{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?sponsor=Lysosomal+and+Rare+Disorders+Research+and+Treatment+Center%2C+Inc.",
    "query": {
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc."
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 10,
    "total_pages": 1,
    "next_page_url": null,
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-10-09T18:26:08.861Z",
  "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": "NCT06614894",
      "title": "An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III",
      "overall_status": "UNKNOWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Sanfilippo Syndrome",
        "MPS3"
      ],
      "interventions": [
        {
          "name": "Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day",
          "type": "DRUG"
        },
        {
          "name": "Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day",
          "type": "DRUG"
        },
        {
          "name": "Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Ozlem Goker-Alpan",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "99 Years",
        "sex": "ALL",
        "summary": "18 Years to 99 Years"
      },
      "enrollment_count": 10,
      "start_date": "2024-12-01",
      "completion_date": "2026-06-30",
      "has_results": false,
      "last_update_posted_date": "2025-08-20",
      "last_synced_at": "2026-10-09T18:26:08.861Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06614894"
    },
    {
      "nct_id": "NCT04055831",
      "title": "Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease Type 1"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "16 Years",
        "maximum_age": "90 Years",
        "sex": "ALL",
        "summary": "16 Years to 90 Years"
      },
      "enrollment_count": 40,
      "start_date": "2019-05-15",
      "completion_date": "2020-05-15",
      "has_results": false,
      "last_update_posted_date": "2019-08-15",
      "last_synced_at": "2026-10-09T18:26:08.861Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04055831"
    },
    {
      "nct_id": "NCT03811496",
      "title": "Biomarker Analysis for GBA Associated Parkinson's Disease",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Parkinson Disease",
        "Gaucher Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "50 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "50 Years and older"
      },
      "enrollment_count": 100,
      "start_date": "2018-02-01",
      "completion_date": "2020-07",
      "has_results": false,
      "last_update_posted_date": "2019-01-23",
      "last_synced_at": "2026-10-09T18:26:08.861Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03811496"
    },
    {
      "nct_id": "NCT04724083",
      "title": "Inflammatory Pathways and Cardiac Growth Factors Associated With Fabry Disease Cardiomyopathy",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry"
      ],
      "interventions": [
        {
          "name": "biomarkers",
          "type": "DIAGNOSTIC_TEST"
        }
      ],
      "intervention_types": [
        "DIAGNOSTIC_TEST"
      ],
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "70 Years",
        "sex": "ALL",
        "summary": "18 Years to 70 Years"
      },
      "enrollment_count": 50,
      "start_date": "2020-12-01",
      "completion_date": "2022-08-01",
      "has_results": false,
      "last_update_posted_date": "2021-01-27",
      "last_synced_at": "2026-10-09T18:26:08.861Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04724083"
    },
    {
      "nct_id": "NCT03812055",
      "title": "Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Lysosomal Storage Diseases"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 50,
      "start_date": "2018-07-06",
      "completion_date": "2020-07",
      "has_results": false,
      "last_update_posted_date": "2019-01-22",
      "last_synced_at": "2026-10-09T18:26:08.861Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03812055"
    },
    {
      "nct_id": "NCT06116071",
      "title": "Biomarkers Related to Bone in Pediatric Gaucher Disease",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease",
        "Bone Diseases"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "5 Years to 21 Years"
      },
      "enrollment_count": 20,
      "start_date": "2023-11-25",
      "completion_date": "2025-12-31",
      "has_results": false,
      "last_update_posted_date": "2024-03-13",
      "last_synced_at": "2026-10-09T18:26:08.861Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06116071"
    },
    {
      "nct_id": "NCT06065605",
      "title": "Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "80 Years",
        "sex": "ALL",
        "summary": "18 Years to 80 Years"
      },
      "enrollment_count": 40,
      "start_date": "2023-09-14",
      "completion_date": "2025-09-12",
      "has_results": false,
      "last_update_posted_date": "2023-10-04",
      "last_synced_at": "2026-10-09T18:26:08.861Z",
      "location_count": 2,
      "location_summary": "Rockville, Maryland • Fairfax, Virginia",
      "locations": [
        {
          "city": "Rockville",
          "state": "Maryland"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06065605"
    },
    {
      "nct_id": "NCT05705674",
      "title": "The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Sanfilippo Syndrome",
        "MPS3"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": "99 Years",
        "sex": "ALL",
        "summary": "5 Years to 99 Years"
      },
      "enrollment_count": 6,
      "start_date": "2023-05-01",
      "completion_date": "2024-12",
      "has_results": false,
      "last_update_posted_date": "2024-03-12",
      "last_synced_at": "2026-10-09T18:26:08.861Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05705674"
    },
    {
      "nct_id": "NCT02605603",
      "title": "SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 30,
      "start_date": "2015-05",
      "completion_date": "2020-05",
      "has_results": false,
      "last_update_posted_date": "2019-08-13",
      "last_synced_at": "2026-10-09T18:26:08.861Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02605603"
    },
    {
      "nct_id": "NCT03812042",
      "title": "Screening of Lysosomal Storage Disorders Diseases in Minority Groups",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Lysosomal Storage Diseases"
      ],
      "interventions": [
        {
          "name": "Enzyme assay and molecular sequencing",
          "type": "DIAGNOSTIC_TEST"
        }
      ],
      "intervention_types": [
        "DIAGNOSTIC_TEST"
      ],
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 100000,
      "start_date": "2016-03-17",
      "completion_date": "2019-12",
      "has_results": false,
      "last_update_posted_date": "2019-01-22",
      "last_synced_at": "2026-10-09T18:26:08.861Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03812042"
    }
  ]
}