{
  "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": "2026-05-21T23:41:06.856Z",
  "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": "NCT00001972",
      "title": "PET Scan of Brain Metabolism in Relation to Age and Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Alzheimer's Disease",
        "Brain Neoplasm",
        "Niemann Pick Disease"
      ],
      "interventions": [
        {
          "name": "15 O Water",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)",
      "sponsor_class": "NIH",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 123,
      "start_date": "1994-09",
      "completion_date": "2002-08",
      "has_results": false,
      "last_update_posted_date": "2008-03-04",
      "last_synced_at": "2026-05-21T23:41:06.856Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00001972"
    },
    {
      "nct_id": "NCT00730314",
      "title": "Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Sickle Cell Disease",
        "Thalassemia",
        "Anemia",
        "Granuloma",
        "Wiskott-Aldrich Syndrome",
        "Chediak Higashi Syndrome",
        "Osteopetrosis",
        "Neutropenia",
        "Thrombocytopenia",
        "Hurler Disease",
        "Niemann-Pick Disease",
        "Fucosidosis"
      ],
      "interventions": [
        {
          "name": "Hematopoietic stem cell transplantation",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "PROCEDURE"
      ],
      "sponsor": "Children's Hospital Los Angeles",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "Up to 21 Years"
      },
      "enrollment_count": 25,
      "start_date": "2008-08",
      "completion_date": "2015-08",
      "has_results": false,
      "last_update_posted_date": "2016-06-23",
      "last_synced_at": "2026-05-21T23:41:06.856Z",
      "location_count": 1,
      "location_summary": "Los Angeles, California",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00730314"
    },
    {
      "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": "2026-05-21T23:41:06.856Z",
      "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": "2026-05-21T23:41:06.856Z",
      "location_count": 1,
      "location_summary": "St Louis, Missouri",
      "locations": [
        {
          "city": "St Louis",
          "state": "Missouri"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03471143"
    },
    {
      "nct_id": "NCT07054515",
      "title": "A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Niemann-Pick Type C Disease",
        "GM1 Gangliosidosis",
        "GM2 Gangliosidosis"
      ],
      "interventions": [
        {
          "name": "AZ-3102",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Azafaros A.G.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "4 Years and older"
      },
      "enrollment_count": 147,
      "start_date": "2025-06-30",
      "completion_date": "2027-11-04",
      "has_results": false,
      "last_update_posted_date": "2026-05-05",
      "last_synced_at": "2026-05-21T23:41:06.856Z",
      "location_count": 5,
      "location_summary": "Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "Rochester",
          "state": "Minnesota"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07054515"
    },
    {
      "nct_id": "NCT03333200",
      "title": "Longitudinal Study of Neurodegenerative Disorders",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "MLD",
        "Krabbe Disease",
        "ALD",
        "MPS I",
        "MPS II",
        "MPS III",
        "Vanishing White Matter Disease",
        "GM3 Gangliosidosis",
        "PKAN",
        "Tay-Sachs Disease",
        "NP Deficiency",
        "Osteopetrosis",
        "Alpha-Mannosidosis",
        "Sandhoff Disease",
        "Niemann-Pick Diseases",
        "MPS IV",
        "Gaucher Disease",
        "GAN",
        "GM1 Gangliosidoses",
        "Morquio Disease",
        "S-Adenosylhomocysteine Hydrolase Deficiency",
        "Batten Disease",
        "Pelizaeus-Merzbacher Disease",
        "Leukodystrophy",
        "Lysosomal Storage Diseases",
        "Purine Nucleoside Phosphorylase Deficiency",
        "Multiple Sulfatase Deficiency Disease"
      ],
      "interventions": [
        {
          "name": "Palliative Care",
          "type": "OTHER"
        },
        {
          "name": "Hematopoetic Stem Cell Transplantation",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "OTHER",
        "BIOLOGICAL"
      ],
      "sponsor": "University of Pittsburgh",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 1500,
      "start_date": "2012-01-11",
      "completion_date": "2035-01",
      "has_results": false,
      "last_update_posted_date": "2026-02-09",
      "last_synced_at": "2026-05-21T23:41:06.856Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03333200"
    },
    {
      "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": "2026-05-21T23:41:06.856Z",
      "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": "NCT07082725",
      "title": "A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Niemann-Pick Type C Disease"
      ],
      "interventions": [
        {
          "name": "Nizubaglustat",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Azafaros A.G.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "4 Years and older"
      },
      "enrollment_count": 72,
      "start_date": "2025-06-30",
      "completion_date": "2027-11-04",
      "has_results": false,
      "last_update_posted_date": "2026-05-05",
      "last_synced_at": "2026-05-21T23:41:06.856Z",
      "location_count": 5,
      "location_summary": "Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "Rochester",
          "state": "Minnesota"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07082725"
    },
    {
      "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": "2026-05-21T23:41:06.856Z",
      "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": "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": "2026-05-21T23:41:06.856Z",
      "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"
    }
  ]
}