{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Niemann+Pick+Diseases",
    "query": {
      "condition": "Niemann Pick Diseases"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 49,
    "total_pages": 5,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Niemann+Pick+Diseases&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-07-23T16:44:17.018Z",
  "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": "NCT04469894",
      "title": "Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Niemann-Pick Diseases"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Niemann-Pick Disease Foundation",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 76,
      "start_date": "2020-06-15",
      "completion_date": "2020-09-21",
      "has_results": false,
      "last_update_posted_date": "2020-10-06",
      "last_synced_at": "2026-07-23T16:44:17.018Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04469894"
    },
    {
      "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": "2026-07-23T16:44:17.018Z",
      "location_count": 1,
      "location_summary": "Rochester, Minnesota",
      "locations": [
        {
          "city": "Rochester",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05163288"
    },
    {
      "nct_id": "NCT03643562",
      "title": "Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Niemann-Pick Type C Disease"
      ],
      "interventions": [
        {
          "name": "Adrabetadex",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Mandos LLC",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "4 Years and older"
      },
      "enrollment_count": 27,
      "start_date": "2018-06-18",
      "completion_date": "2021-11-02",
      "has_results": true,
      "last_update_posted_date": "2024-01-17",
      "last_synced_at": "2026-07-23T16:44:17.018Z",
      "location_count": 17,
      "location_summary": "Little Rock, Arkansas • Loma Linda, California • Los Angeles, California + 13 more",
      "locations": [
        {
          "city": "Little Rock",
          "state": "Arkansas"
        },
        {
          "city": "Loma Linda",
          "state": "California"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03643562"
    },
    {
      "nct_id": "NCT04958642",
      "title": "Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [
        {
          "name": "Adrabetadex",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "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": 66,
      "start_date": "2015-12-23",
      "completion_date": "2022-04-11",
      "has_results": true,
      "last_update_posted_date": "2023-08-29",
      "last_synced_at": "2026-07-23T16:44:17.018Z",
      "location_count": 15,
      "location_summary": "Birmingham, Alabama • Orange, California • San Francisco, California + 12 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04958642"
    },
    {
      "nct_id": "NCT04860960",
      "title": "Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1",
      "overall_status": "UNKNOWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C1"
      ],
      "interventions": [
        {
          "name": "Hydroxypropyl-beta-cyclodextrin",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Cyclo Therapeutics, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "3 Years and older"
      },
      "enrollment_count": 94,
      "start_date": "2021-07-20",
      "completion_date": "2026-06",
      "has_results": false,
      "last_update_posted_date": "2024-06-04",
      "last_synced_at": "2026-07-23T16:44:17.018Z",
      "location_count": 7,
      "location_summary": "Oakland, California • Jacksonville, Florida • Atlanta, Georgia + 4 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Jacksonville",
          "state": "Florida"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        },
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04860960"
    },
    {
      "nct_id": "NCT02193867",
      "title": "Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency",
      "overall_status": "TERMINATED",
      "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": null,
        "maximum_age": "8 Months",
        "sex": "ALL",
        "summary": "Up to 8 Months"
      },
      "enrollment_count": 10,
      "start_date": "2014-06-06",
      "completion_date": "2018-10-30",
      "has_results": true,
      "last_update_posted_date": "2019-11-18",
      "last_synced_at": "2026-07-23T16:44:17.018Z",
      "location_count": 1,
      "location_summary": "Phoenix, Arizona",
      "locations": [
        {
          "city": "Phoenix",
          "state": "Arizona"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02193867"
    },
    {
      "nct_id": "NCT04316637",
      "title": "Early Access Program With Arimoclomol in US Patients With NPC",
      "overall_status": "AVAILABLE",
      "study_type": "EXPANDED_ACCESS",
      "phases": [],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [
        {
          "name": "Arimoclomol",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "ZevraDenmark",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "2 Years and older"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2024-08-16",
      "last_synced_at": "2026-07-23T16:44:17.018Z",
      "location_count": 13,
      "location_summary": "Birmingham, Alabama • Oakland, California • Orange, California + 10 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Miami",
          "state": "Florida"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04316637"
    },
    {
      "nct_id": "NCT01722526",
      "title": "Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Human Acid Sphingomyelinase Deficiency"
      ],
      "interventions": [
        {
          "name": "Recombinant human acid sphingomyelinase",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "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": 5,
      "start_date": "2013-03",
      "completion_date": "2014-01",
      "has_results": false,
      "last_update_posted_date": "2015-07-30",
      "last_synced_at": "2026-07-23T16:44:17.018Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01722526"
    },
    {
      "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": "2026-07-23T16:44:17.018Z",
      "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": "NCT00410566",
      "title": "Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Acid Sphingomyelinase Deficiency",
        "Niemann-Pick Disease"
      ],
      "interventions": [
        {
          "name": "rhASM",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "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": 11,
      "start_date": "2006-12",
      "completion_date": "2009-04",
      "has_results": false,
      "last_update_posted_date": "2015-03-19",
      "last_synced_at": "2026-07-23T16:44:17.018Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00410566"
    }
  ]
}