{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Storage+Disorder",
    "query": {
      "condition": "Lysosomal Storage Disorder"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 555,
    "total_pages": 56,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Storage+Disorder&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-07-22T11:39:50.283Z",
  "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": "NCT04040049",
      "title": "A Fabry Disease Gene Therapy Study",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Fabry Disease",
        "Lysosomal Storage Diseases"
      ],
      "interventions": [
        {
          "name": "FLT190",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "Spur Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "MALE",
        "summary": "18 Years and older · Male only"
      },
      "enrollment_count": 3,
      "start_date": "2019-07-08",
      "completion_date": "2023-05-02",
      "has_results": false,
      "last_update_posted_date": "2023-06-05",
      "last_synced_at": "2026-07-22T11:39:50.283Z",
      "location_count": 4,
      "location_summary": "Los Angeles, California • New York, New York • Pittsburgh, Pennsylvania + 1 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04040049"
    },
    {
      "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": "2026-06-11",
      "last_synced_at": "2026-07-22T11:39:50.283Z",
      "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": "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": "2026-07-22T11:39:50.283Z",
      "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": "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": "2026-07-22T11:39:50.283Z",
      "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": "NCT04049760",
      "title": "Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) 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": "12 Years",
        "maximum_age": "17 Years",
        "sex": "ALL",
        "summary": "12 Years to 17 Years"
      },
      "enrollment_count": 16,
      "start_date": "2019-10-11",
      "completion_date": "2024-11-29",
      "has_results": true,
      "last_update_posted_date": "2026-02-05",
      "last_synced_at": "2026-07-22T11:39:50.283Z",
      "location_count": 5,
      "location_summary": "Tampa, Florida • Atlanta, Georgia • Minneapolis, Minnesota + 2 more",
      "locations": [
        {
          "city": "Tampa",
          "state": "Florida"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04049760"
    },
    {
      "nct_id": "NCT01218659",
      "title": "Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "migalastat hydrochloride",
          "type": "DRUG"
        },
        {
          "name": "agalsidase",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "DRUG",
        "BIOLOGICAL"
      ],
      "sponsor": "Amicus Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "16 Years",
        "maximum_age": "74 Years",
        "sex": "ALL",
        "summary": "16 Years to 74 Years"
      },
      "enrollment_count": 68,
      "start_date": "2011-09-08",
      "completion_date": "2015-05-28",
      "has_results": true,
      "last_update_posted_date": "2018-11-01",
      "last_synced_at": "2026-07-22T11:39:50.283Z",
      "location_count": 8,
      "location_summary": "Los Angeles, California • Aurora, Colorado • Decatur, Georgia + 5 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Decatur",
          "state": "Georgia"
        },
        {
          "city": "Grand Rapids",
          "state": "Michigan"
        },
        {
          "city": "Portland",
          "state": "Oregon"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01218659"
    },
    {
      "nct_id": "NCT03041324",
      "title": "Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Mucopolysaccharidosis II",
        "MPS II"
      ],
      "interventions": [
        {
          "name": "SB-913",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Sangamo Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "5 Years to 65 Years"
      },
      "enrollment_count": 9,
      "start_date": "2017-05-11",
      "completion_date": "2021-05-07",
      "has_results": true,
      "last_update_posted_date": "2022-10-25",
      "last_synced_at": "2026-07-22T11:39:50.283Z",
      "location_count": 5,
      "location_summary": "Oakland, California • Chicago, Illinois • New York, New York + 2 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Chapel Hill",
          "state": "North Carolina"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03041324"
    },
    {
      "nct_id": "NCT04252066",
      "title": "A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "migalastat",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Amicus Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "FEMALE",
        "summary": "Female only"
      },
      "enrollment_count": 20,
      "start_date": "2025-03-18",
      "completion_date": "2030-02",
      "has_results": false,
      "last_update_posted_date": "2025-07-08",
      "last_synced_at": "2026-07-22T11:39:50.283Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04252066"
    },
    {
      "nct_id": "NCT03190837",
      "title": "A Long-term Follow-up Study of Gaucher Disease",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Duke University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 200,
      "start_date": "2017-06-12",
      "completion_date": "2027-01",
      "has_results": false,
      "last_update_posted_date": "2025-09-22",
      "last_synced_at": "2026-07-22T11:39:50.283Z",
      "location_count": 1,
      "location_summary": "Durham, North Carolina",
      "locations": [
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03190837"
    },
    {
      "nct_id": "NCT03952637",
      "title": "A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Lysosomal Diseases",
        "Gangliosidosis",
        "GM1"
      ],
      "interventions": [
        {
          "name": "AAV9-GLB1",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Abdominal ultrasound",
          "type": "PROCEDURE"
        },
        {
          "name": "Rituximab",
          "type": "DRUG"
        },
        {
          "name": "Sirolimus",
          "type": "DRUG"
        },
        {
          "name": "Methylprednisolone",
          "type": "DRUG"
        },
        {
          "name": "Prednisone",
          "type": "DRUG"
        },
        {
          "name": "Audiology assessment with ABR",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Bone density scan (DEXA)",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Electrocardiogram (EKG)",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Echocardiogram",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Electroencephalogram (EEG) awake and extended overnight",
          "type": "OTHER"
        },
        {
          "name": "Laboratory tests",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Lumbar puncture",
          "type": "PROCEDURE"
        },
        {
          "name": "Brain MRI/MRS/fMRI",
          "type": "PROCEDURE"
        },
        {
          "name": "Neurocognitive testing",
          "type": "BEHAVIORAL"
        },
        {
          "name": "Neurology exam",
          "type": "OTHER"
        },
        {
          "name": "PICC or other Central line placement",
          "type": "BEHAVIORAL"
        },
        {
          "name": "Skeletal survey",
          "type": "PROCEDURE"
        },
        {
          "name": "Skin biopsy",
          "type": "PROCEDURE"
        },
        {
          "name": "Speech and modified barium swallow study",
          "type": "PROCEDURE"
        },
        {
          "name": "Ophthalmology exam",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "PROCEDURE",
        "DRUG",
        "DIAGNOSTIC_TEST",
        "OTHER",
        "BEHAVIORAL"
      ],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "12 Years",
        "sex": "ALL",
        "summary": "6 Months to 12 Years"
      },
      "enrollment_count": 54,
      "start_date": "2019-08-19",
      "completion_date": "2028-01-01",
      "has_results": false,
      "last_update_posted_date": "2025-12-11",
      "last_synced_at": "2026-07-22T11:39:50.283Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03952637"
    }
  ]
}