{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Mucopolysaccharidosis+Type+1",
    "query": {
      "condition": "Mucopolysaccharidosis Type 1"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 65,
    "total_pages": 7,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Mucopolysaccharidosis+Type+1&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-05-21T22:31:44.972Z",
  "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": "NCT00146770",
      "title": "Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Mucopolysaccharidosis I",
        "Hurler's Syndrome",
        "Hurler-Scheie Syndrome",
        "Scheie Syndrome"
      ],
      "interventions": [
        {
          "name": "Aldurazyme",
          "type": "BIOLOGICAL"
        },
        {
          "name": "placebo",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 45,
      "start_date": "2001-05",
      "completion_date": "2005-03",
      "has_results": true,
      "last_update_posted_date": "2015-04-03",
      "last_synced_at": "2026-05-21T22:31:44.972Z",
      "location_count": 8,
      "location_summary": "Mobile, Alabama • Atlanta, Georgia • Rochester, New York + 5 more",
      "locations": [
        {
          "city": "Mobile",
          "state": "Alabama"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Rochester",
          "state": "New York"
        },
        {
          "city": "Chapel Hill",
          "state": "North Carolina"
        },
        {
          "city": "Toledo",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00146770"
    },
    {
      "nct_id": "NCT05634512",
      "title": "Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Hematopoietic Cell Transplantation",
        "Mucopolysaccharidosis Type I"
      ],
      "interventions": [
        {
          "name": "Laronidase therapy and a stem cell transplant",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Masonic Cancer Center, University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "0 Years",
        "maximum_age": "3 Years",
        "sex": "ALL",
        "summary": "0 Years to 3 Years"
      },
      "enrollment_count": 13,
      "start_date": "2022-11-17",
      "completion_date": "2026-10-01",
      "has_results": false,
      "last_update_posted_date": "2025-10-30",
      "last_synced_at": "2026-05-21T22:31:44.972Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05634512"
    },
    {
      "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": "2026-05-21T22:31:44.972Z",
      "location_count": 1,
      "location_summary": "Los Angeles, California",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00730314"
    },
    {
      "nct_id": "NCT06103487",
      "title": "Long Term Follow-Up for RGX-111",
      "overall_status": "ENROLLING_BY_INVITATION",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Mucopolysaccharidosis I"
      ],
      "interventions": [
        {
          "name": "No Intervention",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "REGENXBIO Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 21,
      "start_date": "2023-07-24",
      "completion_date": "2027-09",
      "has_results": false,
      "last_update_posted_date": "2026-04-29",
      "last_synced_at": "2026-05-21T22:31:44.972Z",
      "location_count": 1,
      "location_summary": "Orange, California",
      "locations": [
        {
          "city": "Orange",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06103487"
    },
    {
      "nct_id": "NCT04528355",
      "title": "Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Primary Immunodeficiency (PID)",
        "Congenital Bone Marrow Failure Syndromes",
        "Inherited Metabolic Disorders (IMD)",
        "Hereditary Anemias",
        "Inflammatory Conditions"
      ],
      "interventions": [
        {
          "name": "data collection",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Paul Szabolcs",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Months",
        "maximum_age": "60 Years",
        "sex": "ALL",
        "summary": "2 Months to 60 Years"
      },
      "enrollment_count": 50,
      "start_date": "2020-08-20",
      "completion_date": "2028-06-30",
      "has_results": false,
      "last_update_posted_date": "2026-01-13",
      "last_synced_at": "2026-05-21T22:31:44.972Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04528355"
    },
    {
      "nct_id": "NCT01173016",
      "title": "Administration of IV Laronidase Post Bone Marrow Transplant in Hurler",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Hurler Syndrome"
      ],
      "interventions": [
        {
          "name": "Laronidase",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Masonic Cancer Center, University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "14 Years",
        "sex": "ALL",
        "summary": "Up to 14 Years"
      },
      "enrollment_count": 11,
      "start_date": "2012-05-29",
      "completion_date": "2016-03-04",
      "has_results": true,
      "last_update_posted_date": "2020-03-20",
      "last_synced_at": "2026-05-21T22:31:44.972Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01173016"
    },
    {
      "nct_id": "NCT06181136",
      "title": "Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Mucopolysaccharidosis Type IIIA"
      ],
      "interventions": [
        {
          "name": "DNL126",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Denali Therapeutics Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "0 Years",
        "maximum_age": "18 Years",
        "sex": "ALL",
        "summary": "0 Years to 18 Years"
      },
      "enrollment_count": 20,
      "start_date": "2023-12-07",
      "completion_date": "2028-08",
      "has_results": false,
      "last_update_posted_date": "2025-12-05",
      "last_synced_at": "2026-05-21T22:31:44.972Z",
      "location_count": 4,
      "location_summary": "Oakland, California • Iowa City, Iowa • Chapel Hill, North Carolina + 1 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Iowa City",
          "state": "Iowa"
        },
        {
          "city": "Chapel Hill",
          "state": "North Carolina"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06181136"
    },
    {
      "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": "2026-05-21T22:31:44.972Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02171104"
    },
    {
      "nct_id": "NCT00912925",
      "title": "Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Mucopolysaccharidosis I",
        "Hurlers Syndrome",
        "Hurler-Scheie Syndrome"
      ],
      "interventions": [
        {
          "name": "rhIDU (recombinant human-Alpha-L-Iduronidase)",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Placebo",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "5 Years and older"
      },
      "enrollment_count": 45,
      "start_date": "2000-12",
      "completion_date": "2001-09",
      "has_results": true,
      "last_update_posted_date": "2015-04-07",
      "last_synced_at": "2026-05-21T22:31:44.972Z",
      "location_count": 2,
      "location_summary": "New York, New York • Chapel Hill, North Carolina",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Chapel Hill",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00912925"
    },
    {
      "nct_id": "NCT04008849",
      "title": "A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456",
      "overall_status": "TERMINATED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Inherited Metabolic Disorders (IMD)"
      ],
      "interventions": [
        {
          "name": "Safety and efficacy assessments",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Magenta Therapeutics, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "16 Years",
        "sex": "ALL",
        "summary": "Up to 16 Years"
      },
      "enrollment_count": 3,
      "start_date": "2019-05-21",
      "completion_date": "2020-10-08",
      "has_results": false,
      "last_update_posted_date": "2021-03-15",
      "last_synced_at": "2026-05-21T22:31:44.972Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04008849"
    }
  ]
}