Glycogen Storage Disease Type I", "G6PD Deficiency", "Glycogen Storage Disease II", "Galactokinase Deficiency", "Mucopolysaccharidosis Type IV A", "Galactosemias", "Guanidinoacetate Methyltransferase Deficiency", "Agat Deficiency", "Glutaryl-CoA Dehydrogenase Deficiency", "Gtp Cyclohydrolase I Deficiency", "Hyperinsulinism-Hyperammonemia Syndrome", "Primary Hyperoxaluria Type 2", "3-Hydroxyacyl-CoA Dehydrogenase Deficiency", "Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency", "Mitochondrial Trifunctional Protein Deficiency", "Sickle Cell Disease", "Beta-Thalassemia", "Holocarboxylase Synthetase Deficiency", "3-Hydroxy-3-Methylglutaric Aciduria", "Primary Hyperoxaluria Type 3", "Hermansky-Pudlak Syndrome 1", "Hermansky-Pudlak Syndrome 4", "Apparent Mineralocorticoid Excess", "HSDB", "CBAS1", "Mucopolysaccharidosis Type 2", "Mucopolysaccharidosis Type 1", "Severe Combined Immunodeficiency, X Linked", "Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency", "Diabetes Mellitus, Permanent Neonatal", "Isovaleric Acidemia", "Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder)", "Jervell and Lange-Nielsen Syndrome 2", "Hyperinsulinemic Hypoglycemia, Familial, 2", "Diabetes Mellitus, Permanent Neonatal, With Neurologic Features", "Jervell and Lange-Nielsen Syndrome 1", "Lysosomal Acid Lipase Deficiency", "CblF", "3-Methylcrotonyl CoA Carboxylase 1 Deficiency", "3-Methylcrotonyl CoA Carboxylase 2 Deficiency", "Waardenburg Syndrome Type 2A", "Methylmalonic Aciduria cblA Type", "Methylmalonic Aciduria cblB Type", "Methylmalonic Aciduria and Homocystinuria Type cblC", "MAHCD", "Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency", "Congenital Disorder of Glycosylation Type 1B", "Mthfr Deficiency", "Methylcobalamin Deficiency Type Cbl G (Disorder)", "Methylcobalamin Deficiency Type cblE", "Usher Syndrome, Type 1B", "N-acetylglutamate Synthase Deficiency", "Ornithine Transcarbamylase Deficiency", "Phenylketonurias", "Waardenburg Syndrome Type 1", "Congenital Hypothyroidism", "Propionic Acidemia", "Usher Syndrome, Type 1F", "Pancreatic Agenesis 1", "Hereditary Hypophosphatemic Rickets", "Glycogen Storage Disease IXB", "Glycogen Storage Disease IXC", "MOWS", "Epilepsy, Early-Onset, Vitamin B6-Dependent", "Pyridoxal Phosphate-Responsive Seizures", "Pituitary Hormone Deficiency, Combined, 1", "Ptsd", "Dihydropteridine Reductase Deficiency", "Severe Combined Immunodeficiency Due to RAG1 Deficiency", "Severe Combined Immunodeficiency Due to RAG2 Deficiency", "Retinoblastoma", "Multiple Endocrine Neoplasia Type 2B", "Pseudohypoaldosteronism, Type I", "Liddle Syndrome", "Biotin-Responsive Basal Ganglia Disease", "SCD", "DIAR1", "GSD1C", "Acrodermatitis Enteropathica", "Thyroid Dyshormonogenesis 1", "Riboflavin Transporter Deficiency", "Waardenburg Syndrome, Type 2E", "SRD", "Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency", "Barth Syndrome", "Adrenocorticotropic Hormone Deficiency", "Transcobalamin II Deficiency", "Thyroid Dyshormonogenesis 3", "Segawa Syndrome, Autosomal Recessive", "Autosomal Recessive Nonsyndromic Hearing Loss", "Thyroid Dyshormonogenesis 2A", "Congenital Isolated Thyroid Stimulating Hormone Deficiency", "Hypothyroidism Due to TSH Receptor Mutations", "Usher Syndrome Type 1C", "Usher Syndrome Type 1G (Diagnosis)", "Von Willebrand Disease, Type 3", "Combined Immunodeficiency Due to ZAP70 Deficiency", "Adenine Phosphoribosyltransferase Deficiency", "Metachromatic Leukodystrophy", "Canavan Disease", "Menkes Disease", "Carbonic Anhydrase VA Deficiency", "Developmental and Epileptic Encephalopathy 2", "17 Alpha-Hydroxylase Deficiency", "Smith-Lemli-Opitz Syndrome", "Krabbe Disease", "Glutathione Synthetase Deficiency", "Mucopolysaccharidosis Type 7", "Rett Syndrome", "Molybdenum Cofactor Deficiency, Type A", "Niemann-Pick Disease, Type C1", "Niemann-Pick Disease Type C2", "Ornithine Aminotransferase Deficiency", "3-Phosphoglycerate Dehydrogenase Deficiency", "Leber Congenital Amaurosis 2", "Dravet Syndrome", "Mucopolysaccharidosis Type 3 A", "Ornithine Translocase Deficiency", "Carnitine-acylcarnitine Translocase Deficiency", "Glucose Transporter Type 1 Deficiency Syndrome", "Creatine Transporter Deficiency", "Niemann-Pick Disease Type A", "Pitt Hopkins Syndrome", "Tuberous Sclerosis 1", "Tuberous Sclerosis 2", "Ataxia With Isolated Vitamin E Deficiency", "Angelman Syndrome", "Prader-Willi Syndrome", "Homocystinuria", "Permanent Neonatal Diabetes Mellitus", "Transient Neonatal Diabetes Mellitus", "Factor VII Deficiency", "Glycogen Storage Disease Type IXA1", "Glycogen Storage Disease, Type IXA2", "Glycogen Storage Disease IC", "Glycogen Storage Disease Type IB", "Central Hypoventilation Syndrome With or Without Hirschsprung Disease" ], "interventions": [ { "name": "Confirmatory Testing", "type": "DIAGNOSTIC_TEST" } ], "intervention_types": [ "DIAGNOSTIC_TEST" ], "sponsor": "RTI International", "sponsor_class": "OTHER", "healthy_volunteers": true, "eligibility": { "minimum_age": "1 Day", "maximum_age": "31 Days", "sex": "ALL", "summary": "1 Day to 31 Days" }, "enrollment_count": 30000, "start_date": "2018-10-15", "completion_date": "2025-12-31", "has_results": false, "last_update_posted_date": "2025-04-04", "last_synced_at": "2026-05-22T08:13:34.450Z", "location_count": 1, "location_summary": "Research Triangle Park, North Carolina", "locations": [ { "city": "Research Triangle Park", "state": "North Carolina" } ], "official_url": "https://clinicaltrials.gov/study/NCT03655223" }, { "nct_id": "NCT03475173", "title": "New Non-invasive Modalities for Assessing Retinal Structure and Function", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "NA" ], "conditions": [ "Ischemic Optic Neuropathy", "Branch Retinal Artery Occlusion", "Hemianopia", "Leber Hereditary Optic Neuropathy", "Acute Zonal Occult Outer Retinopathy" ], "interventions": [ { "name": "LSFG-NAVI", "type": "DEVICE" } ], "intervention_types": [ "DEVICE" ], "sponsor": "Randy Kardon", "sponsor_class": "OTHER", "healthy_volunteers": true, "eligibility": { "minimum_age": "18 Years", "maximum_age": "99 Years", "sex": "ALL", "summary": "18 Years to 99 Years" }, "enrollment_count": 500, "start_date": "2019-05-06", "completion_date": "2028-03-01", "has_results": false, "last_update_posted_date": "2025-11-24", "last_synced_at": "2026-05-22T08:13:34.450Z", "location_count": 1, "location_summary": "Iowa City, Iowa", "locations": [ { "city": "Iowa City", "state": "Iowa" } ], "official_url": "https://clinicaltrials.gov/study/NCT03475173" }, { "nct_id": "NCT03406104", "title": "RESCUE and REVERSE Long-term Follow-up", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE3" ], "conditions": [ "Leber Hereditary Optic Neuropathy" ], "interventions": [ { "name": "GS010", "type": "GENETIC" }, { "name": "Sham Intravitreal Injection", "type": "OTHER" } ], "intervention_types": [ "GENETIC", "OTHER" ], "sponsor": "GenSight Biologics", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "15 Years", "maximum_age": null, "sex": "ALL", "summary": "15 Years and older" }, "enrollment_count": 62, "start_date": "2018-01-09", "completion_date": "2022-07-04", "has_results": true, "last_update_posted_date": "2026-03-02", "last_synced_at": "2026-05-22T08:13:34.450Z", "location_count": 3, "location_summary": "Pasadena, California • Atlanta, Georgia • Philadelphia, Pennsylvania", "locations": [ { "city": "Pasadena", "state": "California" }, { "city": "Atlanta", "state": "Georgia" }, { "city": "Philadelphia", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT03406104" }, { "nct_id": "NCT01694940", "title": "North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Mitochondrial Disorders", "Mitochondrial Genetic Disorders", "Mitochondrial Diseases", "Disorder of Mitochondrial Respiratory Chain Complexes", "Deletion and Duplication of Mitochondrial DNA" ], "interventions": [], "intervention_types": [], "sponsor": "Columbia University", "sponsor_class": "OTHER", "healthy_volunteers": true, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 1000, "start_date": "2011-01-31", "completion_date": "2026-12-31", "has_results": false, "last_update_posted_date": "2026-02-04", "last_synced_at": "2026-05-22T08:13:34.450Z", "location_count": 16, "location_summary": "San Diego, California • Stanford, California • Aurora, Colorado + 12 more", "locations": [ { "city": "San Diego", "state": "California" }, { "city": "Stanford", "state": "California" }, { "city": "Aurora", "state": "Colorado" }, { "city": "Washington D.C.", "state": "District of Columbia" }, { "city": "Gainsville", "state": "Florida" } ], "official_url": "https://clinicaltrials.gov/study/NCT01694940" } ] }