{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=30", "query": { "condition": "Inborn Errors of Metabolism", "page": 30 }, "page_size": 10 }, "pagination": { "page": 30, "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=31&page_size=10", "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=29&page_size=10" }, "source": "remote", "last_synced_at": "2026-10-16T12:29:41.883Z", "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": "NCT04125459", "title": "Assessing the Role of the NLRP3 Inflammasome in Intercritical Gout", "overall_status": "UNKNOWN", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Gout" ], "interventions": [ { "name": "Joint Biopsy", "type": "PROCEDURE" } ], "intervention_types": [ "PROCEDURE" ], "sponsor": "Attune Health Research, Inc.", "sponsor_class": "INDUSTRY", "healthy_volunteers": true, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 8, "start_date": "2020-01-22", "completion_date": "2024-10-03", "has_results": false, "last_update_posted_date": "2024-08-06", "last_synced_at": "2026-10-16T12:29:41.883Z", "location_count": 1, "location_summary": "Beverly Hills, California", "locations": [ { "city": "Beverly Hills", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT04125459" }, { "nct_id": "NCT00645879", "title": "Anaplerotic Therapy in Propionic Acidemia", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Propionic Acidemia" ], "interventions": [ { "name": "ornithine alpha ketoglutarate", "type": "DRUG" }, { "name": "glutamine", "type": "DRUG" }, { "name": "disodium citrate", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Nicola Longo", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "5 Years", "maximum_age": "12 Years", "sex": "ALL", "summary": "5 Years to 12 Years" }, "enrollment_count": 3, "start_date": "2008-07", "completion_date": "2010-02", "has_results": false, "last_update_posted_date": "2015-01-21", "last_synced_at": "2026-10-16T12:29:41.883Z", "location_count": 1, "location_summary": "Salt Lake City, Utah", "locations": [ { "city": "Salt Lake City", "state": "Utah" } ], "official_url": "https://clinicaltrials.gov/study/NCT00645879" }, { "nct_id": "NCT06567769", "title": "Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)", "overall_status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Sanfilippo Syndrome Type A" ], "interventions": [ { "name": "GC1130A", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "GC Biopharma Corp", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "12 Months", "maximum_age": "18 Years", "sex": "ALL", "summary": "12 Months to 18 Years" }, "enrollment_count": 9, "start_date": "2024-11-21", "completion_date": "2027-06", "has_results": false, "last_update_posted_date": "2026-09-08", "last_synced_at": "2026-10-16T12:29:41.883Z", "location_count": 2, "location_summary": "Oakland, California • Minneapolis, Minnesota", "locations": [ { "city": "Oakland", "state": "California" }, { "city": "Minneapolis", "state": "Minnesota" } ], "official_url": "https://clinicaltrials.gov/study/NCT06567769" }, { "nct_id": "NCT00985127", "title": "Study to Evaluate the Urate-Lowering Activity and Safety of Oral BCX4208 Administered in Subjects With Gout", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Gout" ], "interventions": [ { "name": "placebo", "type": "DRUG" }, { "name": "BCX4208", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "BioCryst Pharmaceuticals", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": "69 Years", "sex": "ALL", "summary": "18 Years to 69 Years" }, "enrollment_count": 99, "start_date": "2009-09", "completion_date": "2010-09", "has_results": false, "last_update_posted_date": "2012-01-23", "last_synced_at": "2026-10-16T12:29:41.883Z", "location_count": 24, "location_summary": "Scottsdale, Arizona • Tucson, Arizona • Irvine, California + 21 more", "locations": [ { "city": "Scottsdale", "state": "Arizona" }, { "city": "Tucson", "state": "Arizona" }, { "city": "Irvine", "state": "California" }, { "city": "San Diego", "state": "California" }, { "city": "DeLand", "state": "Florida" } ], "official_url": "https://clinicaltrials.gov/study/NCT00985127" }, { "nct_id": "NCT05019027", "title": "N-of-1 for Beta-Blockers in Cardiac Amyloidosis", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE4" ], "conditions": [ "Cardiac Amyloidosis", "Heart Diseases", "TTR Cardiac Amyloidosis" ], "interventions": [ { "name": "Beta blocker", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Weill Medical College of Cornell University", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "65 Years", "maximum_age": null, "sex": "ALL", "summary": "65 Years and older" }, "enrollment_count": 14, "start_date": "2024-01-31", "completion_date": "2026-06-04", "has_results": false, "last_update_posted_date": "2026-07-29", "last_synced_at": "2026-10-16T12:29:41.883Z", "location_count": 1, "location_summary": "New York, New York", "locations": [ { "city": "New York", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT05019027" }, { "nct_id": "NCT00875160", "title": "A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101", "overall_status": "TERMINATED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Type 1 Gaucher Disease" ], "interventions": [ { "name": "AT2101", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Amicus Therapeutics", "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": 1, "start_date": "2009-04", "completion_date": "2009-07", "has_results": false, "last_update_posted_date": "2010-08-19", "last_synced_at": "2026-10-16T12:29:41.883Z", "location_count": 1, "location_summary": "Miramar, Florida", "locations": [ { "city": "Miramar", "state": "Florida" } ], "official_url": "https://clinicaltrials.gov/study/NCT00875160" }, { "nct_id": "NCT03934450", "title": "Metabolism and Pharmacokinetics of Primaquine Enantiomers in Human Volunteers Receiving a Seven Day Dose Regimen", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Malaria", "Glucose 6 Phosphate Dehydrogenase Deficiency" ], "interventions": [ { "name": "RPQ", "type": "DRUG" }, { "name": "SPQ", "type": "DRUG" }, { "name": "Primaquine Phosphate", "type": "DRUG" }, { "name": "Placebo", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "University of Mississippi, Oxford", "sponsor_class": "OTHER", "healthy_volunteers": true, "eligibility": { "minimum_age": "18 Years", "maximum_age": "65 Years", "sex": "ALL", "summary": "18 Years to 65 Years" }, "enrollment_count": 36, "start_date": "2018-08-17", "completion_date": "2019-05-01", "has_results": false, "last_update_posted_date": "2019-05-14", "last_synced_at": "2026-10-16T12:29:41.883Z", "location_count": 1, "location_summary": "University, Mississippi", "locations": [ { "city": "University", "state": "Mississippi" } ], "official_url": "https://clinicaltrials.gov/study/NCT03934450" }, { "nct_id": "NCT03718403", "title": "Effect of Theophylline in Pseudohypoparathyroidism", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE4" ], "conditions": [ "PHP Ia", "PHP IB", "Php1C" ], "interventions": [ { "name": "Theophylline", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Massachusetts General Hospital", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "5 Years", "maximum_age": "21 Years", "sex": "ALL", "summary": "5 Years to 21 Years" }, "enrollment_count": 100, "start_date": "2019-11-11", "completion_date": "2032-04-10", "has_results": false, "last_update_posted_date": "2022-07-22", "last_synced_at": "2026-10-16T12:29:41.883Z", "location_count": 1, "location_summary": "Boston, Massachusetts", "locations": [ { "city": "Boston", "state": "Massachusetts" } ], "official_url": "https://clinicaltrials.gov/study/NCT03718403" }, { "nct_id": "NCT06539169", "title": "FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases", "overall_status": "UNKNOWN", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Alpha-Thalassemia", "Beta-Thalassemia", "Amyloidosis", "Amyotrophic Lateral Sclerosis", "Creutzfeld-Jakob Disease", "Cystic Fibrosis", "Duchenne Muscular Dystrophy", "Early-Onset Alzheimer Disease", "Ehlers-Danlos Syndrome", "Huntington Disease", "Gaucher Disease", "GM1 Gangliosidosis", "Myasthenia Gravis", "Pompe Disease", "Sickle Cell Disease", "Transthyretin Amyloid Cardiomyopathy", "Rare Diseases" ], "interventions": [], "intervention_types": [], "sponsor": "xCures", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 1000, "start_date": "2024-06-10", "completion_date": "2026-06-10", "has_results": false, "last_update_posted_date": "2024-11-14", "last_synced_at": "2026-10-16T12:29:41.883Z", "location_count": 1, "location_summary": "Los Altos, California", "locations": [ { "city": "Los Altos", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT06539169" }, { "nct_id": "NCT06092346", "title": "A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "AMPD3, OMIM*102772, AMP Deaminase Deficiency", "AK1, OMIM *103000, Adenylate Kinase Deficiency", "AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency", "TPMT, OMIM *187680, Thoipurines, Poor Metabolism of", "IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11", "APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency", "HPRT1, OMIM *308000 Lesch-Nyhan Disease", "XDH, OMIM *607633, Xanthinuria Type 1", "SLC2A9, OMIM *606142 Hypouricemia", "SLC22A12, OMIM *607096 Hypouricemia", "PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease", "PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity", "AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia", "ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35", "ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency", "PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency", "ADA2, OMIM *607575,Sneddon Syndrome; VAIHS", "CAD, *1140120, Developmental and Epileptic Encephalopathy", "UPB1, OMIM *606673, Beta-ureidopropionase Deficiency", "DPYS, OMIM *613326, Dihydropyrimidinase Deficiency", "DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency", "DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis)", "UMPS, OMIM *613891, Orotic Aciduria", "NT5C3A, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency", "UNG, OMIM *191525, Hyper-IgM Syndrome 5", "AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2", "Purine-Pyrimidine Metabolism", "Metabolic Disease" ], "interventions": [], "intervention_types": [], "sponsor": "National Human Genome Research Institute (NHGRI)", "sponsor_class": "NIH", "healthy_volunteers": false, "eligibility": { "minimum_age": "1 Month", "maximum_age": "100 Years", "sex": "ALL", "summary": "1 Month to 100 Years" }, "enrollment_count": 999, "start_date": "2023-12-19", "completion_date": "2099-01-01", "has_results": false, "last_update_posted_date": "2026-08-19", "last_synced_at": "2026-10-16T12:29:41.883Z", "location_count": 1, "location_summary": "Bethesda, Maryland", "locations": [ { "city": "Bethesda", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT06092346" } ] }