{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Disease&page=19",
    "query": {
      "condition": "Lysosomal Disease",
      "page": 19
    },
    "page_size": 10
  },
  "pagination": {
    "page": 19,
    "page_size": 10,
    "total_count": 567,
    "total_pages": 57,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Disease&page=20&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Disease&page=18&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-15T21:33:33.698Z",
  "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": "NCT00920647",
      "title": "A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Hunter Syndrome"
      ],
      "interventions": [
        {
          "name": "Control",
          "type": "OTHER"
        },
        {
          "name": "Idursulfase IT (1 mg)",
          "type": "DRUG"
        },
        {
          "name": "Idursulfase IT (10 mg)",
          "type": "DRUG"
        },
        {
          "name": "Idursulfase IT (30 mg)",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "OTHER",
        "DRUG"
      ],
      "sponsor": "Shire",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": "18 Years",
        "sex": "MALE",
        "summary": "3 Years to 18 Years · Male only"
      },
      "enrollment_count": 16,
      "start_date": "2009-11-18",
      "completion_date": "2012-10-29",
      "has_results": true,
      "last_update_posted_date": "2021-06-28",
      "last_synced_at": "2026-10-15T21:33:33.698Z",
      "location_count": 1,
      "location_summary": "Chapel Hill, North Carolina",
      "locations": [
        {
          "city": "Chapel Hill",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00920647"
    },
    {
      "nct_id": "NCT02432144",
      "title": "A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Sly Syndrome",
        "MPS VII",
        "Mucopolysaccharidosis",
        "Mucopolysaccharidosis VII"
      ],
      "interventions": [
        {
          "name": "UX003",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Ultragenyx Pharmaceutical Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "5 Years and older"
      },
      "enrollment_count": 12,
      "start_date": "2015-11-10",
      "completion_date": "2019-01-14",
      "has_results": true,
      "last_update_posted_date": "2020-07-30",
      "last_synced_at": "2026-10-15T21:33:33.698Z",
      "location_count": 4,
      "location_summary": "Oakland, California • Orange, California • Minneapolis, Minnesota + 1 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02432144"
    },
    {
      "nct_id": "NCT03887533",
      "title": "Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C1"
      ],
      "interventions": [
        {
          "name": "VTS-270",
          "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": "3 Years",
        "maximum_age": "60 Years",
        "sex": "ALL",
        "summary": "3 Years to 60 Years"
      },
      "enrollment_count": 2,
      "start_date": "2020-01-06",
      "completion_date": "2021-10-25",
      "has_results": true,
      "last_update_posted_date": "2022-08-30",
      "last_synced_at": "2026-10-15T21:33:33.698Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03887533"
    },
    {
      "nct_id": "NCT06019728",
      "title": "A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Fabry's Disease"
      ],
      "interventions": [
        {
          "name": "AGALSIDASE BETA (GZ419828)",
          "type": "DRUG"
        },
        {
          "name": "Acetaminophen",
          "type": "DRUG"
        },
        {
          "name": "Diphenhydramine",
          "type": "DRUG"
        },
        {
          "name": "Dexamethasone",
          "type": "DRUG"
        },
        {
          "name": "Montelukast",
          "type": "DRUG"
        },
        {
          "name": "Loratadine",
          "type": "DRUG"
        },
        {
          "name": "Cetirizine",
          "type": "DRUG"
        },
        {
          "name": "Fexofenadine",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Sanofi",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "2 Years to 65 Years"
      },
      "enrollment_count": 8,
      "start_date": "2023-11-10",
      "completion_date": "2024-10-25",
      "has_results": true,
      "last_update_posted_date": "2025-10-21",
      "last_synced_at": "2026-10-15T21:33:33.698Z",
      "location_count": 5,
      "location_summary": "Santa Monica, California • Atlanta, Georgia • Grand Rapids, Michigan + 2 more",
      "locations": [
        {
          "city": "Santa Monica",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Grand Rapids",
          "state": "Michigan"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06019728"
    },
    {
      "nct_id": "NCT02194985",
      "title": "Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "migalastat HCl 150 mg",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Amicus Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 84,
      "start_date": "2015-03-14",
      "completion_date": "2019-10-23",
      "has_results": true,
      "last_update_posted_date": "2020-12-21",
      "last_synced_at": "2026-10-15T21:33:33.698Z",
      "location_count": 7,
      "location_summary": "Atlanta, Georgia • Kansas City, Kansas • Grand Rapids, Michigan + 4 more",
      "locations": [
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Kansas City",
          "state": "Kansas"
        },
        {
          "city": "Grand Rapids",
          "state": "Michigan"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Portland",
          "state": "Oregon"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02194985"
    },
    {
      "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": "2026-10-15T21:33:33.698Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00001234"
    },
    {
      "nct_id": "NCT01521429",
      "title": "Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Mucopolysaccharidoses"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": "35 Years",
        "sex": "ALL",
        "summary": "5 Years to 35 Years"
      },
      "enrollment_count": 55,
      "start_date": "2009-08",
      "completion_date": "2019-07-31",
      "has_results": false,
      "last_update_posted_date": "2024-10-22",
      "last_synced_at": "2026-10-15T21:33:33.698Z",
      "location_count": 3,
      "location_summary": "Oakland, California • Torrance, California • Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Torrance",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01521429"
    },
    {
      "nct_id": "NCT04713475",
      "title": "Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "GM1 Gangliosidosis",
        "GM1 Gangliosidosis, Type I",
        "GM1 Gangliosidosis, Type 2",
        "Beta-Galactosidase-1 (GLB1) Deficiency"
      ],
      "interventions": [
        {
          "name": "PBGM01",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Gemma Biotherapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Month",
        "maximum_age": "24 Months",
        "sex": "ALL",
        "summary": "1 Month to 24 Months"
      },
      "enrollment_count": 26,
      "start_date": "2021-03-17",
      "completion_date": "2029-02",
      "has_results": false,
      "last_update_posted_date": "2026-06-30",
      "last_synced_at": "2026-10-15T21:33:33.698Z",
      "location_count": 4,
      "location_summary": "Oakland, California • Minneapolis, Minnesota • New Brunswick, New Jersey + 1 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "New Brunswick",
          "state": "New Jersey"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04713475"
    },
    {
      "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": "2026-10-15T21:33:33.698Z",
      "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": "NCT04227600",
      "title": "A Study of JR-171 in Patients With Mucopolysaccharidosis I",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Mucopolysaccharidosis I"
      ],
      "interventions": [
        {
          "name": "JR-171 (lepunafusp alfa)",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "JCR Pharmaceuticals Co., Ltd.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "0 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "0 Years and older"
      },
      "enrollment_count": 18,
      "start_date": "2020-09-01",
      "completion_date": "2022-08-02",
      "has_results": true,
      "last_update_posted_date": "2025-03-19",
      "last_synced_at": "2026-10-15T21:33:33.698Z",
      "location_count": 1,
      "location_summary": "Oakland, California",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04227600"
    }
  ]
}