{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=19", "query": { "condition": "Inborn Errors of Metabolism Disorders", "page": 19 }, "page_size": 10 }, "pagination": { "page": 19, "page_size": 10, "total_count": 1510, "total_pages": 151, "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=20&page_size=10", "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=18&page_size=10" }, "source": "remote", "last_synced_at": "2026-10-24T08:00:35.272Z", "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": "NCT04532047", "title": "PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "MPS I", "MPS II", "MPS IVA", "MPS VI", "Mps VII", "Gaucher Disease, Type 2", "Gaucher Disease, Type 3", "Pompe Disease Infantile-Onset", "Wolman Disease" ], "interventions": [ { "name": "Aldurazyme (laronidase)", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "University of California, San Francisco", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": "50 Years", "sex": "FEMALE", "summary": "18 Years to 50 Years · Female only" }, "enrollment_count": 10, "start_date": "2021-07-01", "completion_date": "2032-07-31", "has_results": false, "last_update_posted_date": "2026-03-17", "last_synced_at": "2026-10-24T08:00:35.272Z", "location_count": 1, "location_summary": "San Francisco, California", "locations": [ { "city": "San Francisco", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT04532047" }, { "nct_id": "NCT01065558", "title": "Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Lesch-Nyhan Disease" ], "interventions": [ { "name": "Ecopipam", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Psyadon Pharma", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "6 Years", "maximum_age": "65 Years", "sex": "MALE", "summary": "6 Years to 65 Years · Male only" }, "enrollment_count": 5, "start_date": "2010-02", "completion_date": "2011-06", "has_results": true, "last_update_posted_date": "2013-04-18", "last_synced_at": "2026-10-24T08:00:35.272Z", "location_count": 2, "location_summary": "San Diego, California • Atlanta, Georgia", "locations": [ { "city": "San Diego", "state": "California" }, { "city": "Atlanta", "state": "Georgia" } ], "official_url": "https://clinicaltrials.gov/study/NCT01065558" }, { "nct_id": "NCT07062848", "title": "An Observational Study Using Artificial Intelligence (AI) Algorithms on Electrocardiography (ECG), Point-of-care Ultrasound (POCUS), and Transthoracic Echocardiophy (TTE) to Estimate the Under-diagnosis of Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Across a Diverse Range of US Health Systems.", "overall_status": "ACTIVE_NOT_RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Transthyretin (TTR) Amyloid Cardiomyopathy" ], "interventions": [ { "name": "AI Toolkit for ATTR-CM Diagnosis", "type": "DIAGNOSTIC_TEST" } ], "intervention_types": [ "DIAGNOSTIC_TEST" ], "sponsor": "Yale University", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "50 Years", "maximum_age": "95 Years", "sex": "ALL", "summary": "50 Years to 95 Years" }, "enrollment_count": 1500000, "start_date": "2025-01-24", "completion_date": "2027-01", "has_results": false, "last_update_posted_date": "2026-07-29", "last_synced_at": "2026-10-24T08:00:35.272Z", "location_count": 12, "location_summary": "San Francisco, California • New Haven, Connecticut • Chicago, Illinois + 9 more", "locations": [ { "city": "San Francisco", "state": "California" }, { "city": "New Haven", "state": "Connecticut" }, { "city": "Chicago", "state": "Illinois" }, { "city": "Detroit", "state": "Michigan" }, { "city": "New York", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT07062848" }, { "nct_id": "NCT01716741", "title": "Identification of Undiagnosed Gaucher Disease", "overall_status": "UNKNOWN", "study_type": "INTERVENTIONAL", "phases": [ "NA" ], "conditions": [ "Gaucher Disease" ], "interventions": [ { "name": "Enzyme analysis", "type": "OTHER" } ], "intervention_types": [ "OTHER" ], "sponsor": "Brigham and Women's Hospital", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 100, "start_date": "2012-08", "completion_date": null, "has_results": false, "last_update_posted_date": "2012-10-30", "last_synced_at": "2026-10-24T08:00:35.272Z", "location_count": 1, "location_summary": "Boston, Massachusetts", "locations": [ { "city": "Boston", "state": "Massachusetts" } ], "official_url": "https://clinicaltrials.gov/study/NCT01716741" }, { "nct_id": "NCT04721366", "title": "A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Gaucher Disease" ], "interventions": [ { "name": "Standard of Care", "type": "OTHER" } ], "intervention_types": [ "OTHER" ], "sponsor": "Takeda", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "5 Years", "sex": "ALL", "summary": "Up to 5 Years" }, "enrollment_count": 11, "start_date": "2021-01-08", "completion_date": "2023-04-17", "has_results": false, "last_update_posted_date": "2024-03-29", "last_synced_at": "2026-10-24T08:00:35.272Z", "location_count": 1, "location_summary": "Fairfax, Virginia", "locations": [ { "city": "Fairfax", "state": "Virginia" } ], "official_url": "https://clinicaltrials.gov/study/NCT04721366" }, { "nct_id": "NCT04152551", "title": "Effects of Bisphosphonates on OI-Related Hearing Loss", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE4" ], "conditions": [ "Osteogenesis Imperfecta" ], "interventions": [ { "name": "Risedronate Oral Tablet", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Hospital for Special Surgery, New York", "sponsor_class": "OTHER", "healthy_volunteers": true, "eligibility": { "minimum_age": "6 Years", "maximum_age": "100 Years", "sex": "ALL", "summary": "6 Years to 100 Years" }, "enrollment_count": 100, "start_date": "2019-11-02", "completion_date": "2027-01", "has_results": false, "last_update_posted_date": "2026-04-28", "last_synced_at": "2026-10-24T08:00:35.272Z", "location_count": 1, "location_summary": "New York, New York", "locations": [ { "city": "New York", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT04152551" }, { "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": "2026-10-24T08:00:35.272Z", "location_count": 1, "location_summary": "Gainesville, Florida", "locations": [ { "city": "Gainesville", "state": "Florida" } ], "official_url": "https://clinicaltrials.gov/study/NCT03513328" }, { "nct_id": "NCT04130204", "title": "Topical Application to Relieve Gout: Efficacy Trial & Safety (TARGETS)", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Gout" ], "interventions": [ { "name": "DYV700", "type": "DRUG" }, { "name": "Placebo", "type": "DRUG" }, { "name": "Colchicine 0.6 mg", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Dyve Biosciences, Inc.", "sponsor_class": "INDUSTRY", "healthy_volunteers": true, "eligibility": { "minimum_age": "18 Years", "maximum_age": "75 Years", "sex": "ALL", "summary": "18 Years to 75 Years" }, "enrollment_count": 284, "start_date": "2020-02-12", "completion_date": "2021-05-21", "has_results": false, "last_update_posted_date": "2022-05-13", "last_synced_at": "2026-10-24T08:00:35.272Z", "location_count": 1, "location_summary": "Poway, California", "locations": [ { "city": "Poway", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT04130204" }, { "nct_id": "NCT03539952", "title": "Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE3" ], "conditions": [ "Wilson Disease" ], "interventions": [ { "name": "Penicillamine (D1-W12)", "type": "DRUG" }, { "name": "TETA 4HCL (W12-60)", "type": "DRUG" }, { "name": "Penicillamine (W12-W60)", "type": "DRUG" }, { "name": "TETA 4HCL (60-