{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=GNE+Myopathy",
    "query": {
      "condition": "GNE Myopathy"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 13,
    "total_pages": 2,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=GNE+Myopathy&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-07-22T04:37:19.837Z",
  "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": "NCT01417533",
      "title": "A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "GNE Myopathy",
        "GNE Related Diseases"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": "80 Years",
        "sex": "ALL",
        "summary": "4 Years to 80 Years"
      },
      "enrollment_count": 78,
      "start_date": "2011-09-14",
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2026-07-16",
      "last_synced_at": "2026-07-22T04:37:19.837Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01417533"
    },
    {
      "nct_id": "NCT02736188",
      "title": "Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Hereditary Inclusion Body Myopathy",
        "Distal Myopathy With Rimmed Vacuoles",
        "Distal Myopathy, Nonaka Type",
        "GNE Myopathy",
        "Quadriceps Sparing Myopathy"
      ],
      "interventions": [
        {
          "name": "Aceneuramic Acid Extended-Release Tablets",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Ultragenyx Pharmaceutical Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 143,
      "start_date": "2016-05-02",
      "completion_date": "2018-01-10",
      "has_results": true,
      "last_update_posted_date": "2023-03-24",
      "last_synced_at": "2026-07-22T04:37:19.837Z",
      "location_count": 5,
      "location_summary": "Los Angeles, California • Orange, California • St Louis, Missouri + 1 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "St Louis",
          "state": "Missouri"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02736188"
    },
    {
      "nct_id": "NCT01634750",
      "title": "Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Hereditary Inclusion Body Myopathy (HIBM)",
        "GNE Myopathy"
      ],
      "interventions": [
        {
          "name": "ManNAc",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "70 Years",
        "sex": "ALL",
        "summary": "18 Years to 70 Years"
      },
      "enrollment_count": 26,
      "start_date": "2012-09-11",
      "completion_date": "2013-05-29",
      "has_results": false,
      "last_update_posted_date": "2017-10-19",
      "last_synced_at": "2026-07-22T04:37:19.837Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01634750"
    },
    {
      "nct_id": "NCT01517880",
      "title": "A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "GNE Myopathy",
        "Hereditary Inclusion Body Myopathy"
      ],
      "interventions": [
        {
          "name": "Sialic Acid Extended Release (SA-ER)",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Ultragenyx Pharmaceutical Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "18 Years to 65 Years"
      },
      "enrollment_count": 46,
      "start_date": "2012-05",
      "completion_date": "2013-11",
      "has_results": false,
      "last_update_posted_date": "2016-06-16",
      "last_synced_at": "2026-07-22T04:37:19.837Z",
      "location_count": 3,
      "location_summary": "Los Angeles, California • St Louis, Missouri • New York, New York",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "St Louis",
          "state": "Missouri"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01517880"
    },
    {
      "nct_id": "NCT07511556",
      "title": "First-in-human Study of UX016 in GNEM",
      "overall_status": "NOT_YET_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "GNE Myopathy"
      ],
      "interventions": [
        {
          "name": "UX016",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Ultragenyx Pharmaceutical Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "18 Years to 55 Years"
      },
      "enrollment_count": 24,
      "start_date": "2026-10",
      "completion_date": "2028-12",
      "has_results": false,
      "last_update_posted_date": "2026-04-06",
      "last_synced_at": "2026-07-22T04:37:19.837Z",
      "location_count": 2,
      "location_summary": "Orange, California • Iselin, New Jersey",
      "locations": [
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Iselin",
          "state": "New Jersey"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07511556"
    },
    {
      "nct_id": "NCT01830972",
      "title": "An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "GNE Myopathy",
        "Hereditary Inclusion Body Myopathy (HIBM)"
      ],
      "interventions": [
        {
          "name": "SA-ER 500 mg",
          "type": "DRUG"
        },
        {
          "name": "SA-IR 500 mg",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Ultragenyx Pharmaceutical Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "18 Years to 65 Years"
      },
      "enrollment_count": 59,
      "start_date": "2013-06-04",
      "completion_date": "2017-02-14",
      "has_results": true,
      "last_update_posted_date": "2018-04-11",
      "last_synced_at": "2026-07-22T04:37:19.837Z",
      "location_count": 3,
      "location_summary": "Los Angeles, California • St Louis, Missouri • New York, New York",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "St Louis",
          "state": "Missouri"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01830972"
    },
    {
      "nct_id": "NCT02731690",
      "title": "A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Hereditary Inclusion Body Myopathy",
        "Distal Myopathy With Rimmed Vacuoles",
        "Distal Myopathy, Nonaka Type",
        "GNE Myopathy",
        "Quadriceps Sparing Myopathy",
        "Inclusion Body Myopathy 2"
      ],
      "interventions": [
        {
          "name": "Aceneuramic Acid Extended-Release",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Ultragenyx Pharmaceutical Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 42,
      "start_date": "2016-04-29",
      "completion_date": "2018-01-10",
      "has_results": true,
      "last_update_posted_date": "2019-02-19",
      "last_synced_at": "2026-07-22T04:37:19.837Z",
      "location_count": 3,
      "location_summary": "Orange, California • St Louis, Missouri • New York, New York",
      "locations": [
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "St Louis",
          "state": "Missouri"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02731690"
    },
    {
      "nct_id": "NCT00195637",
      "title": "Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Hereditary Inclusion Body Myopathy"
      ],
      "interventions": [
        {
          "name": "Immune Globulin",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "70 Years",
        "sex": "ALL",
        "summary": "18 Years to 70 Years"
      },
      "enrollment_count": 4,
      "start_date": "2005-09-16",
      "completion_date": "2006-07-03",
      "has_results": false,
      "last_update_posted_date": "2019-12-04",
      "last_synced_at": "2026-07-22T04:37:19.837Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00195637"
    },
    {
      "nct_id": "NCT02377921",
      "title": "Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Hereditary Inclusion Body Myopathy",
        "Distal Myopathy With Rimmed Vacuoles",
        "Distal Myopathy, Nonaka Type",
        "GNE Myopathy"
      ],
      "interventions": [
        {
          "name": "aceneuramic acid extended-release (Ace-ER)",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Ultragenyx Pharmaceutical Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "18 Years to 55 Years"
      },
      "enrollment_count": 89,
      "start_date": "2015-05-20",
      "completion_date": "2017-06-09",
      "has_results": true,
      "last_update_posted_date": "2019-06-27",
      "last_synced_at": "2026-07-22T04:37:19.837Z",
      "location_count": 4,
      "location_summary": "Irvine, California • St Louis, Missouri • New York, New York",
      "locations": [
        {
          "city": "Irvine",
          "state": "California"
        },
        {
          "city": "St Louis",
          "state": "Missouri"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02377921"
    },
    {
      "nct_id": "NCT04231266",
      "title": "Multi-Center Study of ManNAc for GNE Myopathy",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "GNE Myopathy"
      ],
      "interventions": [
        {
          "name": "ManNAc",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Leadiant Biosciences, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "70 Years",
        "sex": "ALL",
        "summary": "18 Years to 70 Years"
      },
      "enrollment_count": 54,
      "start_date": "2022-04-05",
      "completion_date": "2025-10-30",
      "has_results": false,
      "last_update_posted_date": "2025-06-13",
      "last_synced_at": "2026-07-22T04:37:19.837Z",
      "location_count": 10,
      "location_summary": "Los Angeles, California • Iowa City, Iowa • Kansas City, Kansas + 7 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Iowa City",
          "state": "Iowa"
        },
        {
          "city": "Kansas City",
          "state": "Kansas"
        },
        {
          "city": "Bethesda",
          "state": "Maryland"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04231266"
    }
  ]
}