{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Mucopolysaccharidosis+Type+IV+A&page=2", "query": { "condition": "Mucopolysaccharidosis Type IV A", "page": 2 }, "page_size": 10 }, "pagination": { "page": 2, "page_size": 10, "total_count": 10, "total_pages": 1, "next_page_url": null, "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Mucopolysaccharidosis+Type+IV+A&page=1&page_size=10" }, "source": "remote", "last_synced_at": "2026-05-21T23:33:57.124Z", "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": "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": "2026-05-21T23:33:57.124Z", "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": "NCT05619900", "title": "Registry of Patients Diagnosed With Lysosomal Storage Diseases", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Mucopolysaccharidosis I", "Mucopolysaccharidosis II", "Mucopolysaccharidosis IV A", "Mucopolysaccharidosis VI", "Mucopolysaccharidosis VII", "Pompe Disease Infantile-Onset", "Neuronopathic Gaucher Disease", "Wolman Disease" ], "interventions": [ { "name": "There is no intervention", "type": "OTHER" } ], "intervention_types": [ "OTHER" ], "sponsor": "University of California, San Francisco", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "64 Years", "sex": "ALL", "summary": "Up to 64 Years" }, "enrollment_count": 250, "start_date": "2022-05-31", "completion_date": "2050-05-31", "has_results": false, "last_update_posted_date": "2026-04-08", "last_synced_at": "2026-05-21T23:33:57.124Z", "location_count": 1, "location_summary": "San Francisco, California", "locations": [ { "city": "San Francisco", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT05619900" }, { "nct_id": "NCT01858103", "title": "BMN 110 US Expanded Access Program", "overall_status": "APPROVED_FOR_MARKETING", "study_type": "EXPANDED_ACCESS", "phases": [], "conditions": [ "Mucopolysaccharidosis IVA", "Morquio A Syndrome", "MPS IVA" ], "interventions": [ { "name": "BMN 110", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "BioMarin Pharmaceutical", "sponsor_class": "INDUSTRY", "healthy_volunteers": null, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": null, "start_date": null, "completion_date": null, "has_results": false, "last_update_posted_date": "2014-04-02", "last_synced_at": "2026-05-21T23:33:57.124Z", "location_count": 24, "location_summary": "Birmingham, Alabama • Little Rock, Arkansas • Oakland, California + 21 more", "locations": [ { "city": "Birmingham", "state": "Alabama" }, { "city": "Little Rock", "state": "Arkansas" }, { "city": "Oakland", "state": "California" }, { "city": "Orange", "state": "California" }, { "city": "Aurora", "state": "Colorado" } ], "official_url": "https://clinicaltrials.gov/study/NCT01858103" }, { "nct_id": "NCT05846009", "title": "A First-in-human Single and Repeated Dose Escalation Study of SAR442501 in Healthy Adults Subjects", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Osteochondrodysplasia" ], "interventions": [ { "name": "SAR442501", "type": "DRUG" }, { "name": "Placebo", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Sanofi", "sponsor_class": "INDUSTRY", "healthy_volunteers": true, "eligibility": { "minimum_age": "18 Years", "maximum_age": "45 Years", "sex": "ALL", "summary": "18 Years to 45 Years" }, "enrollment_count": 76, "start_date": "2020-11-18", "completion_date": "2022-01-12", "has_results": false, "last_update_posted_date": "2024-08-27", "last_synced_at": "2026-05-21T23:33:57.124Z", "location_count": 1, "location_summary": "Newark, New Jersey", "locations": [ { "city": "Newark", "state": "New Jersey" } ], "official_url": "https://clinicaltrials.gov/study/NCT05846009" }, { "nct_id": "NCT01515956", "title": "Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Mucopolysaccharidosis IVA", "Morquio A Syndrome", "MPS IVA" ], "interventions": [ { "name": "BMN 110", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "BioMarin Pharmaceutical", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "5 Years", "sex": "ALL", "summary": "Up to 5 Years" }, "enrollment_count": 15, "start_date": "2011-10", "completion_date": "2016-02", "has_results": true, "last_update_posted_date": "2017-08-10", "last_synced_at": "2026-05-21T23:33:57.124Z", "location_count": 2, "location_summary": "Oakland, California • Manhasset, New York", "locations": [ { "city": "Oakland", "state": "California" }, { "city": "Manhasset", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT01515956" }, { "nct_id": "NCT03655223", "title": "Early Check: Expanded Screening in Newborns", "overall_status": "ENROLLING_BY_INVITATION", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Spinal Muscular Atrophy", "Fragile X Syndrome", "Fragile X - Premutation", "Duchenne Muscular Dystrophy", "Hyperinsulinemic Hypoglycemia, Familial 1", "Diabetes Mellitus", "Adrenoleukodystrophy, Neonatal", "Medium-chain Acyl-CoA Dehydrogenase Deficiency", "Very Long Chain Acyl Coa Dehydrogenase Deficiency", "Beta-ketothiolase Deficiency", "Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency", "Primary Hyperoxaluria Type 1", "Congenital Bile Acid Synthesis Defect Type 2", "Pyridoxine-Dependent Epilepsy", "Hereditary Fructose Intolerance", "Hypophosphatasia", "Hyperargininemia", "Mucopolysaccharidosis Type 6", "Argininosuccinic Aciduria", "Citrullinemia, Type I", "Wilson Disease", "Maple Syrup Urine Disease, Type 1A", "Maple Syrup Urine Disease, Type 1B", "Biotinidase Deficiency", "Neonatal Severe Primary Hyperparathyroidism", "Intrinsic Factor Deficiency", "Usher Syndrome Type 1D/F Digenic (Diagnosis)", "Cystic Fibrosis", "Stickler Syndrome Type 2", "Stickler Syndrome Type 1", "Alport Syndrome, Autosomal Recessive", "Alport Syndrome, X-Linked", "Carbamoyl Phosphate Synthetase I Deficiency Disease", "Carnitine Palmitoyl Transferase 1A Deficiency", "Carnitine Palmitoyltransferase II Deficiency", "Cystinosis", "Chronic Granulomatous Disease", "Cerebrotendinous Xanthomatoses", "Maple Syrup Urine Disease, Type 2", "Severe Combined Immunodeficiency Due to DCLRE1C Deficiency", "Thyroid Dyshormonogenesis 6", "Thyroid Dyshormonogenesis 5", "Supravalvar Aortic Stenosis", "Factor X Deficiency", "Hemophilia A", "Hemophilia B", "Tyrosinemia, Type I", "Fructose 1,6 Bisphosphatase Deficiency", "Glycogen Storage Disease Type I", "G6PD Deficiency", "Glycogen Storage Disease II", "Galactokinase Deficiency", "Mucopolysaccharidosis Type IV A", "Galactosemias", "Guanidinoacetate Methyltransferase Deficiency", "Agat Deficiency", "Glutaryl-CoA Dehydrogenase Deficiency", "Gtp Cyclohydrolase I Deficiency", "Hyperinsulinism-Hyperammonemia Syndrome", "Primary Hyperoxaluria Type 2", "3-Hydroxyacyl-CoA Dehydrogenase Deficiency", "Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency", "Mitochondrial Trifunctional Protein Deficiency", "Sickle Cell Disease", "Beta-Thalassemia", "Holocarboxylase Synthetase Deficiency", "3-Hydroxy-3-Methylglutaric Aciduria", "Primary Hyperoxaluria Type 3", "Hermansky-Pudlak Syndrome 1", "Hermansky-Pudlak Syndrome 4", "Apparent Mineralocorticoid Excess", "HSDB", "CBAS1", "Mucopolysaccharidosis Type 2", "Mucopolysaccharidosis Type 1", "Severe Combined Immunodeficiency, X Linked", "Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency", "Diabetes Mellitus, Permanent Neonatal", "Isovaleric Acidemia", "Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder)", "Jervell and Lange-Nielsen Syndrome 2", "Hyperinsulinemic Hypoglycemia, Familial, 2", "Diabetes Mellitus, Permanent Neonatal, With Neurologic Features", "Jervell and Lange-Nielsen Syndrome 1", "Lysosomal Acid Lipase Deficiency", "CblF", "3-Methylcrotonyl CoA Carboxylase 1 Deficiency", "3-Methylcrotonyl CoA Carboxylase 2 Deficiency", "Waardenburg Syndrome Type 2A", "Methylmalonic Aciduria cblA Type", "Methylmalonic Aciduria cblB Type", "Methylmalonic Aciduria and Homocystinuria Type cblC", "MAHCD", "Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency", "Congenital Disorder of Glycosylation Type 1B", "Mthfr Deficiency", "Methylcobalamin Deficiency Type Cbl G (Disorder)", "Methylcobalamin Deficiency Type cblE", "Usher Syndrome, Type 1B", "N-acetylglutamate Synthase Deficiency", "Ornithine Transcarbamylase Deficiency", "Phenylketonurias", "Waardenburg Syndrome Type 1", "Congenital Hypothyroidism", "Propionic Acidemia", "Usher Syndrome, Type 1F", "Pancreatic Agenesis 1", "Hereditary Hypophosphatemic Rickets", "Glycogen Storage Disease IXB", "Glycogen Storage Disease IXC", "MOWS", "Epilepsy, Early-Onset, Vitamin B6-Dependent", "Pyridoxal Phosphate-Responsive Seizures", "Pituitary Hormone Deficiency, Combined, 1", "Ptsd", "Dihydropteridine Reductase Deficiency", "Severe Combined Immunodeficiency Due to RAG1 Deficiency", "Severe Combined Immunodeficiency Due to RAG2 Deficiency", "Retinoblastoma", "Multiple Endocrine Neoplasia Type 2B", "Pseudohypoaldosteronism, Type I", "Liddle Syndrome", "Biotin-Responsive Basal Ganglia Disease", "SCD", "DIAR1", "GSD1C", "Acrodermatitis Enteropathica", "Thyroid Dyshormonogenesis 1", "Riboflavin Transporter Deficiency", "Waardenburg Syndrome, Type 2E", "SRD", "Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency", "Barth Syndrome", "Adrenocorticotropic Hormone Deficiency", "Transcobalamin II Deficiency", "Thyroid Dyshormonogenesis 3", "Segawa Syndrome, Autosomal Recessive", "Autosomal Recessive Nonsyndromic Hearing Loss", "Thyroid Dyshormonogenesis 2A", "Congenital Isolated Thyroid Stimulating Hormone Deficiency", "Hypothyroidism Due to TSH Receptor Mutations", "Usher Syndrome Type 1C", "Usher Syndrome Type 1G (Diagnosis)", "Von Willebrand Disease, Type 3", "Combined Immunodeficiency Due to ZAP70 Deficiency", "Adenine Phosphoribosyltransferase Deficiency", "Metachromatic Leukodystrophy", "Canavan Disease", "Menkes Disease", "Carbonic Anhydrase VA Deficiency", "Developmental and Epileptic Encephalopathy 2", "17 Alpha-Hydroxylase Deficiency", "Smith-Lemli-Opitz Syndrome", "Krabbe Disease", "Glutathione Synthetase Deficiency", "Mucopolysaccharidosis Type 7", "Rett Syndrome", "Molybdenum Cofactor Deficiency, Type A", "Niemann-Pick Disease, Type C1", "Niemann-Pick Disease Type C2", "Ornithine Aminotransferase Deficiency", "3-Phosphoglycerate Dehydrogenase Deficiency", "Leber Congenital Amaurosis 2", "Dravet Syndrome", "Mucopolysaccharidosis Type 3 A", "Ornithine Translocase Deficiency", "Carnitine-acylcarnitine Translocase Deficiency", "Glucose Transporter Type 1 Deficiency Syndrome", "Creatine Transporter Deficiency", "Niemann-Pick Disease Type A", "Pitt Hopkins Syndrome", "Tuberous Sclerosis 1", "Tuberous Sclerosis 2", "Ataxia With Isolated Vitamin E Deficiency", "Angelman Syndrome", "Prader-Willi Syndrome", "Homocystinuria", "Permanent Neonatal Diabetes Mellitus", "Transient Neonatal Diabetes Mellitus", "Factor VII Deficiency", "Glycogen Storage Disease Type IXA1", "Glycogen Storage Disease, Type IXA2", "Glycogen Storage Disease IC", "Glycogen Storage Disease Type IB", "Central Hypoventilation Syndrome With or Without Hirschsprung Disease" ], "interventions": [ { "name": "Confirmatory Testing", "type": "DIAGNOSTIC_TEST" } ], "intervention_types": [ "DIAGNOSTIC_TEST" ], "sponsor": "RTI International", "sponsor_class": "OTHER", "healthy_volunteers": true, "eligibility": { "minimum_age": "1 Day", "maximum_age": "31 Days", "sex": "ALL", "summary": "1 Day to 31 Days" }, "enrollment_count": 30000, "start_date": "2018-10-15", "completion_date": "2025-12-31", "has_results": false, "last_update_posted_date": "2025-04-04", "last_synced_at": "2026-05-21T23:33:57.124Z", "location_count": 1, "location_summary": "Research Triangle Park, North Carolina", "locations": [ { "city": "Research Triangle Park", "state": "North Carolina" } ], "official_url": "https://clinicaltrials.gov/study/NCT03655223" }, { "nct_id": "NCT01707433", "title": "Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Mucopolysaccharidosis IV A", "Mucopolysaccharidosis VI" ], "interventions": [ { "name": "Enzyme testing", "type": "OTHER" } ], "intervention_types": [ "OTHER" ], "sponsor": "Children's Hospitals and Clinics of Minnesota", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "21 Years", "sex": "ALL", "summary": "Up to 21 Years" }, "enrollment_count": 22, "start_date": "2012-10", "completion_date": "2015-06", "has_results": false, "last_update_posted_date": "2015-08-04", "last_synced_at": "2026-05-21T23:33:57.124Z", "location_count": 2, "location_summary": "Minneapolis, Minnesota • Saint Paul, Minnesota", "locations": [ { "city": "Minneapolis", "state": "Minnesota" }, { "city": "Saint Paul", "state": "Minnesota" } ], "official_url": "https://clinicaltrials.gov/study/NCT01707433" }, { "nct_id": "NCT06064656", "title": "A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Fibrodysplasia Ossificans Progressiva (FOP)" ], "interventions": [ { "name": "Non-Interventional", "type": "OTHER" } ], "intervention_types": [ "OTHER" ], "sponsor": "Regeneron Pharmaceuticals", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 131, "start_date": "2023-06-29", "completion_date": "2023-09-29", "has_results": false, "last_update_posted_date": "2025-07-03", "last_synced_at": "2026-05-21T23:33:57.124Z", "location_count": 1, "location_summary": "Tarrytown, New York", "locations": [ { "city": "Tarrytown", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT06064656" }, { "nct_id": "NCT03971227", "title": "Clinical Evaluation of Acuity 200 (Fluoroxyfocon A) RGP Contact Lens", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "NA" ], "conditions": [ "Refractive Errors" ], "interventions": [ { "name": "Acuity 200 (Fluoroxyfocon A) contact lens", "type": "DEVICE" }, { "name": "Acuity 100 (Hexafocon A) contact lens", "type": "DEVICE" } ], "intervention_types": [ "DEVICE" ], "sponsor": "Acuity Polymers, Inc.", "sponsor_class": "INDUSTRY", "healthy_volunteers": true, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 74, "start_date": "2019-04-01", "completion_date": "2020-03-19", "has_results": false, "last_update_posted_date": "2020-05-12", "last_synced_at": "2026-05-21T23:33:57.124Z", "location_count": 3, "location_summary": "Honolulu, Hawaii • Edina, Minnesota • Pittsford, New York", "locations": [ { "city": "Honolulu", "state": "Hawaii" }, { "city": "Edina", "state": "Minnesota" }, { "city": "Pittsford", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT03971227" }, { "nct_id": "NCT02294877", "title": "A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Mucopolysaccharidosis IV Type A", "Morquio A Syndrome", "MPS IVA" ], "interventions": [ { "name": "Vimizim® (elosulfase alfa)", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "BioMarin Pharmaceutical", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 418, "start_date": "2014-09", "completion_date": "2024-02-21", "has_results": false, "last_update_posted_date": "2024-03-05", "last_synced_at": "2026-05-21T23:33:57.124Z", "location_count": 26, "location_summary": "Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 22 more", "locations": [ { "city": "Phoenix", "state": "Arizona" }, { "city": "Little Rock", "state": "Arkansas" }, { "city": "Los Angeles", "state": "California" }, { "city": "Los Angeles", "state": "California" }, { "city": "Orange", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT02294877" } ] }