{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Sphingomyelin+Lipidosis",
    "query": {
      "condition": "Sphingomyelin Lipidosis"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 48,
    "total_pages": 5,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Sphingomyelin+Lipidosis&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-07-22T05:32:54.460Z",
  "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": "NCT00654433",
      "title": "ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Inherited Metabolic Diseases",
        "Lysosomal Storage Disorders",
        "Peroxisomal Storage Diseases",
        "Inborn Errors of Metabolism",
        "Mucopolysaccharidosis"
      ],
      "interventions": [
        {
          "name": "ALD-101",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Aldagen",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "16 Years",
        "sex": "ALL",
        "summary": "Up to 16 Years"
      },
      "enrollment_count": 40,
      "start_date": "2008-03",
      "completion_date": "2011-11",
      "has_results": false,
      "last_update_posted_date": "2014-07-08",
      "last_synced_at": "2026-07-22T05:32:54.460Z",
      "location_count": 3,
      "location_summary": "Los Angeles, California • New York, New York • Durham, North Carolina",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00654433"
    },
    {
      "nct_id": "NCT02939547",
      "title": "Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C1"
      ],
      "interventions": [
        {
          "name": "Hydroxypropyl-beta-cyclodextrin",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Cyclo Therapeutics, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 13,
      "start_date": "2017-10-11",
      "completion_date": "2020-02-10",
      "has_results": false,
      "last_update_posted_date": "2021-02-21",
      "last_synced_at": "2026-07-22T05:32:54.460Z",
      "location_count": 2,
      "location_summary": "Oakland, California • Morristown, New Jersey",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Morristown",
          "state": "New Jersey"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02939547"
    },
    {
      "nct_id": "NCT01899950",
      "title": "Longitudinal Study of Cognition With Niemann-Pick Disease, Type C",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Mayo Clinic",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "99 Years",
        "sex": "ALL",
        "summary": "2 Years to 99 Years"
      },
      "enrollment_count": 70,
      "start_date": "2011-03",
      "completion_date": "2014-09",
      "has_results": false,
      "last_update_posted_date": "2016-01-06",
      "last_synced_at": "2026-07-22T05:32:54.460Z",
      "location_count": 1,
      "location_summary": "Rochester, Minnesota",
      "locations": [
        {
          "city": "Rochester",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01899950"
    },
    {
      "nct_id": "NCT01586455",
      "title": "Human Placental-Derived Stem Cell Transplantation",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Mucopolysaccharidosis I",
        "Mucopolysaccharidosis VI",
        "Adrenoleukodystrophy",
        "Niemann-Pick Disease",
        "Metachromatic Leukodystrophy",
        "Wolman Disease",
        "Krabbe's Disease",
        "Gaucher's Disease",
        "Fucosidosis",
        "Batten Disease",
        "Severe Aplastic Anemia",
        "Diamond-Blackfan Anemia",
        "Amegakaryocytic Thrombocytopenia",
        "Myelodysplastic Syndrome",
        "Acute Myelogenous Leukemia",
        "Acute Lymphocytic Leukemia"
      ],
      "interventions": [
        {
          "name": "Human Placental Derived Stem Cell",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "New York Medical College",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "0 Years",
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "0 Years to 55 Years"
      },
      "enrollment_count": 43,
      "start_date": "2013-04",
      "completion_date": "2022-09",
      "has_results": false,
      "last_update_posted_date": "2022-10-25",
      "last_synced_at": "2026-07-22T05:32:54.460Z",
      "location_count": 3,
      "location_summary": "Denver, Colorado • Valhalla, New York • Salt Lake City, Utah",
      "locations": [
        {
          "city": "Denver",
          "state": "Colorado"
        },
        {
          "city": "Valhalla",
          "state": "New York"
        },
        {
          "city": "Salt Lake City",
          "state": "Utah"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01586455"
    },
    {
      "nct_id": "NCT02171104",
      "title": "MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Mucopolysaccharidosis Disorders",
        "Hurler Syndrome",
        "Hunter Syndrome",
        "Maroteaux Lamy Syndrome",
        "Sly Syndrome",
        "Alpha-Mannosidosis",
        "Fucosidosis",
        "Aspartylglucosaminuria",
        "Glycoprotein Metabolic Disorders",
        "Sphingolipidoses",
        "Recessive Leukodystrophies",
        "Globoid Cell Leukodystrophy",
        "Metachromatic Leukodystrophy",
        "Niemann-Pick B",
        "Niemann-Pick C Subtype 2",
        "Sphingomyelin Deficiency",
        "Peroxisomal Disorders",
        "Adrenoleukodystrophy With Cerebral Involvement",
        "Zellweger Syndrome",
        "Neonatal Adrenoleukodystrophy",
        "Infantile Refsum Disease",
        "Acyl-CoA Oxidase Deficiency",
        "D-Bifunctional Enzyme Deficiency",
        "Multifunctional Enzyme Deficiency",
        "Alpha-methylacyl-CoA Racmase Deficiency",
        "Mitochondrial Neurogastrointestingal Encephalopathy",
        "Severe Osteopetrosis",
        "Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)",
        "Inherited Metabolic Disorders"
      ],
      "interventions": [
        {
          "name": "Stem Cell Transplantation",
          "type": "BIOLOGICAL"
        },
        {
          "name": "IMD Preparative Regimen",
          "type": "DRUG"
        },
        {
          "name": "Osteopetrosis Only Preparative Regimen",
          "type": "DRUG"
        },
        {
          "name": "Osteopetrosis Haploidentical Only Preparative Regimen",
          "type": "DRUG"
        },
        {
          "name": "cALD SR-A (Standard-Risk, Regimen A)",
          "type": "DRUG"
        },
        {
          "name": "cALD SR-B (Standard-Risk, Regimen B)",
          "type": "DRUG"
        },
        {
          "name": "cALD HR-D (High-Risk, Regimen C)",
          "type": "DRUG"
        },
        {
          "name": "cALD HR-D (High-Risk, Regimen D)",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "DRUG"
      ],
      "sponsor": "Masonic Cancer Center, University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "Up to 55 Years"
      },
      "enrollment_count": 149,
      "start_date": "2014-07-10",
      "completion_date": "2029-07-14",
      "has_results": false,
      "last_update_posted_date": "2026-01-07",
      "last_synced_at": "2026-07-22T05:32:54.460Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02171104"
    },
    {
      "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": "2026-07-22T05:32:54.460Z",
      "location_count": 1,
      "location_summary": "St Louis, Missouri",
      "locations": [
        {
          "city": "St Louis",
          "state": "Missouri"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03471143"
    },
    {
      "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": "2026-07-22T05:32:54.460Z",
      "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": "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": "2026-07-22T05:32:54.460Z",
      "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": "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": "2026-07-22T05:32:54.460Z",
      "location_count": 1,
      "location_summary": "Rochester, Minnesota",
      "locations": [
        {
          "city": "Rochester",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05163288"
    },
    {
      "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": "2026-07-22T05:32:54.460Z",
      "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"
    }
  ]
}