{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Disease&page=10",
    "query": {
      "condition": "Lysosomal Disease",
      "page": 10
    },
    "page_size": 10
  },
  "pagination": {
    "page": 10,
    "page_size": 10,
    "total_count": 567,
    "total_pages": 57,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Disease&page=11&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Disease&page=9&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-14T13:50:57.675Z",
  "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": "NCT02254863",
      "title": "UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Adrenoleukodystrophy",
        "Batten Disease",
        "Mucopolysaccharidosis II",
        "Leukodystrophy, Globoid Cell",
        "Leukodystrophy, Metachromatic",
        "Neimann Pick Disease",
        "Pelizaeus-Merzbacher Disease",
        "Sandhoff Disease",
        "Tay-Sachs Disease",
        "Brain Diseases, Metabolic, Inborn",
        "Alpha-Mannosidosis",
        "Sanfilippo Mucopolysaccharidoses"
      ],
      "interventions": [
        {
          "name": "DUOC-01",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Joanne Kurtzberg, MD",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Week",
        "maximum_age": "22 Years",
        "sex": "ALL",
        "summary": "1 Week to 22 Years"
      },
      "enrollment_count": 40,
      "start_date": "2014-09",
      "completion_date": "2027-10",
      "has_results": false,
      "last_update_posted_date": "2026-09-03",
      "last_synced_at": "2026-10-14T13:50:57.675Z",
      "location_count": 1,
      "location_summary": "Durham, North Carolina",
      "locations": [
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02254863"
    },
    {
      "nct_id": "NCT01885936",
      "title": "Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Pompe Disease"
      ],
      "interventions": [
        {
          "name": "Albuterol",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Duke University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 16,
      "start_date": "2013-06",
      "completion_date": "2016-12-16",
      "has_results": true,
      "last_update_posted_date": "2019-07-15",
      "last_synced_at": "2026-10-14T13:50:57.675Z",
      "location_count": 1,
      "location_summary": "Durham, North Carolina",
      "locations": [
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01885936"
    },
    {
      "nct_id": "NCT01307098",
      "title": "Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Cholesterol Ester Storage Disease(CESD)",
        "Lysosomal Acid Lipase Deficiency",
        "LAL-Deficiency"
      ],
      "interventions": [
        {
          "name": "Sebelipase alfa 0.35 mg/kg",
          "type": "DRUG"
        },
        {
          "name": "Sebelipase alfa 1 mg/kg",
          "type": "DRUG"
        },
        {
          "name": "Sebelipase alfa 3 mg/kg",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Alexion Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "18 Years to 65 Years"
      },
      "enrollment_count": 9,
      "start_date": "2011-04-25",
      "completion_date": "2012-01-06",
      "has_results": true,
      "last_update_posted_date": "2018-12-11",
      "last_synced_at": "2026-10-14T13:50:57.675Z",
      "location_count": 3,
      "location_summary": "Stanford, California • New York, New York • Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01307098"
    },
    {
      "nct_id": "NCT01173016",
      "title": "Administration of IV Laronidase Post Bone Marrow Transplant in Hurler",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Hurler Syndrome"
      ],
      "interventions": [
        {
          "name": "Laronidase",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Masonic Cancer Center, University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "14 Years",
        "sex": "ALL",
        "summary": "Up to 14 Years"
      },
      "enrollment_count": 11,
      "start_date": "2012-05-29",
      "completion_date": "2016-03-04",
      "has_results": true,
      "last_update_posted_date": "2020-03-20",
      "last_synced_at": "2026-10-14T13:50:57.675Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01173016"
    },
    {
      "nct_id": "NCT05146830",
      "title": "A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04",
      "overall_status": "ENROLLING_BY_INVITATION",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Cystinosis"
      ],
      "interventions": [
        {
          "name": "Safety and Efficacy Assessments",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Stephanie Cherqui",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "14 Years",
        "maximum_age": "50 Years",
        "sex": "ALL",
        "summary": "14 Years to 50 Years"
      },
      "enrollment_count": 50,
      "start_date": "2022-11-14",
      "completion_date": "2036-11-30",
      "has_results": false,
      "last_update_posted_date": "2024-07-03",
      "last_synced_at": "2026-10-14T13:50:57.675Z",
      "location_count": 1,
      "location_summary": "La Jolla, California",
      "locations": [
        {
          "city": "La Jolla",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05146830"
    },
    {
      "nct_id": "NCT06116071",
      "title": "Biomarkers Related to Bone in Pediatric Gaucher Disease",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease",
        "Bone Diseases"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "5 Years to 21 Years"
      },
      "enrollment_count": 20,
      "start_date": "2023-11-25",
      "completion_date": "2025-12-31",
      "has_results": false,
      "last_update_posted_date": "2024-03-13",
      "last_synced_at": "2026-10-14T13:50:57.675Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06116071"
    },
    {
      "nct_id": "NCT05206773",
      "title": "A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "Venglustat (GZ402671)",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Sanofi",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "16 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "16 Years and older"
      },
      "enrollment_count": 122,
      "start_date": "2022-03-11",
      "completion_date": "2027-01-04",
      "has_results": false,
      "last_update_posted_date": "2026-10-13",
      "last_synced_at": "2026-10-14T13:50:57.675Z",
      "location_count": 11,
      "location_summary": "Birmingham, Alabama • Los Angeles, California • Orange, California + 8 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Orlando",
          "state": "Florida"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05206773"
    },
    {
      "nct_id": "NCT02208661",
      "title": "Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Morquio A Syndrome",
        "Mucopolysaccharidosis IV A"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Nadia Ali, PhD",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 12,
      "start_date": "2014-03",
      "completion_date": "2018-03",
      "has_results": false,
      "last_update_posted_date": "2018-04-11",
      "last_synced_at": "2026-10-14T13:50:57.675Z",
      "location_count": 1,
      "location_summary": "Decatur, Georgia",
      "locations": [
        {
          "city": "Decatur",
          "state": "Georgia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02208661"
    },
    {
      "nct_id": "NCT06488261",
      "title": "Long Term Follow-Up for Safety of AVR-RD-02",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "AVR-RD-02",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "John Bernat",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 2,
      "start_date": "2024-02-21",
      "completion_date": "2037-10",
      "has_results": false,
      "last_update_posted_date": "2025-07-22",
      "last_synced_at": "2026-10-14T13:50:57.675Z",
      "location_count": 1,
      "location_summary": "Iowa City, Iowa",
      "locations": [
        {
          "city": "Iowa City",
          "state": "Iowa"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06488261"
    },
    {
      "nct_id": "NCT00144768",
      "title": "A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Mucopolysaccharidosis I",
        "Hurler's Syndrome",
        "Hurler-Scheie Syndrome",
        "Scheie's Syndrome"
      ],
      "interventions": [
        {
          "name": "laronidase",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "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": 25,
      "start_date": "2004-07",
      "completion_date": "2007-05",
      "has_results": false,
      "last_update_posted_date": "2014-05-05",
      "last_synced_at": "2026-10-14T13:50:57.675Z",
      "location_count": 3,
      "location_summary": "Los Angles, California • Hartford, Connecticut • Milwaukee, Wisconsin",
      "locations": [
        {
          "city": "Los Angles",
          "state": "California"
        },
        {
          "city": "Hartford",
          "state": "Connecticut"
        },
        {
          "city": "Milwaukee",
          "state": "Wisconsin"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00144768"
    }
  ]
}