{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Disease&page=6", "query": { "condition": "Lysosomal Disease", "page": 6 }, "page_size": 10 }, "pagination": { "page": 6, "page_size": 10, "total_count": 567, "total_pages": 57, "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Disease&page=7&page_size=10", "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Disease&page=5&page_size=10" }, "source": "remote", "last_synced_at": "2026-10-11T11:33:08.241Z", "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": "NCT06270316", "title": "Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Fabry Disease" ], "interventions": [ { "name": "AMT-191", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "UniQure Biopharma B.V.", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": "65 Years", "sex": "MALE", "summary": "18 Years to 65 Years · Male only" }, "enrollment_count": 20, "start_date": "2024-06-05", "completion_date": "2031-11-30", "has_results": false, "last_update_posted_date": "2026-09-24", "last_synced_at": "2026-10-11T11:33:08.241Z", "location_count": 10, "location_summary": "Birmingham, Alabama • Los Angeles, California • Atlanta, Georgia + 7 more", "locations": [ { "city": "Birmingham", "state": "Alabama" }, { "city": "Los Angeles", "state": "California" }, { "city": "Atlanta", "state": "Georgia" }, { "city": "Chicago", "state": "Illinois" }, { "city": "Minneapolis", "state": "Minnesota" } ], "official_url": "https://clinicaltrials.gov/study/NCT06270316" }, { "nct_id": "NCT03604835", "title": "Mucopolysaccharidosis VII Disease Monitoring Program", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Mucopolysaccharidosis VII", "MPS VII", "MPS 7", "Sly Syndrome" ], "interventions": [ { "name": "No Intervention", "type": "OTHER" } ], "intervention_types": [ "OTHER" ], "sponsor": "Ultragenyx Pharmaceutical Inc", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 50, "start_date": "2018-01-29", "completion_date": "2032-04", "has_results": false, "last_update_posted_date": "2026-08-27", "last_synced_at": "2026-10-11T11:33:08.241Z", "location_count": 7, "location_summary": "Orange, California • Washington D.C., District of Columbia • Chicago, Illinois + 4 more", "locations": [ { "city": "Orange", "state": "California" }, { "city": "Washington D.C.", "state": "District of Columbia" }, { "city": "Chicago", "state": "Illinois" }, { "city": "Ann Arbor", "state": "Michigan" }, { "city": "New York", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT03604835" }, { "nct_id": "NCT03513328", "title": "Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Bone Marrow Failure Syndrome", "Thalassemia", "Sickle Cell Disease", "Diamond Blackfan Anemia", "Acquired Neutropenia in Newborn", "Acquired Anemia Hemolytic", "Acquired Thrombocytopenia", "Hemophagocytic Lymphohistiocytoses", "Wiskott-Aldrich Syndrome", "Chronic Granulomatous Disease", "Common Variable Immunodeficiency", "X-linked Lymphoproliferative Disease", "Severe Combined Immunodeficiency", "Hurler Syndrome", "Mannosidosis", "Adrenoleukodystrophy" ], "interventions": [ { "name": "Thiotepa--single daily dose", "type": "DRUG" }, { "name": "Thiotepa--escalated dose", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "University of Florida", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "3 Months", "maximum_age": "39 Years", "sex": "ALL", "summary": "3 Months to 39 Years" }, "enrollment_count": 6, "start_date": "2018-06-15", "completion_date": "2023-02-19", "has_results": true, "last_update_posted_date": "2023-09-15", "last_synced_at": "2026-10-11T11:33:08.241Z", "location_count": 1, "location_summary": "Gainesville, Florida", "locations": [ { "city": "Gainesville", "state": "Florida" } ], "official_url": "https://clinicaltrials.gov/study/NCT03513328" }, { "nct_id": "NCT01678898", "title": "Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Fabry Disease" ], "interventions": [ { "name": "PRX-102", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Protalix", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 18, "start_date": "2012-10", "completion_date": "2016-03-06", "has_results": true, "last_update_posted_date": "2023-09-13", "last_synced_at": "2026-10-11T11:33:08.241Z", "location_count": 9, "location_summary": "Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 6 more", "locations": [ { "city": "Sacramento", "state": "California" }, { "city": "Atlanta", "state": "Georgia" }, { "city": "Iowa City", "state": "Iowa" }, { "city": "Kansas City", "state": "Kansas" }, { "city": "Baltimore", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT01678898" }, { "nct_id": "NCT01556516", "title": "Pregnancy and Birth Outcome in Women With Pompe Disease", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Pompe Disease" ], "interventions": [], "intervention_types": [], "sponsor": "O & O Alpan LLC", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "FEMALE", "summary": "18 Years and older · Female only" }, "enrollment_count": 20, "start_date": "2012-02", "completion_date": "2016-07", "has_results": false, "last_update_posted_date": "2016-09-28", "last_synced_at": "2026-10-11T11:33:08.241Z", "location_count": 1, "location_summary": "Fairfax, Virginia", "locations": [ { "city": "Fairfax", "state": "Virginia" } ], "official_url": "https://clinicaltrials.gov/study/NCT01556516" }, { "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": "2026-10-11T11:33:08.241Z", "location_count": 1, "location_summary": "Minneapolis, Minnesota", "locations": [ { "city": "Minneapolis", "state": "Minnesota" } ], "official_url": "https://clinicaltrials.gov/study/NCT02171104" }, { "nct_id": "NCT00001234", "title": "Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Gaucher's Disease" ], "interventions": [ { "name": "human glucocerebrosidase cDNA", "type": "GENETIC" } ], "intervention_types": [ "GENETIC" ], "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)", "sponsor_class": "NIH", "healthy_volunteers": true, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 120, "start_date": "1988-01", "completion_date": "2002-04", "has_results": false, "last_update_posted_date": "2008-03-04", "last_synced_at": "2026-10-11T11:33:08.241Z", "location_count": 1, "location_summary": "Bethesda, Maryland", "locations": [ { "city": "Bethesda", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT00001234" }, { "nct_id": "NCT00744692", "title": "Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Non Malignant Disorders", "Immunodeficiencies", "Congenital Marrow Failures", "Hemoglobinopathies", "Inborn Errors of Metabolism", "Sickle Cell", "Thalassemia", "Lysosomal Storage Disease" ], "interventions": [ { "name": "Unrelated Umbilical Cord Blood Transplant", "type": "BIOLOGICAL" }, { "name": "Reduced Intensity Conditioning", "type": "DRUG" } ], "intervention_types": [ "BIOLOGICAL", "DRUG" ], "sponsor": "Duke University", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "21 Years", "sex": "ALL", "summary": "Up to 21 Years" }, "enrollment_count": 22, "start_date": "2008-10", "completion_date": "2014-04", "has_results": true, "last_update_posted_date": "2014-08-13", "last_synced_at": "2026-10-11T11:33:08.241Z", "location_count": 1, "location_summary": "Durham, North Carolina", "locations": [ { "city": "Durham", "state": "North Carolina" } ], "official_url": "https://clinicaltrials.gov/study/NCT00744692" }, { "nct_id": "NCT00383448", "title": "HSCT for High Risk Inherited Inborn Errors", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Adrenoleukodystrophy", "Metachromatic Leukodystrophy", "Globoid Cell Leukodystrophy", "Tay Sachs Disease", "Sandhoffs Disease", "Wolman Disease", "I-Cell Disease", "Sanfilippo Syndrome", "GM1 Gangliosidosis" ], "interventions": [ { "name": "Clofarabine", "type": "DRUG" }, { "name": "Total body Irradiation", "type": "PROCEDURE" }, { "name": "Melphalan", "type": "DRUG" }, { "name": "Hematopoietic Stem Cell Transplantation", "type": "BIOLOGICAL" }, { "name": "Alemtuzumab", "type": "DRUG" }, { "name": "mycophenylate mofetil", "type": "DRUG" }, { "name": "Cyclosporine A", "type": "DEVICE" }, { "name": "Hydroxyurea", "type": "DRUG" } ], "intervention_types": [ "DRUG", "PROCEDURE", "BIOLOGICAL", "DEVICE" ], "sponsor": "Masonic Cancer Center, University of Minnesota", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "70 Years", "sex": "ALL", "summary": "Up to 70 Years" }, "enrollment_count": 38, "start_date": "2006-09", "completion_date": "2014-09", "has_results": true, "last_update_posted_date": "2019-07-11", "last_synced_at": "2026-10-11T11:33:08.241Z", "location_count": 1, "location_summary": "Minneapolis, Minnesota", "locations": [ { "city": "Minneapolis", "state": "Minnesota" } ], "official_url": "https://clinicaltrials.gov/study/NCT00383448" }, { "nct_id": "NCT02141503", "title": "Clinical Biomarkers in Alpha-mannosidosis", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Alpha-Mannosidosis" ], "interventions": [], "intervention_types": [], "sponsor": "Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)", "sponsor_class": "NIH", "healthy_volunteers": false, "eligibility": { "minimum_age": "5 Years", "maximum_age": "60 Years", "sex": "ALL", "summary": "5 Years to 60 Years" }, "enrollment_count": 11, "start_date": "2014-07-24", "completion_date": "2019-11-29", "has_results": false, "last_update_posted_date": "2019-12-03", "last_synced_at": "2026-10-11T11:33:08.241Z", "location_count": 1, "location_summary": "Bethesda, Maryland", "locations": [ { "city": "Bethesda", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT02141503" } ] }