{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=X-Linked+Severe+Combined+Immunodeficiency&page=2",
    "query": {
      "condition": "X-Linked Severe Combined Immunodeficiency",
      "page": 2
    },
    "page_size": 10
  },
  "pagination": {
    "page": 2,
    "page_size": 10,
    "total_count": 10,
    "total_pages": 1,
    "next_page_url": null,
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=X-Linked+Severe+Combined+Immunodeficiency&page=1&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-06-10T13:42:38.865Z",
  "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": "NCT02231710",
      "title": "Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Primary Immune Deficiency Disorders",
        "Hemophagocytic Lymphohistiocytosis",
        "Inherited Bone Marrow Failure Syndrome",
        "Hemoglobinopathies",
        "Metabolic Disorders"
      ],
      "interventions": [
        {
          "name": "BPX-501 and Rimiducid",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Bellicum Pharmaceuticals",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Months",
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "4 Months to 55 Years"
      },
      "enrollment_count": 1,
      "start_date": "2015-02",
      "completion_date": "2018-01",
      "has_results": true,
      "last_update_posted_date": "2024-03-25",
      "last_synced_at": "2026-06-10T13:42:38.865Z",
      "location_count": 1,
      "location_summary": "Seattle, Washington",
      "locations": [
        {
          "city": "Seattle",
          "state": "Washington"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02231710"
    },
    {
      "nct_id": "NCT00744692",
      "title": "Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Non Malignant Disorders",
        "Immunodeficiencies",
        "Congenital Marrow Failures",
        "Hemoglobinopathies",
        "Inborn Errors of Metabolism",
        "Sickle Cell",
        "Thalassemia",
        "Lysosomal Storage Disease"
      ],
      "interventions": [
        {
          "name": "Unrelated Umbilical Cord Blood Transplant",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Reduced Intensity Conditioning",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "DRUG"
      ],
      "sponsor": "Duke University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "Up to 21 Years"
      },
      "enrollment_count": 22,
      "start_date": "2008-10",
      "completion_date": "2014-04",
      "has_results": true,
      "last_update_posted_date": "2014-08-13",
      "last_synced_at": "2026-06-10T13:42:38.865Z",
      "location_count": 1,
      "location_summary": "Durham, North Carolina",
      "locations": [
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00744692"
    },
    {
      "nct_id": "NCT01529827",
      "title": "Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Accelerated Phase Chronic Myelogenous Leukemia",
        "Adult Acute Lymphoblastic Leukemia in Remission",
        "Adult Acute Myeloid Leukemia in Remission",
        "Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities",
        "Adult Acute Myeloid Leukemia With Del(5q)",
        "Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)",
        "Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)",
        "Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)",
        "Adult Acute Myeloid Leukemia With t(8;21)(q22;q22)",
        "Adult Grade III Lymphomatoid Granulomatosis",
        "Adult Nasal Type Extranodal NK/T-cell Lymphoma",
        "Anaplastic Large Cell Lymphoma",
        "Angioimmunoblastic T-cell Lymphoma",
        "Aplastic Anemia",
        "Burkitt Lymphoma",
        "Childhood Acute Lymphoblastic Leukemia in Remission",
        "Childhood Acute Myeloid Leukemia in Remission",
        "Childhood Chronic Myelogenous Leukemia",
        "Childhood Diffuse Large Cell Lymphoma",
        "Childhood Grade III Lymphomatoid Granulomatosis",
        "Childhood Immunoblastic Large Cell Lymphoma",
        "Childhood Myelodysplastic Syndromes",
        "Childhood Nasal Type Extranodal NK/T-cell Lymphoma",
        "Chronic Myelomonocytic Leukemia",
        "Chronic Phase Chronic Myelogenous Leukemia",
        "Congenital Amegakaryocytic Thrombocytopenia",
        "Diamond-Blackfan Anemia",
        "Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue",
        "Hepatosplenic T-cell Lymphoma",
        "Juvenile Myelomonocytic Leukemia",
        "Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable",
        "Nodal Marginal Zone B-cell Lymphoma",
        "Paroxysmal Nocturnal Hemoglobinuria",
        "Peripheral T-cell Lymphoma",
        "Polycythemia Vera",
        "Post-transplant Lymphoproliferative Disorder",
        "Previously Treated Myelodysplastic Syndromes",
        "Primary Myelofibrosis",
        "Recurrent Adult Acute Lymphoblastic Leukemia",
        "Recurrent Adult Acute Myeloid Leukemia",
        "Recurrent Adult Burkitt Lymphoma",
        "Recurrent Adult Diffuse Large Cell Lymphoma",
        "Recurrent Adult Diffuse Mixed Cell Lymphoma",
        "Recurrent Adult Diffuse Small Cleaved Cell Lymphoma",
        "Recurrent Adult Grade III Lymphomatoid Granulomatosis",
        "Recurrent Adult Hodgkin Lymphoma",
        "Recurrent Adult Immunoblastic Large Cell Lymphoma",
        "Recurrent Adult Lymphoblastic Lymphoma",
        "Recurrent Adult T-cell Leukemia/Lymphoma",
        "Recurrent Childhood Acute Lymphoblastic Leukemia",
        "Recurrent Childhood Acute Myeloid Leukemia",
        "Recurrent Childhood Anaplastic Large Cell Lymphoma",
        "Recurrent Childhood Grade III Lymphomatoid Granulomatosis",
        "Recurrent Childhood Large Cell Lymphoma",
        "Recurrent Childhood Lymphoblastic Lymphoma",
        "Recurrent Childhood Small Noncleaved Cell Lymphoma",
        "Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma",
        "Recurrent Grade 1 Follicular Lymphoma",
        "Recurrent Grade 2 Follicular Lymphoma",
        "Recurrent Grade 3 Follicular Lymphoma",
        "Recurrent Mantle Cell Lymphoma",
        "Recurrent Marginal Zone Lymphoma",
        "Recurrent Mycosis Fungoides/Sezary Syndrome",
        "Recurrent Small Lymphocytic Lymphoma",
        "Recurrent/Refractory Childhood Hodgkin Lymphoma",
        "Refractory Chronic Lymphocytic Leukemia",
        "Refractory Hairy Cell Leukemia",
        "Refractory Multiple Myeloma",
        "Secondary Acute Myeloid Leukemia",
        "Secondary Myelodysplastic Syndromes",
        "Secondary Myelofibrosis",
        "Severe Combined Immunodeficiency",
        "Severe Congenital Neutropenia",
        "Shwachman-Diamond Syndrome",
        "Splenic Marginal Zone Lymphoma",
        "T-cell Large Granular Lymphocyte Leukemia",
        "Waldenstrom Macroglobulinemia",
        "Wiskott-Aldrich Syndrome"
      ],
      "interventions": [
        {
          "name": "fludarabine phosphate",
          "type": "DRUG"
        },
        {
          "name": "melphalan",
          "type": "DRUG"
        },
        {
          "name": "total-body irradiation",
          "type": "RADIATION"
        },
        {
          "name": "tacrolimus",
          "type": "DRUG"
        },
        {
          "name": "mycophenolate mofetil",
          "type": "DRUG"
        },
        {
          "name": "methotrexate",
          "type": "DRUG"
        },
        {
          "name": "laboratory biomarker analysis",
          "type": "OTHER"
        },
        {
          "name": "allogeneic hematopoietic stem cell transplantation",
          "type": "PROCEDURE"
        },
        {
          "name": "peripheral blood stem cell transplantation",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "DRUG",
        "RADIATION",
        "OTHER",
        "PROCEDURE"
      ],
      "sponsor": "Roswell Park Cancer Institute",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": "75 Years",
        "sex": "ALL",
        "summary": "3 Years to 75 Years"
      },
      "enrollment_count": 94,
      "start_date": "2012-02-28",
      "completion_date": "2019-08-29",
      "has_results": true,
      "last_update_posted_date": "2019-09-24",
      "last_synced_at": "2026-06-10T13:42:38.865Z",
      "location_count": 1,
      "location_summary": "Buffalo, New York",
      "locations": [
        {
          "city": "Buffalo",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01529827"
    },
    {
      "nct_id": "NCT00579137",
      "title": "Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Severe Combined Immunodeficiency Disease",
        "Severe Primary Immunodeficiency Disorder",
        "Undefined T Cell Deficiency Disorder",
        "Wiskott-Aldrick Syndrome"
      ],
      "interventions": [
        {
          "name": "Campath -1H",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Fludarabine",
          "type": "DRUG"
        },
        {
          "name": "Anti-CD45",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Stem cell infusion",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "DRUG",
        "PROCEDURE"
      ],
      "sponsor": "Baylor College of Medicine",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 3,
      "start_date": "2007-10",
      "completion_date": "2009-10",
      "has_results": true,
      "last_update_posted_date": "2013-07-02",
      "last_synced_at": "2026-06-10T13:42:38.865Z",
      "location_count": 1,
      "location_summary": "Houston, Texas",
      "locations": [
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00579137"
    },
    {
      "nct_id": "NCT01962415",
      "title": "Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Primary Immunodeficiency (PID)",
        "Congenital Bone Marrow Failure Syndromes",
        "Inherited Metabolic Disorders (IMD)",
        "Hereditary Anemias",
        "Inflammatory Conditions",
        "Systemic Juvenile Idiopathic Arthritis (sJIA)",
        "Juvenile Rheumatoid Arthritis (JRA)"
      ],
      "interventions": [
        {
          "name": "Hydroxyurea",
          "type": "DRUG"
        },
        {
          "name": "Alemtuzumab",
          "type": "DRUG"
        },
        {
          "name": "Fludarabine",
          "type": "DRUG"
        },
        {
          "name": "Melphalan",
          "type": "DRUG"
        },
        {
          "name": "Thiotepa",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Paul Szabolcs",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Months",
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "2 Months to 55 Years"
      },
      "enrollment_count": 100,
      "start_date": "2014-02-04",
      "completion_date": "2027-11",
      "has_results": false,
      "last_update_posted_date": "2025-12-15",
      "last_synced_at": "2026-06-10T13:42:38.865Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01962415"
    },
    {
      "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": "2026-06-10T13:42:38.865Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04528355"
    },
    {
      "nct_id": "NCT01852370",
      "title": "Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases",
      "overall_status": "ENROLLING_BY_INVITATION",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Severe Combined Immunodeficiency (SCID)",
        "Immunodeficiency With Predominant T-cell Defect, Unspecified",
        "Severe Chronic Neutropenia",
        "Chronic Granulomatous Disease (CGD)",
        "Hyper IgE Syndromes",
        "Hyper IgM Deficiencies",
        "Wiskott-Aldrich Syndrome",
        "Mendelian Susceptibility to Mycobacterial Disease",
        "Common Variable Immune Deficiency (CVID)"
      ],
      "interventions": [
        {
          "name": "CD3/CD19 negative allogeneic hematopoietic stem cells",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Paul Szabolcs",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": "45 Years",
        "sex": "ALL",
        "summary": "5 Years to 45 Years"
      },
      "enrollment_count": 16,
      "start_date": "2013-06-20",
      "completion_date": "2027-11",
      "has_results": false,
      "last_update_posted_date": "2025-12-15",
      "last_synced_at": "2026-06-10T13:42:38.865Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01852370"
    },
    {
      "nct_id": "NCT01512888",
      "title": "Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants",
      "overall_status": "SUSPENDED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Severe Combined Immunodeficiency Disease, X-linked"
      ],
      "interventions": [
        {
          "name": "CL20-i4-EF1α-hγc-OPT",
          "type": "GENETIC"
        },
        {
          "name": "Busulfan",
          "type": "DRUG"
        },
        {
          "name": "CliniMacs",
          "type": "DEVICE"
        }
      ],
      "intervention_types": [
        "GENETIC",
        "DRUG",
        "DEVICE"
      ],
      "sponsor": "St. Jude Children's Research Hospital",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "24 Months",
        "sex": "MALE",
        "summary": "Up to 24 Months · Male only"
      },
      "enrollment_count": 28,
      "start_date": "2016-08-17",
      "completion_date": "2034-08",
      "has_results": false,
      "last_update_posted_date": "2025-11-10",
      "last_synced_at": "2026-06-10T13:42:38.865Z",
      "location_count": 3,
      "location_summary": "San Francisco, California • Memphis, Tennessee • Seattle, Washington",
      "locations": [
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Memphis",
          "state": "Tennessee"
        },
        {
          "city": "Seattle",
          "state": "Washington"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01512888"
    },
    {
      "nct_id": "NCT03655223",
      "title": "Early Check: Expanded Screening in Newborns",
      "overall_status": "ENROLLING_BY_INVITATION",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Spinal Muscular Atrophy",
        "Fragile X Syndrome",
        "Fragile X - Premutation",
        "Duchenne Muscular Dystrophy",
        "Hyperinsulinemic Hypoglycemia, Familial 1",
        "Diabetes Mellitus",
        "Adrenoleukodystrophy, Neonatal",
        "Medium-chain Acyl-CoA Dehydrogenase Deficiency",
        "Very Long Chain Acyl Coa Dehydrogenase Deficiency",
        "Beta-ketothiolase Deficiency",
        "Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency",
        "Primary Hyperoxaluria Type 1",
        "Congenital Bile Acid Synthesis Defect Type 2",
        "Pyridoxine-Dependent Epilepsy",
        "Hereditary Fructose Intolerance",
        "Hypophosphatasia",
        "Hyperargininemia",
        "Mucopolysaccharidosis Type 6",
        "Argininosuccinic Aciduria",
        "Citrullinemia, Type I",
        "Wilson Disease",
        "Maple Syrup Urine Disease, Type 1A",
        "Maple Syrup Urine Disease, Type 1B",
        "Biotinidase Deficiency",
        "Neonatal Severe Primary Hyperparathyroidism",
        "Intrinsic Factor Deficiency",
        "Usher Syndrome Type 1D/F Digenic (Diagnosis)",
        "Cystic Fibrosis",
        "Stickler Syndrome Type 2",
        "Stickler Syndrome Type 1",
        "Alport Syndrome, Autosomal Recessive",
        "Alport Syndrome, X-Linked",
        "Carbamoyl Phosphate Synthetase I Deficiency Disease",
        "Carnitine Palmitoyl Transferase 1A Deficiency",
        "Carnitine Palmitoyltransferase II Deficiency",
        "Cystinosis",
        "Chronic Granulomatous Disease",
        "Cerebrotendinous Xanthomatoses",
        "Maple Syrup Urine Disease, Type 2",
        "Severe Combined Immunodeficiency Due to DCLRE1C Deficiency",
        "Thyroid Dyshormonogenesis 6",
        "Thyroid Dyshormonogenesis 5",
        "Supravalvar Aortic Stenosis",
        "Factor X Deficiency",
        "Hemophilia A",
        "Hemophilia B",
        "Tyrosinemia, Type I",
        "Fructose 1,6 Bisphosphatase Deficiency",
        "Glycogen Storage Disease Type I",
        "G6PD Deficiency",
        "Glycogen Storage Disease II",
        "Galactokinase Deficiency",
        "Mucopolysaccharidosis Type IV A",
        "Galactosemias",
        "Guanidinoacetate Methyltransferase Deficiency",
        "Agat Deficiency",
        "Glutaryl-CoA Dehydrogenase Deficiency",
        "Gtp Cyclohydrolase I Deficiency",
        "Hyperinsulinism-Hyperammonemia Syndrome",
        "Primary Hyperoxaluria Type 2",
        "3-Hydroxyacyl-CoA Dehydrogenase Deficiency",
        "Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency",
        "Mitochondrial Trifunctional Protein Deficiency",
        "Sickle Cell Disease",
        "Beta-Thalassemia",
        "Holocarboxylase Synthetase Deficiency",
        "3-Hydroxy-3-Methylglutaric Aciduria",
        "Primary Hyperoxaluria Type 3",
        "Hermansky-Pudlak Syndrome 1",
        "Hermansky-Pudlak Syndrome 4",
        "Apparent Mineralocorticoid Excess",
        "HSDB",
        "CBAS1",
        "Mucopolysaccharidosis Type 2",
        "Mucopolysaccharidosis Type 1",
        "Severe Combined Immunodeficiency, X Linked",
        "Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency",
        "Diabetes Mellitus, Permanent Neonatal",
        "Isovaleric Acidemia",
        "Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder)",
        "Jervell and Lange-Nielsen Syndrome 2",
        "Hyperinsulinemic Hypoglycemia, Familial, 2",
        "Diabetes Mellitus, Permanent Neonatal, With Neurologic Features",
        "Jervell and Lange-Nielsen Syndrome 1",
        "Lysosomal Acid Lipase Deficiency",
        "CblF",
        "3-Methylcrotonyl CoA Carboxylase 1 Deficiency",
        "3-Methylcrotonyl CoA Carboxylase 2 Deficiency",
        "Waardenburg Syndrome Type 2A",
        "Methylmalonic Aciduria cblA Type",
        "Methylmalonic Aciduria cblB Type",
        "Methylmalonic Aciduria and Homocystinuria Type cblC",
        "MAHCD",
        "Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency",
        "Congenital Disorder of Glycosylation Type 1B",
        "Mthfr Deficiency",
        "Methylcobalamin Deficiency Type Cbl G (Disorder)",
        "Methylcobalamin Deficiency Type cblE",
        "Usher Syndrome, Type 1B",
        "N-acetylglutamate Synthase Deficiency",
        "Ornithine Transcarbamylase Deficiency",
        "Phenylketonurias",
        "Waardenburg Syndrome Type 1",
        "Congenital Hypothyroidism",
        "Propionic Acidemia",
        "Usher Syndrome, Type 1F",
        "Pancreatic Agenesis 1",
        "Hereditary Hypophosphatemic Rickets",
        "Glycogen Storage Disease IXB",
        "Glycogen Storage Disease IXC",
        "MOWS",
        "Epilepsy, Early-Onset, Vitamin B6-Dependent",
        "Pyridoxal Phosphate-Responsive Seizures",
        "Pituitary Hormone Deficiency, Combined, 1",
        "Ptsd",
        "Dihydropteridine Reductase Deficiency",
        "Severe Combined Immunodeficiency Due to RAG1 Deficiency",
        "Severe Combined Immunodeficiency Due to RAG2 Deficiency",
        "Retinoblastoma",
        "Multiple Endocrine Neoplasia Type 2B",
        "Pseudohypoaldosteronism, Type I",
        "Liddle Syndrome",
        "Biotin-Responsive Basal Ganglia Disease",
        "SCD",
        "DIAR1",
        "GSD1C",
        "Acrodermatitis Enteropathica",
        "Thyroid Dyshormonogenesis 1",
        "Riboflavin Transporter Deficiency",
        "Waardenburg Syndrome, Type 2E",
        "SRD",
        "Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency",
        "Barth Syndrome",
        "Adrenocorticotropic Hormone Deficiency",
        "Transcobalamin II Deficiency",
        "Thyroid Dyshormonogenesis 3",
        "Segawa Syndrome, Autosomal Recessive",
        "Autosomal Recessive Nonsyndromic Hearing Loss",
        "Thyroid Dyshormonogenesis 2A",
        "Congenital Isolated Thyroid Stimulating Hormone Deficiency",
        "Hypothyroidism Due to TSH Receptor Mutations",
        "Usher Syndrome Type 1C",
        "Usher Syndrome Type 1G (Diagnosis)",
        "Von Willebrand Disease, Type 3",
        "Combined Immunodeficiency Due to ZAP70 Deficiency",
        "Adenine Phosphoribosyltransferase Deficiency",
        "Metachromatic Leukodystrophy",
        "Canavan Disease",
        "Menkes Disease",
        "Carbonic Anhydrase VA Deficiency",
        "Developmental and Epileptic Encephalopathy 2",
        "17 Alpha-Hydroxylase Deficiency",
        "Smith-Lemli-Opitz Syndrome",
        "Krabbe Disease",
        "Glutathione Synthetase Deficiency",
        "Mucopolysaccharidosis Type 7",
        "Rett Syndrome",
        "Molybdenum Cofactor Deficiency, Type A",
        "Niemann-Pick Disease, Type C1",
        "Niemann-Pick Disease Type C2",
        "Ornithine Aminotransferase Deficiency",
        "3-Phosphoglycerate Dehydrogenase Deficiency",
        "Leber Congenital Amaurosis 2",
        "Dravet Syndrome",
        "Mucopolysaccharidosis Type 3 A",
        "Ornithine Translocase Deficiency",
        "Carnitine-acylcarnitine Translocase Deficiency",
        "Glucose Transporter Type 1 Deficiency Syndrome",
        "Creatine Transporter Deficiency",
        "Niemann-Pick Disease Type A",
        "Pitt Hopkins Syndrome",
        "Tuberous Sclerosis 1",
        "Tuberous Sclerosis 2",
        "Ataxia With Isolated Vitamin E Deficiency",
        "Angelman Syndrome",
        "Prader-Willi Syndrome",
        "Homocystinuria",
        "Permanent Neonatal Diabetes Mellitus",
        "Transient Neonatal Diabetes Mellitus",
        "Factor VII Deficiency",
        "Glycogen Storage Disease Type IXA1",
        "Glycogen Storage Disease, Type IXA2",
        "Glycogen Storage Disease IC",
        "Glycogen Storage Disease Type IB",
        "Central Hypoventilation Syndrome With or Without Hirschsprung Disease"
      ],
      "interventions": [
        {
          "name": "Confirmatory Testing",
          "type": "DIAGNOSTIC_TEST"
        }
      ],
      "intervention_types": [
        "DIAGNOSTIC_TEST"
      ],
      "sponsor": "RTI International",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "1 Day",
        "maximum_age": "31 Days",
        "sex": "ALL",
        "summary": "1 Day to 31 Days"
      },
      "enrollment_count": 30000,
      "start_date": "2018-10-15",
      "completion_date": "2025-12-31",
      "has_results": false,
      "last_update_posted_date": "2025-04-04",
      "last_synced_at": "2026-06-10T13:42:38.865Z",
      "location_count": 1,
      "location_summary": "Research Triangle Park, North Carolina",
      "locations": [
        {
          "city": "Research Triangle Park",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03655223"
    },
    {
      "nct_id": "NCT03513328",
      "title": "Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Bone Marrow Failure Syndrome",
        "Thalassemia",
        "Sickle Cell Disease",
        "Diamond Blackfan Anemia",
        "Acquired Neutropenia in Newborn",
        "Acquired Anemia Hemolytic",
        "Acquired Thrombocytopenia",
        "Hemophagocytic Lymphohistiocytoses",
        "Wiskott-Aldrich Syndrome",
        "Chronic Granulomatous Disease",
        "Common Variable Immunodeficiency",
        "X-linked Lymphoproliferative Disease",
        "Severe Combined Immunodeficiency",
        "Hurler Syndrome",
        "Mannosidosis",
        "Adrenoleukodystrophy"
      ],
      "interventions": [
        {
          "name": "Thiotepa--single daily dose",
          "type": "DRUG"
        },
        {
          "name": "Thiotepa--escalated dose",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of Florida",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Months",
        "maximum_age": "39 Years",
        "sex": "ALL",
        "summary": "3 Months to 39 Years"
      },
      "enrollment_count": 6,
      "start_date": "2018-06-15",
      "completion_date": "2023-02-19",
      "has_results": true,
      "last_update_posted_date": "2023-09-15",
      "last_synced_at": "2026-06-10T13:42:38.865Z",
      "location_count": 1,
      "location_summary": "Gainesville, Florida",
      "locations": [
        {
          "city": "Gainesville",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03513328"
    }
  ]
}