{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Storage+Diseases",
    "query": {
      "condition": "Lysosomal Storage Diseases"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 553,
    "total_pages": 56,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Storage+Diseases&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-05-21T23:40:22.466Z",
  "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": "NCT03227042",
      "title": "A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Mucopolysaccharidosis Type IIIB"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Allievex Corporation",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "18 Years",
        "sex": "ALL",
        "summary": "Up to 18 Years"
      },
      "enrollment_count": 44,
      "start_date": "2017-11-16",
      "completion_date": "2025-12",
      "has_results": false,
      "last_update_posted_date": "2022-10-26",
      "last_synced_at": "2026-05-21T23:40:22.466Z",
      "location_count": 1,
      "location_summary": "Oakland, California",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03227042"
    },
    {
      "nct_id": "NCT01920828",
      "title": "Gait Analysis in MPS IVA",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "MPS IVA",
        "Morquio Syndrome"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Oregon Health and Science University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "1 Year and older"
      },
      "enrollment_count": 10,
      "start_date": "2013-08",
      "completion_date": "2017-06",
      "has_results": false,
      "last_update_posted_date": "2019-10-17",
      "last_synced_at": "2026-05-21T23:40:22.466Z",
      "location_count": 1,
      "location_summary": "Portland, Oregon",
      "locations": [
        {
          "city": "Portland",
          "state": "Oregon"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01920828"
    },
    {
      "nct_id": "NCT00146770",
      "title": "Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Mucopolysaccharidosis I",
        "Hurler's Syndrome",
        "Hurler-Scheie Syndrome",
        "Scheie Syndrome"
      ],
      "interventions": [
        {
          "name": "Aldurazyme",
          "type": "BIOLOGICAL"
        },
        {
          "name": "placebo",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 45,
      "start_date": "2001-05",
      "completion_date": "2005-03",
      "has_results": true,
      "last_update_posted_date": "2015-04-03",
      "last_synced_at": "2026-05-21T23:40:22.466Z",
      "location_count": 8,
      "location_summary": "Mobile, Alabama • Atlanta, Georgia • Rochester, New York + 5 more",
      "locations": [
        {
          "city": "Mobile",
          "state": "Alabama"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Rochester",
          "state": "New York"
        },
        {
          "city": "Chapel Hill",
          "state": "North Carolina"
        },
        {
          "city": "Toledo",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00146770"
    },
    {
      "nct_id": "NCT06164951",
      "title": "A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Achondroplasia"
      ],
      "interventions": [
        {
          "name": "Infigratinib 0.25 mg/kg/day",
          "type": "DRUG"
        },
        {
          "name": "Placebo Comparator 0.25 mg/kg/day",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "QED Therapeutics, a BridgeBio company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": "17 Years",
        "sex": "ALL",
        "summary": "3 Years to 17 Years"
      },
      "enrollment_count": 114,
      "start_date": "2023-11-10",
      "completion_date": "2025-12-18",
      "has_results": false,
      "last_update_posted_date": "2026-03-17",
      "last_synced_at": "2026-05-21T23:40:22.466Z",
      "location_count": 7,
      "location_summary": "San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 4 more",
      "locations": [
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Baltimore",
          "state": "Maryland"
        },
        {
          "city": "Columbia",
          "state": "Missouri"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06164951"
    },
    {
      "nct_id": "NCT05634512",
      "title": "Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Hematopoietic Cell Transplantation",
        "Mucopolysaccharidosis Type I"
      ],
      "interventions": [
        {
          "name": "Laronidase therapy and a stem cell transplant",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Masonic Cancer Center, University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "0 Years",
        "maximum_age": "3 Years",
        "sex": "ALL",
        "summary": "0 Years to 3 Years"
      },
      "enrollment_count": 13,
      "start_date": "2022-11-17",
      "completion_date": "2026-10-01",
      "has_results": false,
      "last_update_posted_date": "2025-10-30",
      "last_synced_at": "2026-05-21T23:40:22.466Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05634512"
    },
    {
      "nct_id": "NCT00001972",
      "title": "PET Scan of Brain Metabolism in Relation to Age and Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Alzheimer's Disease",
        "Brain Neoplasm",
        "Niemann Pick Disease"
      ],
      "interventions": [
        {
          "name": "15 O Water",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "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": 123,
      "start_date": "1994-09",
      "completion_date": "2002-08",
      "has_results": false,
      "last_update_posted_date": "2008-03-04",
      "last_synced_at": "2026-05-21T23:40:22.466Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00001972"
    },
    {
      "nct_id": "NCT00343577",
      "title": "Antiproteinuric Agents and Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease",
        "Proteinuria"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Alabama at Birmingham",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "14 Years",
        "maximum_age": "95 Years",
        "sex": "ALL",
        "summary": "14 Years to 95 Years"
      },
      "enrollment_count": 12,
      "start_date": "2001-01",
      "completion_date": "2006-12",
      "has_results": false,
      "last_update_posted_date": "2013-11-19",
      "last_synced_at": "2026-05-21T23:40:22.466Z",
      "location_count": 1,
      "location_summary": "Birmingham, Alabama",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00343577"
    },
    {
      "nct_id": "NCT04221451",
      "title": "A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Tay-Sachs Disease",
        "Sandhoff Disease"
      ],
      "interventions": [
        {
          "name": "venglustat GZ402671",
          "type": "DRUG"
        },
        {
          "name": "placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "2 Years and older"
      },
      "enrollment_count": 75,
      "start_date": "2020-06-29",
      "completion_date": "2024-12-26",
      "has_results": true,
      "last_update_posted_date": "2026-01-28",
      "last_synced_at": "2026-05-21T23:40:22.466Z",
      "location_count": 5,
      "location_summary": "Los Angeles, California • Atlanta, Georgia • Bethesda, Maryland + 2 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Bethesda",
          "state": "Maryland"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04221451"
    },
    {
      "nct_id": "NCT04721366",
      "title": "A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "Standard of Care",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Takeda",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "5 Years",
        "sex": "ALL",
        "summary": "Up to 5 Years"
      },
      "enrollment_count": 11,
      "start_date": "2021-01-08",
      "completion_date": "2023-04-17",
      "has_results": false,
      "last_update_posted_date": "2024-03-29",
      "last_synced_at": "2026-05-21T23:40:22.466Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04721366"
    },
    {
      "nct_id": "NCT02574286",
      "title": "Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "Velaglucerase alfa",
          "type": "DRUG"
        },
        {
          "name": "Vitamin D",
          "type": "DIETARY_SUPPLEMENT"
        }
      ],
      "intervention_types": [
        "DRUG",
        "DIETARY_SUPPLEMENT"
      ],
      "sponsor": "Shire",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "70 Years",
        "sex": "ALL",
        "summary": "18 Years to 70 Years"
      },
      "enrollment_count": 21,
      "start_date": "2016-06-29",
      "completion_date": "2020-11-30",
      "has_results": true,
      "last_update_posted_date": "2022-02-01",
      "last_synced_at": "2026-05-21T23:40:22.466Z",
      "location_count": 8,
      "location_summary": "Beverly Hills, California • Los Angeles, California • Atlanta, Georgia + 5 more",
      "locations": [
        {
          "city": "Beverly Hills",
          "state": "California"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02574286"
    }
  ]
}