{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Diseases",
    "query": {
      "condition": "Lysosomal Diseases"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 564,
    "total_pages": 57,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lysosomal+Diseases&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-06-26T11:31:23.182Z",
  "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": "NCT06539169",
      "title": "FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Alpha-Thalassemia",
        "Beta-Thalassemia",
        "Amyloidosis",
        "Amyotrophic Lateral Sclerosis",
        "Creutzfeld-Jakob Disease",
        "Cystic Fibrosis",
        "Duchenne Muscular Dystrophy",
        "Early-Onset Alzheimer Disease",
        "Ehlers-Danlos Syndrome",
        "Huntington Disease",
        "Gaucher Disease",
        "GM1 Gangliosidosis",
        "Myasthenia Gravis",
        "Pompe Disease",
        "Sickle Cell Disease",
        "Transthyretin Amyloid Cardiomyopathy",
        "Rare Diseases"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "xCures",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 1000,
      "start_date": "2024-06-10",
      "completion_date": "2026-06-10",
      "has_results": false,
      "last_update_posted_date": "2024-11-14",
      "last_synced_at": "2026-06-26T11:31:23.182Z",
      "location_count": 1,
      "location_summary": "Los Altos, California",
      "locations": [
        {
          "city": "Los Altos",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06539169"
    },
    {
      "nct_id": "NCT01074944",
      "title": "A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "Eliglustat tartrate",
          "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": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 170,
      "start_date": "2010-06",
      "completion_date": "2015-10",
      "has_results": true,
      "last_update_posted_date": "2017-02-06",
      "last_synced_at": "2026-06-26T11:31:23.182Z",
      "location_count": 10,
      "location_summary": "San Diego, California • New Haven, Connecticut • Decatur, Georgia + 6 more",
      "locations": [
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "New Haven",
          "state": "Connecticut"
        },
        {
          "city": "Decatur",
          "state": "Georgia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01074944"
    },
    {
      "nct_id": "NCT05588167",
      "title": "Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Months",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "3 Months and older"
      },
      "enrollment_count": 100,
      "start_date": "2022-11-28",
      "completion_date": "2026-09-30",
      "has_results": false,
      "last_update_posted_date": "2026-06-18",
      "last_synced_at": "2026-06-26T11:31:23.182Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05588167"
    },
    {
      "nct_id": "NCT02612129",
      "title": "Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [
        {
          "name": "arimoclomol",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "ZevraDenmark",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "18 Years",
        "sex": "ALL",
        "summary": "2 Years to 18 Years"
      },
      "enrollment_count": 50,
      "start_date": "2016-06-14",
      "completion_date": "2024-10-31",
      "has_results": true,
      "last_update_posted_date": "2024-11-29",
      "last_synced_at": "2026-06-26T11:31:23.182Z",
      "location_count": 2,
      "location_summary": "Oakland, California • Rochester, Minnesota",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Rochester",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02612129"
    },
    {
      "nct_id": "NCT02037880",
      "title": "Natural History Studies of Mucopolysaccharidosis III",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Mucopolysaccharidosis Type IIIA",
        "Mucopolysaccharidosis Type IIIB"
      ],
      "interventions": [
        {
          "name": "Lumbar puncture",
          "type": "PROCEDURE"
        },
        {
          "name": "Magnetic Resonance Imaging (MRI) of the brain",
          "type": "DEVICE"
        }
      ],
      "intervention_types": [
        "PROCEDURE",
        "DEVICE"
      ],
      "sponsor": "Nationwide Children's Hospital",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "2 Years and older"
      },
      "enrollment_count": 25,
      "start_date": "2014-02",
      "completion_date": "2015-11",
      "has_results": false,
      "last_update_posted_date": "2015-11-16",
      "last_synced_at": "2026-06-26T11:31:23.182Z",
      "location_count": 1,
      "location_summary": "Columbus, Ohio",
      "locations": [
        {
          "city": "Columbus",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02037880"
    },
    {
      "nct_id": "NCT02112994",
      "title": "Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Lysosomal Acid Lipase Deficiency"
      ],
      "interventions": [
        {
          "name": "Sebelipase Alfa",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Alexion Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Months",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "8 Months and older"
      },
      "enrollment_count": 31,
      "start_date": "2014-06-24",
      "completion_date": "2017-12-28",
      "has_results": true,
      "last_update_posted_date": "2019-12-04",
      "last_synced_at": "2026-06-26T11:31:23.182Z",
      "location_count": 3,
      "location_summary": "Chicago, Illinois • Shreveport, Louisiana • Cincinnati, Ohio",
      "locations": [
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Shreveport",
          "state": "Louisiana"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02112994"
    },
    {
      "nct_id": "NCT01609062",
      "title": "Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Mucopolysaccharidosis IVA",
        "Morquio A Syndrome",
        "MPS IVA"
      ],
      "interventions": [
        {
          "name": "BMN 110",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "7 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "7 Years and older"
      },
      "enrollment_count": 25,
      "start_date": "2012-04",
      "completion_date": "2014-11",
      "has_results": true,
      "last_update_posted_date": "2016-02-01",
      "last_synced_at": "2026-06-26T11:31:23.182Z",
      "location_count": 4,
      "location_summary": "Oakland, California • Chicago, Illinois • New York, New York + 1 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01609062"
    },
    {
      "nct_id": "NCT02004691",
      "title": "Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Sphingomyelin Lipidosis"
      ],
      "interventions": [
        {
          "name": "placebo (saline)",
          "type": "DRUG"
        },
        {
          "name": "Olipudase alfa",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 36,
      "start_date": "2015-12-18",
      "completion_date": "2023-10-19",
      "has_results": true,
      "last_update_posted_date": "2024-11-07",
      "last_synced_at": "2026-06-26T11:31:23.182Z",
      "location_count": 3,
      "location_summary": "San Francisco, California • Decatur, Georgia • The Bronx, New York",
      "locations": [
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Decatur",
          "state": "Georgia"
        },
        {
          "city": "The Bronx",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02004691"
    },
    {
      "nct_id": "NCT05186324",
      "title": "Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "Interview",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Chiesi Farmaceutici S.p.A.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 23,
      "start_date": "2022-01-26",
      "completion_date": "2022-08-31",
      "has_results": false,
      "last_update_posted_date": "2023-03-21",
      "last_synced_at": "2026-06-26T11:31:23.182Z",
      "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/NCT05186324"
    },
    {
      "nct_id": "NCT00048620",
      "title": "Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Mucopolysaccharidosis VI"
      ],
      "interventions": [
        {
          "name": "N-acetylgalactosamine 4-sulfatase",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": null,
      "start_date": "2000-09",
      "completion_date": "2005-11",
      "has_results": false,
      "last_update_posted_date": "2006-11-03",
      "last_synced_at": "2026-06-26T11:31:23.182Z",
      "location_count": 1,
      "location_summary": "Novato, California",
      "locations": [
        {
          "city": "Novato",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00048620"
    }
  ]
}