{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Orphan+Diseases",
    "query": {
      "condition": "Orphan Diseases"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 23,
    "total_pages": 3,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Orphan+Diseases&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-05-22T06:43:39.576Z",
  "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": "NCT03495921",
      "title": "A Trial For Participants With Ewing's Sarcoma Treated With Vigil in Combination With Irinotecan and Temozolomide",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Ewing Sarcoma",
        "Ewing Family of Tumors",
        "Ewing's Tumor Metastatic",
        "Ewing's Sarcoma Metastatic",
        "Ewing's Tumor Recurrent",
        "Rare Diseases",
        "Sarcoma",
        "Neoplasms, Connective and Soft Tissue",
        "Neoplasms by Histologic Type",
        "Neoplasms, Bone Tissue",
        "Neoplasms, Connective Tissue",
        "Sarcoma, Ewing",
        "Neoplasms"
      ],
      "interventions": [
        {
          "name": "Vigil",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Irinotecan",
          "type": "DRUG"
        },
        {
          "name": "Temozolomide",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "DRUG"
      ],
      "sponsor": "Gradalis, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "2 Years and older"
      },
      "enrollment_count": 32,
      "start_date": "2018-08-21",
      "completion_date": "2022-01-20",
      "has_results": true,
      "last_update_posted_date": "2023-04-26",
      "last_synced_at": "2026-05-22T06:43:39.576Z",
      "location_count": 15,
      "location_summary": "Little Rock, Arkansas • Los Angeles, California • Jacksonville, Florida + 12 more",
      "locations": [
        {
          "city": "Little Rock",
          "state": "Arkansas"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Jacksonville",
          "state": "Florida"
        },
        {
          "city": "Miami",
          "state": "Florida"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03495921"
    },
    {
      "nct_id": "NCT06912763",
      "title": "Reversing External-beam Radiotherapy-associated Fibrosis Syndrome: an Interventional Bayesian Adaptive Randomized-controlled Orphan Drug Platform Trial for Orodental Sequelae (Reverse-fibrose)",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Fibrosis Syndrome",
        "Lymphedema",
        "Head &Amp; Neck Cancer",
        "Fibrosis"
      ],
      "interventions": [
        {
          "name": "Pravastatin (drug)",
          "type": "DRUG"
        },
        {
          "name": "Pentoxifylline",
          "type": "DRUG"
        },
        {
          "name": "ketoprofen",
          "type": "DRUG"
        },
        {
          "name": "Pirfenidoneone",
          "type": "DRUG"
        },
        {
          "name": "Standard of Care (SOC)",
          "type": "OTHER"
        },
        {
          "name": "tocopherol",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "M.D. Anderson Cancer Center",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "FEMALE",
        "summary": "Female only"
      },
      "enrollment_count": 250,
      "start_date": "2025-08-08",
      "completion_date": "2033-03-01",
      "has_results": false,
      "last_update_posted_date": "2026-01-26",
      "last_synced_at": "2026-05-22T06:43:39.576Z",
      "location_count": 1,
      "location_summary": "Houston, Texas",
      "locations": [
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06912763"
    },
    {
      "nct_id": "NCT03088878",
      "title": "A Study of Cirmtuzumab and Ibrutinib in Patients With B-Cell Lymphoid Malignancies",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "B-cell Chronic Lymphocytic Leukemia",
        "Small Lymphocytic Lymphoma",
        "Mantle Cell Lymphoma",
        "Marginal Zone Lymphoma"
      ],
      "interventions": [
        {
          "name": "Cirmtuzumab (2-16 kg/mg) plus Ibrutinib",
          "type": "DRUG"
        },
        {
          "name": "Cirmtuzumab (300mg) plus Ibrutinib",
          "type": "DRUG"
        },
        {
          "name": "Cirmtuzumab (600 mg) plus ibrutinib",
          "type": "DRUG"
        },
        {
          "name": "Cirmtuzumab (RDR) plus ibrutinib",
          "type": "DRUG"
        },
        {
          "name": "Cirmtuzumab plus ibrutinib",
          "type": "DRUG"
        },
        {
          "name": "Ibrutinib alone",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Oncternal Therapeutics, Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 95,
      "start_date": "2018-01-03",
      "completion_date": "2024-09-25",
      "has_results": true,
      "last_update_posted_date": "2025-02-12",
      "last_synced_at": "2026-05-22T06:43:39.576Z",
      "location_count": 12,
      "location_summary": "Duarte, California • La Jolla, California • Sacramento, California + 8 more",
      "locations": [
        {
          "city": "Duarte",
          "state": "California"
        },
        {
          "city": "La Jolla",
          "state": "California"
        },
        {
          "city": "Sacramento",
          "state": "California"
        },
        {
          "city": "New Haven",
          "state": "Connecticut"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03088878"
    },
    {
      "nct_id": "NCT04220190",
      "title": "RAPA-501 Therapy for ALS",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Amyotrophic Lateral Sclerosis"
      ],
      "interventions": [
        {
          "name": "RAPA-501 Autologous T stem cells",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Rapa Therapeutics LLC",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 41,
      "start_date": "2025-01-02",
      "completion_date": "2027-06-01",
      "has_results": false,
      "last_update_posted_date": "2025-06-18",
      "last_synced_at": "2026-05-22T06:43:39.576Z",
      "location_count": 1,
      "location_summary": "Boston, Massachusetts",
      "locations": [
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04220190"
    },
    {
      "nct_id": "NCT01412034",
      "title": "Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Homozygous Familial Hypercholesterolemia"
      ],
      "interventions": [
        {
          "name": "CER-001",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Cerenis Therapeutics, SA",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "12 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "12 Years and older"
      },
      "enrollment_count": 23,
      "start_date": "2011-11",
      "completion_date": "2014-08",
      "has_results": false,
      "last_update_posted_date": "2015-08-18",
      "last_synced_at": "2026-05-22T06:43:39.576Z",
      "location_count": 2,
      "location_summary": "Hartford, Connecticut • North Massapequa, New York",
      "locations": [
        {
          "city": "Hartford",
          "state": "Connecticut"
        },
        {
          "city": "North Massapequa",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01412034"
    },
    {
      "nct_id": "NCT02743845",
      "title": "Gene Discovery Core, The Manton Center",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Undiagnosed Conditions",
        "Rare Disorders",
        "Orphan Diseases"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Boston Children's Hospital",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 10000,
      "start_date": "2010-02",
      "completion_date": "2030-12",
      "has_results": false,
      "last_update_posted_date": "2026-03-25",
      "last_synced_at": "2026-05-22T06:43:39.576Z",
      "location_count": 1,
      "location_summary": "Boston, Massachusetts",
      "locations": [
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02743845"
    },
    {
      "nct_id": "NCT01000961",
      "title": "Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Cystinosis"
      ],
      "interventions": [
        {
          "name": "Cystagon® (Cysteamine Bitartrate)",
          "type": "DRUG"
        },
        {
          "name": "Cysteamine Bitartrate Delayed-release Capsules (RP103)",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Amgen",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "6 Years and older"
      },
      "enrollment_count": 43,
      "start_date": "2010-06",
      "completion_date": "2011-08",
      "has_results": true,
      "last_update_posted_date": "2024-12-19",
      "last_synced_at": "2026-05-22T06:43:39.576Z",
      "location_count": 3,
      "location_summary": "Stanford, California • Atlanta, Georgia • Chicago, Illinois",
      "locations": [
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01000961"
    },
    {
      "nct_id": "NCT01573754",
      "title": "Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Porphyria Cutanea Tarda"
      ],
      "interventions": [
        {
          "name": "Hydroxychloroquine",
          "type": "DRUG"
        },
        {
          "name": "Phlebotomy",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "DRUG",
        "PROCEDURE"
      ],
      "sponsor": "The University of Texas Medical Branch, Galveston",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "100 Years",
        "sex": "ALL",
        "summary": "18 Years to 100 Years"
      },
      "enrollment_count": 48,
      "start_date": "2006-03-21",
      "completion_date": "2021-07-06",
      "has_results": true,
      "last_update_posted_date": "2023-02-17",
      "last_synced_at": "2026-05-22T06:43:39.576Z",
      "location_count": 1,
      "location_summary": "Galveston, Texas",
      "locations": [
        {
          "city": "Galveston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01573754"
    },
    {
      "nct_id": "NCT06169176",
      "title": "RAPA-501 Therapy of ALS Expanded Access Protocol",
      "overall_status": "TEMPORARILY_NOT_AVAILABLE",
      "study_type": "EXPANDED_ACCESS",
      "phases": [],
      "conditions": [
        "Amyotrophic Lateral Sclerosis"
      ],
      "interventions": [
        {
          "name": "RAPA-501 Autologous T stem cells",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Rapa Therapeutics LLC",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2026-02-12",
      "last_synced_at": "2026-05-22T06:43:39.576Z",
      "location_count": 10,
      "location_summary": "Phoenix, Arizona • Scottsdale, Arizona • Irvine, California + 7 more",
      "locations": [
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "Scottsdale",
          "state": "Arizona"
        },
        {
          "city": "Irvine",
          "state": "California"
        },
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06169176"
    },
    {
      "nct_id": "NCT01744782",
      "title": "Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Cystinosis"
      ],
      "interventions": [
        {
          "name": "RP103",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Amgen",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "6 Years",
        "sex": "ALL",
        "summary": "Up to 6 Years"
      },
      "enrollment_count": 17,
      "start_date": "2012-12-20",
      "completion_date": "2016-12-13",
      "has_results": true,
      "last_update_posted_date": "2024-12-27",
      "last_synced_at": "2026-05-22T06:43:39.576Z",
      "location_count": 1,
      "location_summary": "Chicago, Illinois",
      "locations": [
        {
          "city": "Chicago",
          "state": "Illinois"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01744782"
    }
  ]
}