{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=48", "query": { "condition": "Inborn Errors of Metabolism", "page": 48 }, "page_size": 10 }, "pagination": { "page": 48, "page_size": 10, "total_count": 1605, "total_pages": 161, "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=49&page_size=10", "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=47&page_size=10" }, "source": "remote", "last_synced_at": "2026-10-19T00:03:45.258Z", "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": "NCT06164730", "title": "A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Heterozygous Familial Hypercholesterolemia", "Premature Coronary Heart Disease" ], "interventions": [ { "name": "VERVE-102", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Verve Therapeutics, 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": 85, "start_date": "2024-04-30", "completion_date": "2027-10", "has_results": false, "last_update_posted_date": "2026-09-02", "last_synced_at": "2026-10-19T00:03:45.258Z", "location_count": 10, "location_summary": "Dothan, Alabama • Pomona, California • Boca Raton, Florida + 7 more", "locations": [ { "city": "Dothan", "state": "Alabama" }, { "city": "Pomona", "state": "California" }, { "city": "Boca Raton", "state": "Florida" }, { "city": "Jacksonville", "state": "Florida" }, { "city": "Winter Park", "state": "Florida" } ], "official_url": "https://clinicaltrials.gov/study/NCT06164730" }, { "nct_id": "NCT01419028", "title": "A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Hypophosphatasia (HPP)" ], "interventions": [], "intervention_types": [], "sponsor": "Alexion Pharmaceuticals, Inc.", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 48, "start_date": "2012-08", "completion_date": "2014-02", "has_results": true, "last_update_posted_date": "2019-04-01", "last_synced_at": "2026-10-19T00:03:45.258Z", "location_count": 5, "location_summary": "Los Angeles, California • Indianapolis, Indiana • St Louis, Missouri + 2 more", "locations": [ { "city": "Los Angeles", "state": "California" }, { "city": "Indianapolis", "state": "Indiana" }, { "city": "St Louis", "state": "Missouri" }, { "city": "Portland", "state": "Oregon" }, { "city": "Fort Worth", "state": "Texas" } ], "official_url": "https://clinicaltrials.gov/study/NCT01419028" }, { "nct_id": "NCT01465100", "title": "Liver Cell Transplant for Phenylketonuria", "overall_status": "TERMINATED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Phenylketonuria" ], "interventions": [ { "name": "Preparative Radiation Therapy", "type": "RADIATION" }, { "name": "Hepatocyte Transplant", "type": "PROCEDURE" }, { "name": "Immunosuppression", "type": "DRUG" }, { "name": "Liver Evaluation", "type": "OTHER" }, { "name": "Neuro-psychological Assessment", "type": "BEHAVIORAL" }, { "name": "Whole body Phe oxidation testing", "type": "DIAGNOSTIC_TEST" }, { "name": "Liver Biopsy", "type": "PROCEDURE" } ], "intervention_types": [ "RADIATION", "PROCEDURE", "DRUG", "OTHER", "BEHAVIORAL", "DIAGNOSTIC_TEST" ], "sponsor": "Ira Fox", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "14 Years", "maximum_age": "55 Years", "sex": "ALL", "summary": "14 Years to 55 Years" }, "enrollment_count": 1, "start_date": "2011-10-12", "completion_date": "2016-06-17", "has_results": false, "last_update_posted_date": "2023-08-16", "last_synced_at": "2026-10-19T00:03:45.258Z", "location_count": 1, "location_summary": "Pittsburgh, Pennsylvania", "locations": [ { "city": "Pittsburgh", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT01465100" }, { "nct_id": "NCT02322177", "title": "Maternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry Protocol", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Inborn Errors of Metabolism", "Pregnancy", "Acidemias" ], "interventions": [], "intervention_types": [], "sponsor": "National Human Genome Research Institute (NHGRI)", "sponsor_class": "NIH", "healthy_volunteers": false, "eligibility": { "minimum_age": "14 Years", "maximum_age": "50 Years", "sex": "FEMALE", "summary": "14 Years to 50 Years · Female only" }, "enrollment_count": 2, "start_date": "2014-12-19", "completion_date": "2018-08-20", "has_results": false, "last_update_posted_date": "2026-10-16", "last_synced_at": "2026-10-19T00:03:45.258Z", "location_count": 2, "location_summary": "Bethesda, Maryland", "locations": [ { "city": "Bethesda", "state": "Maryland" }, { "city": "Bethesda", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT02322177" }, { "nct_id": "NCT05797857", "title": "Exercise Training in Transthyretin Cardiac Amyloidosis", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "NA" ], "conditions": [ "Amyloid Cardiomyopathy", "Transthyretin Cardiac Amyloidosis" ], "interventions": [ { "name": "Exercise Training", "type": "OTHER" } ], "intervention_types": [ "OTHER" ], "sponsor": "Brigham and Women's Hospital", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "60 Years", "maximum_age": "90 Years", "sex": "ALL", "summary": "60 Years to 90 Years" }, "enrollment_count": 40, "start_date": "2024-07-10", "completion_date": "2030-06-30", "has_results": false, "last_update_posted_date": "2025-03-18", "last_synced_at": "2026-10-19T00:03:45.258Z", "location_count": 1, "location_summary": "Boston, Massachusetts", "locations": [ { "city": "Boston", "state": "Massachusetts" } ], "official_url": "https://clinicaltrials.gov/study/NCT05797857" }, { "nct_id": "NCT06092034", "title": "A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Danon Disease" ], "interventions": [ { "name": "RP-A501", "type": "GENETIC" } ], "intervention_types": [ "GENETIC" ], "sponsor": "Rocket Pharmaceuticals Inc.", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "8 Years", "maximum_age": null, "sex": "MALE", "summary": "8 Years and older · Male only" }, "enrollment_count": 18, "start_date": "2023-09-05", "completion_date": "2032-06", "has_results": false, "last_update_posted_date": "2026-10-06", "last_synced_at": "2026-10-19T00:03:45.258Z", "location_count": 3, "location_summary": "La Jolla, California • Boston, Massachusetts • Philadelphia, Pennsylvania", "locations": [ { "city": "La Jolla", "state": "California" }, { "city": "Boston", "state": "Massachusetts" }, { "city": "Philadelphia", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT06092034" }, { "nct_id": "NCT02742298", "title": "Pompe Disease QMUS and EIM", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "NA" ], "conditions": [ "Pompe Disease" ], "interventions": [ { "name": "Muscle ultrasound", "type": "DEVICE" }, { "name": "Electrical Impedance Myography", "type": "OTHER" } ], "intervention_types": [ "DEVICE", "OTHER" ], "sponsor": "Duke University", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 25, "start_date": "2016-10-24", "completion_date": "2020-07-31", "has_results": false, "last_update_posted_date": "2020-09-25", "last_synced_at": "2026-10-19T00:03:45.258Z", "location_count": 1, "location_summary": "Durham, North Carolina", "locations": [ { "city": "Durham", "state": "North Carolina" } ], "official_url": "https://clinicaltrials.gov/study/NCT02742298" }, { "nct_id": "NCT00730314", "title": "Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Sickle Cell Disease", "Thalassemia", "Anemia", "Granuloma", "Wiskott-Aldrich Syndrome", "Chediak Higashi Syndrome", "Osteopetrosis", "Neutropenia", "Thrombocytopenia", "Hurler Disease", "Niemann-Pick Disease", "Fucosidosis" ], "interventions": [ { "name": "Hematopoietic stem cell transplantation", "type": "PROCEDURE" } ], "intervention_types": [ "PROCEDURE" ], "sponsor": "Children's Hospital Los Angeles", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "21 Years", "sex": "ALL", "summary": "Up to 21 Years" }, "enrollment_count": 25, "start_date": "2008-08", "completion_date": "2015-08", "has_results": false, "last_update_posted_date": "2016-06-23", "last_synced_at": "2026-10-19T00:03:45.258Z", "location_count": 1, "location_summary": "Los Angeles, California", "locations": [ { "city": "Los Angeles", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT00730314" }, { "nct_id": "NCT07629999", "title": "Single and Multiple Dose Study to Evaluate Safety and Pharmacokinetics of BMS-986533 in Healthy Participants and Assessments of Food and pH Effects on Relative Bioavailability, and Drug-Drug Interaction Potential in Healthy Participants", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Healthy Volunteers" ], "interventions": [ { "name": "BMS-986533", "type": "DRUG" }, { "name": "Famotidine", "type": "DRUG" }, { "name": "Placebo", "type": "DRUG" }, { "name": "Dabigatran Etexilate", "type": "DRUG" }, { "name": "Rosuvastatin", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Bristol-Myers Squibb", "sponsor_class": "INDUSTRY", "healthy_volunteers": true, "eligibility": { "minimum_age": "18 Years", "maximum_age": "55 Years", "sex": "ALL", "summary": "18 Years to 55 Years" }, "enrollment_count": 136, "start_date": "2026-07-13", "completion_date": "2027-06-01", "has_results": false, "last_update_posted_date": "2026-07-24", "last_synced_at": "2026-10-19T00:03:45.258Z", "location_count": 1, "location_summary": "Lincoln, Nebraska", "locations": [ { "city": "Lincoln", "state": "Nebraska" } ], "official_url": "https://clinicaltrials.gov/study/NCT07629999" }, { "nct_id": "NCT00061763", "title": "Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Beta-thalassemia", "Myelodysplastic Syndromes", "Fanconi Syndrome", "Anemia, Diamond-Blackfan", "Anemia, Aplastic" ], "interventions": [ { "name": "Deferasirox", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Novartis Pharmaceuticals", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "2 Years", "maximum_age": null, "sex": "ALL", "summary": "2 Years and older" }, "enrollment_count": 175, "start_date": "2003-05", "completion_date": null, "has_results": false, "last_update_posted_date": "2017-08-22", "last_synced_at": "2026-10-19T00:03:45.258Z", "location_count": 6, "location_summary": "Oakland, California • Stanford, California • Arlington Heights, Illinois + 3 more", "locations": [ { "city": "Oakland", "state": "California" }, { "city": "Stanford", "state": "California" }, { "city": "Arlington Heights", "state": "Illinois" }, { "city": "Boston", "state": "Massachusetts" }, { "city": "New York", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT00061763" } ] }