{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Enzyme+Deficiency",
    "query": {
      "condition": "Enzyme Deficiency"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 36,
    "total_pages": 4,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Enzyme+Deficiency&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-05-22T08:55:16.661Z",
  "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": "NCT01881984",
      "title": "Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency"
      ],
      "interventions": [
        {
          "name": "Ravicti",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of Pittsburgh",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 4,
      "start_date": "2013-06",
      "completion_date": "2016-02",
      "has_results": true,
      "last_update_posted_date": "2017-09-25",
      "last_synced_at": "2026-05-22T08:55:16.661Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01881984"
    },
    {
      "nct_id": "NCT00207948",
      "title": "Optimizing Antiretroviral Therapy in HIV-Infected Children and Adolescents",
      "overall_status": "TERMINATED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "HIV Infections"
      ],
      "interventions": [
        {
          "name": "Dose adjustment of Kaletra",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Children's National Research Institute",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "4 Years to 21 Years"
      },
      "enrollment_count": 4,
      "start_date": "2004-11",
      "completion_date": "2009-07",
      "has_results": false,
      "last_update_posted_date": "2015-08-05",
      "last_synced_at": "2026-05-22T08:55:16.661Z",
      "location_count": 1,
      "location_summary": "Washington D.C., District of Columbia",
      "locations": [
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00207948"
    },
    {
      "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": "2026-05-22T08:55:16.661Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02171104"
    },
    {
      "nct_id": "NCT04885179",
      "title": "SPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International)",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Sphingolipidoses",
        "Enzyme Deficiency"
      ],
      "interventions": [
        {
          "name": "no intervention",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "University of California, San Francisco",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 120,
      "start_date": "2025-04-22",
      "completion_date": "2028-12-30",
      "has_results": false,
      "last_update_posted_date": "2025-10-24",
      "last_synced_at": "2026-05-22T08:55:16.661Z",
      "location_count": 1,
      "location_summary": "San Francisco, California",
      "locations": [
        {
          "city": "San Francisco",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04885179"
    },
    {
      "nct_id": "NCT01554800",
      "title": "Effect of ACP-501 on Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Subjects With Coronary Artery Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Coronary Artery Disease",
        "Atherosclerosis"
      ],
      "interventions": [
        {
          "name": "ACP-501",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "MedImmune LLC",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "30 Years",
        "maximum_age": "85 Years",
        "sex": "ALL",
        "summary": "30 Years to 85 Years"
      },
      "enrollment_count": 16,
      "start_date": "2012-03",
      "completion_date": "2012-09",
      "has_results": false,
      "last_update_posted_date": "2019-03-14",
      "last_synced_at": "2026-05-22T08:55:16.661Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01554800"
    },
    {
      "nct_id": "NCT03406611",
      "title": "Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Homocystinuria"
      ],
      "interventions": [
        {
          "name": "Pegtibatinase",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Travere Therapeutics, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "12 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "12 Years to 65 Years"
      },
      "enrollment_count": 32,
      "start_date": "2019-01-22",
      "completion_date": "2026-12",
      "has_results": false,
      "last_update_posted_date": "2024-11-22",
      "last_synced_at": "2026-05-22T08:55:16.661Z",
      "location_count": 8,
      "location_summary": "Culver City, California • Aurora, Colorado • Miami, Florida + 5 more",
      "locations": [
        {
          "city": "Culver City",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Miami",
          "state": "Florida"
        },
        {
          "city": "Indianapolis",
          "state": "Indiana"
        },
        {
          "city": "Portland",
          "state": "Maine"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03406611"
    },
    {
      "nct_id": "NCT01894165",
      "title": "Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Molybdenum Cofactor Deficiency (MoCD)",
        "Rare Autosomal Recessive Disorder",
        "Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)"
      ],
      "interventions": [
        {
          "name": "ALXN1101",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Origin Biosciences",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "60 Years",
        "sex": "ALL",
        "summary": "18 Years to 60 Years"
      },
      "enrollment_count": 24,
      "start_date": "2013-06",
      "completion_date": "2013-09",
      "has_results": false,
      "last_update_posted_date": "2019-03-19",
      "last_synced_at": "2026-05-22T08:55:16.661Z",
      "location_count": 1,
      "location_summary": "Baltimore, Maryland",
      "locations": [
        {
          "city": "Baltimore",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01894165"
    },
    {
      "nct_id": "NCT00103883",
      "title": "A Novel Method to Determine HIV Incidence Among Youth",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "HIV Infection"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of North Carolina, Chapel Hill",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "12 Years",
        "maximum_age": "24 Years",
        "sex": "ALL",
        "summary": "12 Years to 24 Years"
      },
      "enrollment_count": 440,
      "start_date": "2003-02",
      "completion_date": "2007-03",
      "has_results": false,
      "last_update_posted_date": "2017-03-01",
      "last_synced_at": "2026-05-22T08:55:16.661Z",
      "location_count": 14,
      "location_summary": "Los Angeles, California • San Diego, California • San Francisco, California + 11 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Fort Lauderdale",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00103883"
    },
    {
      "nct_id": "NCT02191358",
      "title": "YouScript IMPACT Registry",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Adverse Drug Events",
        "Adverse Drug Reactions",
        "Drug Interaction Potentiation",
        "Drug Metabolism, Poor, CYP2D6-RELATED",
        "Drug Metabolism, Poor, CYP2C19-RELATED",
        "Cytochrome P450 Enzyme Deficiency",
        "Cytochrome P450 CYP2D6 Enzyme Deficiency",
        "Cytochrome P450 CYP2C9 Enzyme Deficiency",
        "Cytochrome P450 CYP2C19 Enzyme Deficiency",
        "Cytochrome P450 CYP3A Enzyme Deficiency",
        "Poor Metabolizer Due to Cytochrome P450 CYP2C9 Variant",
        "Poor Metabolizer Due to Cytochrome P450 CYP2C19 Variant",
        "Poor Metabolizer Due to Cytochrome P450 CYP2D6 Variant"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Genelex Corporation",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "65 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "65 Years and older"
      },
      "enrollment_count": 800,
      "start_date": "2014-10",
      "completion_date": "2015-08",
      "has_results": false,
      "last_update_posted_date": "2017-03-01",
      "last_synced_at": "2026-05-22T08:55:16.661Z",
      "location_count": 7,
      "location_summary": "Garden Grove, California • Denver, Colorado • Lexington, Kentucky + 3 more",
      "locations": [
        {
          "city": "Garden Grove",
          "state": "California"
        },
        {
          "city": "Denver",
          "state": "Colorado"
        },
        {
          "city": "Lexington",
          "state": "Kentucky"
        },
        {
          "city": "Towson",
          "state": "Maryland"
        },
        {
          "city": "Fall River",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02191358"
    },
    {
      "nct_id": "NCT00037076",
      "title": "Prevalence of Hepatitis C Virus Infection in HIV-Infected Children",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "HIV Infections",
        "Hepatitis C"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Institute of Allergy and Infectious Diseases (NIAID)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": "20 Years",
        "sex": "ALL",
        "summary": "1 Year to 20 Years"
      },
      "enrollment_count": 600,
      "start_date": null,
      "completion_date": "2003-07",
      "has_results": false,
      "last_update_posted_date": "2012-10-31",
      "last_synced_at": "2026-05-22T08:55:16.661Z",
      "location_count": 37,
      "location_summary": "Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 29 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "San Diego",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00037076"
    }
  ]
}