{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Single+Gene+Disorders",
    "query": {
      "condition": "Single Gene Disorders"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 165,
    "total_pages": 17,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Single+Gene+Disorders&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-09-03T23:32:15.244Z",
  "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": "NCT05317780",
      "title": "Canavan-Single Patient IND",
      "overall_status": "NO_LONGER_AVAILABLE",
      "study_type": "EXPANDED_ACCESS",
      "phases": [],
      "conditions": [
        "Canavan Disease"
      ],
      "interventions": [
        {
          "name": "rAAV9-CB6-AspA",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of Florida",
      "sponsor_class": "OTHER",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": "18 Months",
        "maximum_age": "24 Months",
        "sex": "MALE",
        "summary": "18 Months to 24 Months · Male only"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2023-08-23",
      "last_synced_at": "2026-09-03T23:32:15.244Z",
      "location_count": 1,
      "location_summary": "Gainesville, Florida",
      "locations": [
        {
          "city": "Gainesville",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05317780"
    },
    {
      "nct_id": "NCT01197872",
      "title": "Clinical Use of Parental Support To Detect Single Gene Mutations",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Single Gene Disorders"
      ],
      "interventions": [
        {
          "name": "Preimplantation Genetic Diagnosis",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Natera, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 240,
      "start_date": "2010-09",
      "completion_date": "2013-06",
      "has_results": false,
      "last_update_posted_date": "2013-07-16",
      "last_synced_at": "2026-09-03T23:32:15.244Z",
      "location_count": 1,
      "location_summary": "Redwood City, California",
      "locations": [
        {
          "city": "Redwood City",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01197872"
    },
    {
      "nct_id": "NCT03087071",
      "title": "Panitumumab With or Without Trametinib in Treating Patients With Stage IV Colorectal Cancer",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "EGFR NP_005219.2:p.S492R",
        "KRAS Gene Mutation",
        "MAP2K1 Gene Mutation",
        "Metastatic Colorectal Adenocarcinoma",
        "Refractory Colorectal Adenocarcinoma",
        "Stage IV Colorectal Cancer AJCC v7",
        "Stage IVA Colorectal Cancer AJCC v7",
        "Stage IVB Colorectal Cancer AJCC v7"
      ],
      "interventions": [
        {
          "name": "Laboratory Biomarker Analysis",
          "type": "OTHER"
        },
        {
          "name": "Panitumumab",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Trametinib",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "OTHER",
        "BIOLOGICAL",
        "DRUG"
      ],
      "sponsor": "M.D. Anderson Cancer Center",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 59,
      "start_date": "2017-12-29",
      "completion_date": "2027-01-31",
      "has_results": false,
      "last_update_posted_date": "2026-07-16",
      "last_synced_at": "2026-09-03T23:32:15.244Z",
      "location_count": 1,
      "location_summary": "Houston, Texas",
      "locations": [
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03087071"
    },
    {
      "nct_id": "NCT02071862",
      "title": "Study of the Glutaminase Inhibitor CB-839 in Solid Tumors",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Solid Tumors",
        "Triple-Negative Breast Cancer",
        "Non Small Cell Lung Cancer",
        "Renal Cell Carcinoma",
        "Mesothelioma",
        "Fumarate Hydratase (FH)-Deficient Tumors",
        "Succinate Dehydrogenase (SDH)-Deficient Gastrointestinal Stromal Tumors (GIST)",
        "Succinate Dehydrogenase (SDH)-Deficient Non-gastrointestinal Stromal Tumors",
        "Tumors Harboring Isocitrate Dehydrogenase-1 (IDH1) and IDH2 Mutations",
        "Tumors Harboring Amplifications in the cMyc Gene"
      ],
      "interventions": [
        {
          "name": "CB-839",
          "type": "DRUG"
        },
        {
          "name": "Pac-CB",
          "type": "DRUG"
        },
        {
          "name": "CBE",
          "type": "DRUG"
        },
        {
          "name": "CB-Erl",
          "type": "DRUG"
        },
        {
          "name": "CBD",
          "type": "DRUG"
        },
        {
          "name": "CB-Cabo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Calithera Biosciences, Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 210,
      "start_date": "2014-02",
      "completion_date": "2019-03",
      "has_results": false,
      "last_update_posted_date": "2022-07-20",
      "last_synced_at": "2026-09-03T23:32:15.244Z",
      "location_count": 13,
      "location_summary": "San Francisco, California • Stanford, California • Sarasota, Florida + 7 more",
      "locations": [
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Sarasota",
          "state": "Florida"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02071862"
    },
    {
      "nct_id": "NCT07627971",
      "title": "Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "GJB2-mediated Hearing Loss",
        "GJB2 Gene Mutation",
        "DFNB1A"
      ],
      "interventions": [
        {
          "name": "SKY-GJB2",
          "type": "COMBINATION_PRODUCT"
        }
      ],
      "intervention_types": [
        "COMBINATION_PRODUCT"
      ],
      "sponsor": "Skylark Bio Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "9 Months",
        "maximum_age": "7 Years",
        "sex": "ALL",
        "summary": "9 Months to 7 Years"
      },
      "enrollment_count": 10,
      "start_date": "2026-05-28",
      "completion_date": "2028-06",
      "has_results": false,
      "last_update_posted_date": "2026-08-27",
      "last_synced_at": "2026-09-03T23:32:15.244Z",
      "location_count": 4,
      "location_summary": "Bethesda, Maryland • Boston, Massachusetts • Oklahoma City, Oklahoma + 1 more",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "Oklahoma City",
          "state": "Oklahoma"
        },
        {
          "city": "Allentown",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07627971"
    },
    {
      "nct_id": "NCT01044654",
      "title": "Phase 1 Dose Escalation Study of Autologous T-cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Patients",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "HIV Infection",
        "HIV Infections"
      ],
      "interventions": [
        {
          "name": "SB-728-T",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "Sangamo Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 19,
      "start_date": "2009-12",
      "completion_date": "2014-12",
      "has_results": false,
      "last_update_posted_date": "2015-03-13",
      "last_synced_at": "2026-09-03T23:32:15.244Z",
      "location_count": 9,
      "location_summary": "Los Angeles, California • Newport Beach, California • San Francisco, California + 6 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Newport Beach",
          "state": "California"
        },
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Norwalk",
          "state": "Connecticut"
        },
        {
          "city": "Orlando",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01044654"
    },
    {
      "nct_id": "NCT03569891",
      "title": "HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Hemophilia B"
      ],
      "interventions": [
        {
          "name": "AAV5-hFIXco-Padua",
          "type": "GENETIC"
        },
        {
          "name": "Factor IX (FIX)",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "GENETIC",
        "BIOLOGICAL"
      ],
      "sponsor": "CSL Behring",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "MALE",
        "summary": "18 Years and older · Male only"
      },
      "enrollment_count": 67,
      "start_date": "2018-06-27",
      "completion_date": "2025-03-19",
      "has_results": true,
      "last_update_posted_date": "2026-03-27",
      "last_synced_at": "2026-09-03T23:32:15.244Z",
      "location_count": 19,
      "location_summary": "Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 14 more",
      "locations": [
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "Little Rock",
          "state": "Arkansas"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Sacramento",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03569891"
    },
    {
      "nct_id": "NCT06297486",
      "title": "Study of a Gene Therapy Treatment for Hemophilia A",
      "overall_status": "WITHDRAWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Hemophilia A"
      ],
      "interventions": [
        {
          "name": "SPK-8011",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "Spark Therapeutics, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "MALE",
        "summary": "18 Years and older · Male only"
      },
      "enrollment_count": 0,
      "start_date": "2024-03-13",
      "completion_date": "2035-09-04",
      "has_results": false,
      "last_update_posted_date": "2024-12-13",
      "last_synced_at": "2026-09-03T23:32:15.244Z",
      "location_count": 27,
      "location_summary": "Phoenix, Arizona • Loma Linda, California • Los Angeles, California + 23 more",
      "locations": [
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "Loma Linda",
          "state": "California"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Roseville",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06297486"
    },
    {
      "nct_id": "NCT01143519",
      "title": "Study of the Effect of SNPs in p53 and p53 Response Elements on the Inflammatory Response to DNA Damage",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "HIV-1 Seropositive",
        "Inflammation",
        "Cancer",
        "Cardiomyopathy"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Institute of Environmental Health Sciences (NIEHS)",
      "sponsor_class": "NIH",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "100 Years",
        "sex": "ALL",
        "summary": "18 Years to 100 Years"
      },
      "enrollment_count": 178,
      "start_date": "2012-05-21",
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2026-04-17",
      "last_synced_at": "2026-09-03T23:32:15.244Z",
      "location_count": 1,
      "location_summary": "Research Triangle Park, North Carolina",
      "locations": [
        {
          "city": "Research Triangle Park",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01143519"
    },
    {
      "nct_id": "NCT00573092",
      "title": "Analyzing Gene Regions That May Interact With the Effectiveness of High Blood Pressure Drugs",
      "overall_status": "WITHDRAWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Myocardial Infarction",
        "Cerebrovascular Accident",
        "Death, Sudden, Cardiac"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Washington",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "30 Years",
        "maximum_age": "79 Years",
        "sex": "ALL",
        "summary": "30 Years to 79 Years"
      },
      "enrollment_count": 0,
      "start_date": "2007-09",
      "completion_date": "2016-11",
      "has_results": false,
      "last_update_posted_date": "2016-11-09",
      "last_synced_at": "2026-09-03T23:32:15.244Z",
      "location_count": 1,
      "location_summary": "Seattle, Washington",
      "locations": [
        {
          "city": "Seattle",
          "state": "Washington"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00573092"
    }
  ]
}