{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Niemann-Pick+Disease&page=3",
    "query": {
      "condition": "Niemann-Pick Disease",
      "page": 3
    },
    "page_size": 10
  },
  "pagination": {
    "page": 3,
    "page_size": 10,
    "total_count": 49,
    "total_pages": 5,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Niemann-Pick+Disease&page=4&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Niemann-Pick+Disease&page=2&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-10T16:49:47.072Z",
  "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": "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": "2026-10-10T16:49:47.072Z",
      "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": "NCT02534844",
      "title": "VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [
        {
          "name": "Parts A/B: Adrabetadex",
          "type": "DRUG"
        },
        {
          "name": "Parts A/B: Sham Control",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Mandos LLC",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "4 Years to 21 Years"
      },
      "enrollment_count": 56,
      "start_date": "2015-10",
      "completion_date": "2018-03-28",
      "has_results": true,
      "last_update_posted_date": "2023-02-22",
      "last_synced_at": "2026-10-10T16:49:47.072Z",
      "location_count": 14,
      "location_summary": "Birmingham, Alabama • Orange, California • San Francisco, California + 11 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Gainesville",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02534844"
    },
    {
      "nct_id": "NCT00668564",
      "title": "Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Hurler's Syndrome",
        "Maroteaux-Lamy Syndrome",
        "Sly Syndrome",
        "Alpha Mannosidosis",
        "Fucosidosis",
        "Aspartylglucosaminuria",
        "Sphingolipidoses",
        "Krabbe Disease",
        "Wolman's Disease",
        "Niemann-Pick Disease Type B",
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [
        {
          "name": "Stem Cell Transplantation",
          "type": "PROCEDURE"
        },
        {
          "name": "Cyclophosphamide",
          "type": "DRUG"
        },
        {
          "name": "Campath-1H",
          "type": "DRUG"
        },
        {
          "name": "Busulfan",
          "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": "21 Years",
        "sex": "ALL",
        "summary": "Up to 21 Years"
      },
      "enrollment_count": 18,
      "start_date": "2008-03",
      "completion_date": "2010-02",
      "has_results": true,
      "last_update_posted_date": "2017-12-28",
      "last_synced_at": "2026-10-10T16:49:47.072Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00668564"
    },
    {
      "nct_id": "NCT03759639",
      "title": "N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [
        {
          "name": "IB1001",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "IntraBio Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "6 Years and older"
      },
      "enrollment_count": 33,
      "start_date": "2019-09-04",
      "completion_date": "2022-11-07",
      "has_results": true,
      "last_update_posted_date": "2023-11-28",
      "last_synced_at": "2026-10-10T16:49:47.072Z",
      "location_count": 1,
      "location_summary": "Rochester, Minnesota",
      "locations": [
        {
          "city": "Rochester",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03759639"
    },
    {
      "nct_id": "NCT01747135",
      "title": "Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C1"
      ],
      "interventions": [
        {
          "name": "VTS-270",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "25 Years",
        "sex": "ALL",
        "summary": "2 Years to 25 Years"
      },
      "enrollment_count": 14,
      "start_date": "2013-01",
      "completion_date": "2017-03",
      "has_results": false,
      "last_update_posted_date": "2021-07-23",
      "last_synced_at": "2026-10-10T16:49:47.072Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01747135"
    },
    {
      "nct_id": "NCT05163288",
      "title": "A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [
        {
          "name": "N-Acetyl-L-Leucine",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "IntraBio Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "4 Years and older"
      },
      "enrollment_count": 53,
      "start_date": "2022-06-30",
      "completion_date": "2030-12",
      "has_results": false,
      "last_update_posted_date": "2026-04-03",
      "last_synced_at": "2026-10-10T16:49:47.072Z",
      "location_count": 1,
      "location_summary": "Rochester, Minnesota",
      "locations": [
        {
          "city": "Rochester",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05163288"
    },
    {
      "nct_id": "NCT02000310",
      "title": "Molecular and Cellular Mechanisms of Lysosomal Storage Diseases",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Lysosomal Storage Disorders"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "O & O Alpan LLC",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "1 Day",
        "maximum_age": "100 Years",
        "sex": "ALL",
        "summary": "1 Day to 100 Years"
      },
      "enrollment_count": 80,
      "start_date": "2013-11",
      "completion_date": "2022-12",
      "has_results": false,
      "last_update_posted_date": "2021-02-23",
      "last_synced_at": "2026-10-10T16:49:47.072Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02000310"
    },
    {
      "nct_id": "NCT06192576",
      "title": "A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Niemann-Pick Diseases",
        "Acid Sphingomyelinase Deficiency"
      ],
      "interventions": [
        {
          "name": "Olipudase alfa",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Sanofi",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "2 Years",
        "sex": "ALL",
        "summary": "Up to 2 Years"
      },
      "enrollment_count": 10,
      "start_date": "2024-04-16",
      "completion_date": "2029-01-15",
      "has_results": false,
      "last_update_posted_date": "2026-09-16",
      "last_synced_at": "2026-10-10T16:49:47.072Z",
      "location_count": 6,
      "location_summary": "Chicago, Illinois • Cincinnati, Ohio • Columbus, Ohio + 3 more",
      "locations": [
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        },
        {
          "city": "Columbus",
          "state": "Ohio"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        },
        {
          "city": "Austin",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06192576"
    },
    {
      "nct_id": "NCT03887533",
      "title": "Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C1"
      ],
      "interventions": [
        {
          "name": "VTS-270",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": "60 Years",
        "sex": "ALL",
        "summary": "3 Years to 60 Years"
      },
      "enrollment_count": 2,
      "start_date": "2020-01-06",
      "completion_date": "2021-10-25",
      "has_results": true,
      "last_update_posted_date": "2022-08-30",
      "last_synced_at": "2026-10-10T16:49:47.072Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03887533"
    },
    {
      "nct_id": "NCT03471143",
      "title": "Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [
        {
          "name": "VTS-270",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Washington University School of Medicine",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "6 Months",
        "sex": "ALL",
        "summary": "Up to 6 Months"
      },
      "enrollment_count": 4,
      "start_date": "2019-02-22",
      "completion_date": "2024-10-04",
      "has_results": true,
      "last_update_posted_date": "2026-01-05",
      "last_synced_at": "2026-10-10T16:49:47.072Z",
      "location_count": 1,
      "location_summary": "St Louis, Missouri",
      "locations": [
        {
          "city": "St Louis",
          "state": "Missouri"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03471143"
    }
  ]
}