{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Niemann-Pick+Disease%2C+Type+C", "query": { "condition": "Niemann-Pick Disease, Type C" }, "page_size": 10 }, "pagination": { "page": 1, "page_size": 10, "total_count": 29, "total_pages": 3, "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Niemann-Pick+Disease%2C+Type+C&page=2&page_size=10", "previous_page_url": null }, "source": "remote", "last_synced_at": "2026-05-21T23:23:00.292Z", "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": "NCT02171104", "title": "MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis", "overall_status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Mucopolysaccharidosis Disorders", "Hurler Syndrome", "Hunter Syndrome", "Maroteaux Lamy Syndrome", "Sly Syndrome", "Alpha-Mannosidosis", "Fucosidosis", "Aspartylglucosaminuria", "Glycoprotein Metabolic Disorders", "Sphingolipidoses", "Recessive Leukodystrophies", "Globoid Cell Leukodystrophy", "Metachromatic Leukodystrophy", "Niemann-Pick B", "Niemann-Pick C Subtype 2", "Sphingomyelin Deficiency", "Peroxisomal Disorders", "Adrenoleukodystrophy With Cerebral Involvement", "Zellweger Syndrome", "Neonatal Adrenoleukodystrophy", "Infantile Refsum Disease", "Acyl-CoA Oxidase Deficiency", "D-Bifunctional Enzyme Deficiency", "Multifunctional Enzyme Deficiency", "Alpha-methylacyl-CoA Racmase Deficiency", "Mitochondrial Neurogastrointestingal Encephalopathy", "Severe Osteopetrosis", "Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)", "Inherited Metabolic Disorders" ], "interventions": [ { "name": "Stem Cell Transplantation", "type": "BIOLOGICAL" }, { "name": "IMD Preparative Regimen", "type": "DRUG" }, { "name": "Osteopetrosis Only Preparative Regimen", "type": "DRUG" }, { "name": "Osteopetrosis Haploidentical Only Preparative Regimen", "type": "DRUG" }, { "name": "cALD SR-A (Standard-Risk, Regimen A)", "type": "DRUG" }, { "name": "cALD SR-B (Standard-Risk, Regimen B)", "type": "DRUG" }, { "name": "cALD HR-D (High-Risk, Regimen C)", "type": "DRUG" }, { "name": "cALD HR-D (High-Risk, Regimen D)", "type": "DRUG" } ], "intervention_types": [ "BIOLOGICAL", "DRUG" ], "sponsor": "Masonic Cancer Center, University of Minnesota", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "55 Years", "sex": "ALL", "summary": "Up to 55 Years" }, "enrollment_count": 149, "start_date": "2014-07-10", "completion_date": "2029-07-14", "has_results": false, "last_update_posted_date": "2026-01-07", "last_synced_at": "2026-05-21T23:23:00.292Z", "location_count": 1, "location_summary": "Minneapolis, Minnesota", "locations": [ { "city": "Minneapolis", "state": "Minnesota" } ], "official_url": "https://clinicaltrials.gov/study/NCT02171104" }, { "nct_id": "NCT03471143", "title": "Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Niemann-Pick Disease, Type C" ], "interventions": [ { "name": "VTS-270", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Washington University School of Medicine", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "6 Months", "sex": "ALL", "summary": "Up to 6 Months" }, "enrollment_count": 4, "start_date": "2019-02-22", "completion_date": "2024-10-04", "has_results": true, "last_update_posted_date": "2026-01-05", "last_synced_at": "2026-05-21T23:23:00.292Z", "location_count": 1, "location_summary": "St Louis, Missouri", "locations": [ { "city": "St Louis", "state": "Missouri" } ], "official_url": "https://clinicaltrials.gov/study/NCT03471143" }, { "nct_id": "NCT07054515", "title": "A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE3" ], "conditions": [ "Niemann-Pick Type C Disease", "GM1 Gangliosidosis", "GM2 Gangliosidosis" ], "interventions": [ { "name": "AZ-3102", "type": "DRUG" }, { "name": "Placebo", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Azafaros A.G.", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "4 Years", "maximum_age": null, "sex": "ALL", "summary": "4 Years and older" }, "enrollment_count": 147, "start_date": "2025-06-30", "completion_date": "2027-11-04", "has_results": false, "last_update_posted_date": "2026-05-05", "last_synced_at": "2026-05-21T23:23:00.292Z", "location_count": 5, "location_summary": "Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more", "locations": [ { "city": "Oakland", "state": "California" }, { "city": "Minneapolis", "state": "Minnesota" }, { "city": "Rochester", "state": "Minnesota" }, { "city": "Dallas", "state": "Texas" }, { "city": "Fairfax", "state": "Virginia" } ], "official_url": "https://clinicaltrials.gov/study/NCT07054515" }, { "nct_id": "NCT02612129", "title": "Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2", "PHASE3" ], "conditions": [ "Niemann-Pick Disease, Type C" ], "interventions": [ { "name": "arimoclomol", "type": "DRUG" }, { "name": "Placebo", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "ZevraDenmark", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "2 Years", "maximum_age": "18 Years", "sex": "ALL", "summary": "2 Years to 18 Years" }, "enrollment_count": 50, "start_date": "2016-06-14", "completion_date": "2024-10-31", "has_results": true, "last_update_posted_date": "2024-11-29", "last_synced_at": "2026-05-21T23:23:00.292Z", "location_count": 2, "location_summary": "Oakland, California • Rochester, Minnesota", "locations": [ { "city": "Oakland", "state": "California" }, { "city": "Rochester", "state": "Minnesota" } ], "official_url": "https://clinicaltrials.gov/study/NCT02612129" }, { "nct_id": "NCT07082725", "title": "A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE3" ], "conditions": [ "Niemann-Pick Type C Disease" ], "interventions": [ { "name": "Nizubaglustat", "type": "DRUG" }, { "name": "Placebo", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Azafaros A.G.", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "4 Years", "maximum_age": null, "sex": "ALL", "summary": "4 Years and older" }, "enrollment_count": 72, "start_date": "2025-06-30", "completion_date": "2027-11-04", "has_results": false, "last_update_posted_date": "2026-05-05", "last_synced_at": "2026-05-21T23:23:00.292Z", "location_count": 5, "location_summary": "Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more", "locations": [ { "city": "Oakland", "state": "California" }, { "city": "Minneapolis", "state": "Minnesota" }, { "city": "Rochester", "state": "Minnesota" }, { "city": "Dallas", "state": "Texas" }, { "city": "Fairfax", "state": "Virginia" } ], "official_url": "https://clinicaltrials.gov/study/NCT07082725" }, { "nct_id": "NCT02124083", "title": "Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Neimann-Pick Disease" ], "interventions": [ { "name": "Vorinostat", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)", "sponsor_class": "NIH", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": "60 Years", "sex": "ALL", "summary": "18 Years to 60 Years" }, "enrollment_count": 12, "start_date": "2014-04-25", "completion_date": "2016-12-13", "has_results": true, "last_update_posted_date": "2018-02-22", "last_synced_at": "2026-05-21T23:23:00.292Z", "location_count": 1, "location_summary": "Bethesda, Maryland", "locations": [ { "city": "Bethesda", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT02124083" }, { "nct_id": "NCT03643562", "title": "Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System", "overall_status": "TERMINATED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE3" ], "conditions": [ "Niemann-Pick Type C Disease" ], "interventions": [ { "name": "Adrabetadex", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Mandos LLC", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "4 Years", "maximum_age": null, "sex": "ALL", "summary": "4 Years and older" }, "enrollment_count": 27, "start_date": "2018-06-18", "completion_date": "2021-11-02", "has_results": true, "last_update_posted_date": "2024-01-17", "last_synced_at": "2026-05-21T23:23:00.292Z", "location_count": 17, "location_summary": "Little Rock, Arkansas • Loma Linda, California • Los Angeles, California + 13 more", "locations": [ { "city": "Little Rock", "state": "Arkansas" }, { "city": "Loma Linda", "state": "California" }, { "city": "Los Angeles", "state": "California" }, { "city": "Orange", "state": "California" }, { "city": "Aurora", "state": "Colorado" } ], "official_url": "https://clinicaltrials.gov/study/NCT03643562" }, { "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": "2026-05-21T23:23:00.292Z", "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": "NCT04316637", "title": "Early Access Program With Arimoclomol in US Patients With NPC", "overall_status": "AVAILABLE", "study_type": "EXPANDED_ACCESS", "phases": [], "conditions": [ "Niemann-Pick Disease, Type C" ], "interventions": [ { "name": "Arimoclomol", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "ZevraDenmark", "sponsor_class": "INDUSTRY", "healthy_volunteers": null, "eligibility": { "minimum_age": "2 Years", "maximum_age": null, "sex": "ALL", "summary": "2 Years and older" }, "enrollment_count": null, "start_date": null, "completion_date": null, "has_results": false, "last_update_posted_date": "2024-08-16", "last_synced_at": "2026-05-21T23:23:00.292Z", "location_count": 13, "location_summary": "Birmingham, Alabama • Oakland, California • Orange, California + 10 more", "locations": [ { "city": "Birmingham", "state": "Alabama" }, { "city": "Oakland", "state": "California" }, { "city": "Orange", "state": "California" }, { "city": "Miami", "state": "Florida" }, { "city": "Chicago", "state": "Illinois" } ], "official_url": "https://clinicaltrials.gov/study/NCT04316637" }, { "nct_id": "NCT01747135", "title": "Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Niemann-Pick Disease, Type C1" ], "interventions": [ { "name": "VTS-270", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "2 Years", "maximum_age": "25 Years", "sex": "ALL", "summary": "2 Years to 25 Years" }, "enrollment_count": 14, "start_date": "2013-01", "completion_date": "2017-03", "has_results": false, "last_update_posted_date": "2021-07-23", "last_synced_at": "2026-05-21T23:23:00.292Z", "location_count": 1, "location_summary": "Bethesda, Maryland", "locations": [ { "city": "Bethesda", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT01747135" } ] }