{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Disease&page=21",
    "query": {
      "condition": "Lysosomal Disease",
      "page": 21
    },
    "page_size": 10
  },
  "pagination": {
    "page": 21,
    "page_size": 10,
    "total_count": 567,
    "total_pages": 57,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Disease&page=22&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Disease&page=20&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-16T04:53:18.103Z",
  "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": "NCT01917708",
      "title": "Bone Marrow Transplant With Abatacept for Non-Malignant Diseases",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Hurler Syndrome",
        "Fanconi Anemia",
        "Glanzmann Thrombasthenia",
        "Wiskott-Aldrich Syndrome",
        "Chronic Granulomatous Disease",
        "Severe Congenital Neutropenia",
        "Leukocyte Adhesion Deficiency",
        "Shwachman-Diamond Syndrome",
        "Diamond-Blackfan Anemia",
        "Dyskeratosis-congenita",
        "Chediak-Higashi Syndrome",
        "Severe Aplastic Anemia",
        "Thalassemia Major",
        "Hemophagocytic Lymphohistiocytosis",
        "Sickle Cell Disease"
      ],
      "interventions": [
        {
          "name": "Abatacept",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Emory University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "Up to 21 Years"
      },
      "enrollment_count": 10,
      "start_date": "2014-01",
      "completion_date": "2019-09-19",
      "has_results": false,
      "last_update_posted_date": "2019-12-26",
      "last_synced_at": "2026-10-16T04:53:18.103Z",
      "location_count": 1,
      "location_summary": "Atlanta, Georgia",
      "locations": [
        {
          "city": "Atlanta",
          "state": "Georgia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01917708"
    },
    {
      "nct_id": "NCT06308718",
      "title": "Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients",
      "overall_status": "TERMINATED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Krabbe Disease"
      ],
      "interventions": [
        {
          "name": "FBX-101",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Forge Biologics, Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 2,
      "start_date": "2024-08-06",
      "completion_date": "2025-04-11",
      "has_results": false,
      "last_update_posted_date": "2025-09-05",
      "last_synced_at": "2026-10-16T04:53:18.103Z",
      "location_count": 1,
      "location_summary": "Ann Arbor, Michigan",
      "locations": [
        {
          "city": "Ann Arbor",
          "state": "Michigan"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06308718"
    },
    {
      "nct_id": "NCT04046224",
      "title": "Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "ST-920",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Sangamo Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 36,
      "start_date": "2019-07-23",
      "completion_date": "2025-04-10",
      "has_results": true,
      "last_update_posted_date": "2026-04-14",
      "last_synced_at": "2026-10-16T04:53:18.103Z",
      "location_count": 9,
      "location_summary": "Irvine, California • Tampa, Florida • Atlanta, Georgia + 6 more",
      "locations": [
        {
          "city": "Irvine",
          "state": "California"
        },
        {
          "city": "Tampa",
          "state": "Florida"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Iowa City",
          "state": "Iowa"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04046224"
    },
    {
      "nct_id": "NCT02838368",
      "title": "Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Pompe Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of California, Irvine",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "8 Years and older"
      },
      "enrollment_count": 921,
      "start_date": "2015-07",
      "completion_date": "2018-12-01",
      "has_results": false,
      "last_update_posted_date": "2019-02-25",
      "last_synced_at": "2026-10-16T04:53:18.103Z",
      "location_count": 1,
      "location_summary": "Irvine, California",
      "locations": [
        {
          "city": "Irvine",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02838368"
    },
    {
      "nct_id": "NCT02489344",
      "title": "Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "GZ/SAR402671",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "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": 8,
      "start_date": "2015-07-07",
      "completion_date": "2018-11-20",
      "has_results": true,
      "last_update_posted_date": "2021-03-23",
      "last_synced_at": "2026-10-16T04:53:18.103Z",
      "location_count": 3,
      "location_summary": "Atlanta, Georgia • Cincinnati, Ohio • Fairfax, Virginia",
      "locations": [
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02489344"
    },
    {
      "nct_id": "NCT00030134",
      "title": "Data Collection in Women With Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "FEMALE",
        "summary": "Female only"
      },
      "enrollment_count": 60,
      "start_date": "2002-01",
      "completion_date": "2003-02",
      "has_results": false,
      "last_update_posted_date": "2008-03-04",
      "last_synced_at": "2026-10-16T04:53:18.103Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00030134"
    },
    {
      "nct_id": "NCT05492799",
      "title": "Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE",
      "overall_status": "ENROLLING_BY_INVITATION",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "MPS III B"
      ],
      "interventions": [
        {
          "name": "AX 250",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Allievex Corporation",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 15,
      "start_date": "2022-12-02",
      "completion_date": "2027-12",
      "has_results": false,
      "last_update_posted_date": "2023-08-09",
      "last_synced_at": "2026-10-16T04:53:18.103Z",
      "location_count": 2,
      "location_summary": "Oakland, California • Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05492799"
    },
    {
      "nct_id": "NCT03614234",
      "title": "Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "pegunigalsidase alfa",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Chiesi Farmaceutici S.p.A.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 29,
      "start_date": "2018-11-13",
      "completion_date": "2026-04-13",
      "has_results": false,
      "last_update_posted_date": "2026-05-22",
      "last_synced_at": "2026-10-16T04:53:18.103Z",
      "location_count": 7,
      "location_summary": "Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Iowa City",
          "state": "Iowa"
        },
        {
          "city": "Grand Rapids",
          "state": "Michigan"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03614234"
    },
    {
      "nct_id": "NCT04628364",
      "title": "The Natural History of Metachromatic Leukodystrophy Study (HOME Study)",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Metachromatic Leukodystrophy"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Organization for Rare Disorders",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 21,
      "start_date": "2020-10-01",
      "completion_date": "2025-03-30",
      "has_results": false,
      "last_update_posted_date": "2025-06-08",
      "last_synced_at": "2026-10-16T04:53:18.103Z",
      "location_count": 1,
      "location_summary": "Danbury, Connecticut",
      "locations": [
        {
          "city": "Danbury",
          "state": "Connecticut"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04628364"
    },
    {
      "nct_id": "NCT00176891",
      "title": "Stem Cell Transplant w/Laronidase for Hurler",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Mucopolysaccharidosis I",
        "Hurler Syndrome"
      ],
      "interventions": [
        {
          "name": "Stem Cell Transplant",
          "type": "PROCEDURE"
        },
        {
          "name": "Laronidase ERT",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "PROCEDURE",
        "DRUG"
      ],
      "sponsor": "Masonic Cancer Center, University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "7 Years",
        "sex": "ALL",
        "summary": "Up to 7 Years"
      },
      "enrollment_count": 25,
      "start_date": "2004-03",
      "completion_date": "2012-11",
      "has_results": true,
      "last_update_posted_date": "2019-07-11",
      "last_synced_at": "2026-10-16T04:53:18.103Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00176891"
    }
  ]
}