{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Canavan+Disease", "query": { "condition": "Canavan Disease" }, "page_size": 10 }, "pagination": { "page": 1, "page_size": 10, "total_count": 9, "total_pages": 1, "next_page_url": null, "previous_page_url": null }, "source": "remote", "last_synced_at": "2026-05-22T00:08:14.077Z", "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": "NCT03047369", "title": "The Myelin Disorders Biorepository Project", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Leukodystrophy", "White Matter Disease", "Leukoencephalopathies", "4H Syndrome", "Adrenoleukodystrophy", "AMN", "ALD", "ALD Gene Mutation", "ALD (Adrenoleukodystrophy)", "X-linked Adrenoleukodystrophy", "X-ALD", "Adrenomyeloneuropathy", "Aicardi Goutieres Syndrome", "AGS", "Alexander Disease", "Alexanders Leukodystrophy", "AxD", "ADLD", "Canavan Disease", "CTX", "Cerebrotendinous Xanthomatoses", "Krabbe Disease", "GALC Deficiency", "Globoid Leukodystrophy", "TUBB4A-Related Leukodystrophy", "H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum", "HBSL", "HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity", "LBSL", "Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)", "Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation", "ALSP", "CSF1R Gene Mutation", "HCC - Hypomyelination and Congenital Cataract", "MLC1", "Megalencephalic Leukoencephalopathy With Subcortical Cysts", "MLD", "Metachromatic Leukodystrophy", "PMD", "Pelizaeus-Merzbacher Disease", "PLP1 Null Syndrome", "PLP1 Gene Duplication | Blood or Tissue | Mutations", "Pelizaeus Merzbacher Like Disease", "Peroxisomal Biogenesis Disorder", "Zellweger Syndrome", "Refsum Disease", "Salla Disease", "Sialic Storage Disease", "Sjögren", "Sjogren-Larsson Syndrome", "Van Der Knapp Disease", "Vanishing White Matter Disease", "Charcot-Marie-Tooth", "CMT", "Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency", "Allan-Herndon-Dudley Syndrome", "Cadasil", "Cockayne Syndrome", "Multiple Sulfatase Deficiency", "Gangliosidoses", "GM2 Gangliosidosis", "BPAN", "Labrune Syndrome", "LCC", "Mucopolysaccharidoses", "TBCK-Related Intellectual Disability Syndrome" ], "interventions": [], "intervention_types": [], "sponsor": "Children's Hospital of Philadelphia", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 12000, "start_date": "2016-12-08", "completion_date": "2030-12-08", "has_results": false, "last_update_posted_date": "2025-10-23", "last_synced_at": "2026-05-22T00:08:14.077Z", "location_count": 23, "location_summary": "Los Angeles, California • Orange, California • Palo Alto, California + 18 more", "locations": [ { "city": "Los Angeles", "state": "California" }, { "city": "Orange", "state": "California" }, { "city": "Palo Alto", "state": "California" }, { "city": "Sacramento", "state": "California" }, { "city": "San Diego", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT03047369" }, { "nct_id": "NCT04833907", "title": "rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease", "overall_status": "ENROLLING_BY_INVITATION", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Canavan Disease" ], "interventions": [ { "name": "rAAV-Olig001-ASPA", "type": "DRUG" }, { "name": "Levetiracetam", "type": "DRUG" }, { "name": "Prednisone", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Myrtelle Inc.", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "3 Months", "maximum_age": "60 Months", "sex": "ALL", "summary": "3 Months to 60 Months" }, "enrollment_count": 24, "start_date": "2021-04-01", "completion_date": "2027-08-31", "has_results": false, "last_update_posted_date": "2025-11-14", "last_synced_at": "2026-05-22T00:08:14.077Z", "location_count": 1, "location_summary": "Dayton, Ohio", "locations": [ { "city": "Dayton", "state": "Ohio" } ], "official_url": "https://clinicaltrials.gov/study/NCT04833907" }, { "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": "2026-05-22T00:08:14.077Z", "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": "NCT05317780", "title": "Canavan-Single Patient IND", "overall_status": "NO_LONGER_AVAILABLE", "study_type": "EXPANDED_ACCESS", "phases": [], "conditions": [ "Canavan Disease" ], "interventions": [ { "name": "rAAV9-CB6-AspA", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "University of Florida", "sponsor_class": "OTHER", "healthy_volunteers": null, "eligibility": { "minimum_age": "18 Months", "maximum_age": "24 Months", "sex": "MALE", "summary": "18 Months to 24 Months · Male only" }, "enrollment_count": null, "start_date": null, "completion_date": null, "has_results": false, "last_update_posted_date": "2023-08-23", "last_synced_at": "2026-05-22T00:08:14.077Z", "location_count": 1, "location_summary": "Gainesville, Florida", "locations": [ { "city": "Gainesville", "state": "Florida" } ], "official_url": "https://clinicaltrials.gov/study/NCT05317780" }, { "nct_id": "NCT00654433", "title": "ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases", "overall_status": "TERMINATED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE3" ], "conditions": [ "Inherited Metabolic Diseases", "Lysosomal Storage Disorders", "Peroxisomal Storage Diseases", "Inborn Errors of Metabolism", "Mucopolysaccharidosis" ], "interventions": [ { "name": "ALD-101", "type": "BIOLOGICAL" } ], "intervention_types": [ "BIOLOGICAL" ], "sponsor": "Aldagen", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "16 Years", "sex": "ALL", "summary": "Up to 16 Years" }, "enrollment_count": 40, "start_date": "2008-03", "completion_date": "2011-11", "has_results": false, "last_update_posted_date": "2014-07-08", "last_synced_at": "2026-05-22T00:08:14.077Z", "location_count": 3, "location_summary": "Los Angeles, California • New York, New York • Durham, North Carolina", "locations": [ { "city": "Los Angeles", "state": "California" }, { "city": "New York", "state": "New York" }, { "city": "Durham", "state": "North Carolina" } ], "official_url": "https://clinicaltrials.gov/study/NCT00654433" }, { "nct_id": "NCT02851563", "title": "A Natural History Study of Canavan Disease", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Canavan Disease" ], "interventions": [], "intervention_types": [], "sponsor": "NYU Langone Health", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "99 Years", "sex": "ALL", "summary": "Up to 99 Years" }, "enrollment_count": 21, "start_date": "2016-05", "completion_date": "2019-04", "has_results": false, "last_update_posted_date": "2019-11-01", "last_synced_at": "2026-05-22T00:08:14.077Z", "location_count": 2, "location_summary": "Boston, Massachusetts • New York, New York", "locations": [ { "city": "Boston", "state": "Massachusetts" }, { "city": "New York", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT02851563" }, { "nct_id": "NCT04998396", "title": "A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Canavan Disease" ], "interventions": [ { "name": "AAV9 BBP-812", "type": "BIOLOGICAL" } ], "intervention_types": [ "BIOLOGICAL" ], "sponsor": "Aspa Therapeutics", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "30 Months", "sex": "ALL", "summary": "Up to 30 Months" }, "enrollment_count": 26, "start_date": "2021-09-08", "completion_date": "2032-10-08", "has_results": false, "last_update_posted_date": "2026-04-17", "last_synced_at": "2026-05-22T00:08:14.077Z", "location_count": 4, "location_summary": "Oakland, California • Chicago, Illinois • Boston, Massachusetts + 1 more", "locations": [ { "city": "Oakland", "state": "California" }, { "city": "Chicago", "state": "Illinois" }, { "city": "Boston", "state": "Massachusetts" }, { "city": "New York", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT04998396" }, { "nct_id": "NCT02699190", "title": "LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Leukodystrophy", "White Matter Disease", "4H Syndrome", "Adrenoleukodystrophy", "AMN", "ALD", "ALD (Adrenoleukodystrophy)", "X-linked Adrenoleukodystrophy", "X-ALD", "Adrenomyeloneuropathy", "Aicardi Goutieres Syndrome", "AGS", "Alexander Disease", "Alexanders Leukodystrophy", "AxD", "ADLD", "Canavan Disease", "CTX", "Cerebrotendinous Xanthomatoses", "Krabbe Disease", "GALC Deficiency", "Globoid Leukodystrophy", "TUBB4A-Related Leukodystrophy", "H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum", "HBSL", "HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity", "LBSL", "Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)", "Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation", "ALSP", "CSF1R Gene Mutation", "HCC - Hypomyelination and Congenital Cataract", "MLC1", "Megalencephalic Leukoencephalopathy With Subcortical Cysts 1", "MLD", "Metachromatic Leukodystrophy", "PMD", "Pelizaeus-Merzbacher Disease", "PLP1 Null Syndrome", "PLP1 Gene Duplication | Blood or Tissue | Mutations", "Pelizaeus-Merzbacher-Like Disease, 1", "Peroxisomal Biogenesis Disorder", "Zellweger Syndrome", "Refsum Disease", "Salla Disease", "Sialic Storage Disease", "Sjögren", "Sjogren-Larsson Syndrome", "Van Der Knapp Disease", "Vanishing White Matter Disease", "Charcot-Marie-Tooth", "CMT", "Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency", "Allan-Herndon-Dudley Syndrome", "Cadasil", "Cockayne Syndrome", "Multiple Sulfatase Deficiency", "Gangliosidoses", "GM2 Gangliosidosis", "BPAN", "Labrune Syndrome", "LCC", "Mucopolysaccharidoses", "TBCK-Related Intellectual Disability Syndrome" ], "interventions": [], "intervention_types": [], "sponsor": "Children's Hospital of Philadelphia", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "18 Years", "sex": "ALL", "summary": "Up to 18 Years" }, "enrollment_count": 236, "start_date": "2017-01-06", "completion_date": "2024-10-31", "has_results": true, "last_update_posted_date": "2025-11-10", "last_synced_at": "2026-05-22T00:08:14.077Z", "location_count": 1, "location_summary": "Philadelphia, Pennsylvania", "locations": [ { "city": "Philadelphia", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT02699190" }, { "nct_id": "NCT04126005", "title": "Natural History Study of Patients With Canavan Disease (CANinform Study)", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Canavan Disease" ], "interventions": [], "intervention_types": [], "sponsor": "Aspa Therapeutics", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 67, "start_date": "2019-10-10", "completion_date": "2025-08-31", "has_results": false, "last_update_posted_date": "2026-04-17", "last_synced_at": "2026-05-22T00:08:14.077Z", "location_count": 2, "location_summary": "Oakland, California • Boston, Massachusetts", "locations": [ { "city": "Oakland", "state": "California" }, { "city": "Boston", "state": "Massachusetts" } ], "official_url": "https://clinicaltrials.gov/study/NCT04126005" } ] }