13 terminated. 13 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 4 | 13 | Terminated 13 |
Follow Metabolism, Inborn Errors: new trials by email Literature Review of Metabolism, Inborn Errors trials Research Report on Metabolism, Inborn Errors
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Cardiac Uptake of 18F Florbetapir in Patients Undergoing Chemotherapy F18 Florbetapir (amyvid) cardiac PET/CT imaging - Cardiac Amyloidosis | Phase 4 | Terminated | Medical College of Wisconsin | 4 | 2020-10-01 | 2026-07-16 |
| Impact of LDL-cholesterol Lowering on Platelet Activation Evolocumab - Familial Hypercholesterolemia | Phase 4 | Terminated | Columbia University | 4 | 2018-11-16 | 2021-04-01 |
| Assessing a Risk Model for G6PD Deficiency primaquine, chloroquine + primaquine - Malaria, Vivax, G6PD Deficiency | Phase 4 | Terminated | PATH | 54 | 2018-05-07 | 2021-11-12 |
| A Phase 4 Safety and Efficacy Study to Evaluate Lesinurad 200 mg in Participants With Gout and Renal Impairment Lesinurad, XOI, Placebo - Gout, Chronic Kidney Disease (CKD) | Phase 4 | Terminated | Ironwood Pharmaceuticals, Inc. | 242 | 2017-07-19 | 2021-11-04 |
| The Role of F-18 Florbetapir in the Early Detection of Cardiac Amyloidosis F-18 florbetapir - Cardiac Amyloidosis | Phase 4 | Terminated | The Cleveland Clinic | 12 | 2017-01-01 | 2021-07-27 |
| Synergistic Enteral Regimen for Treatment of the Gangliosidoses miglustat, Ketogenic Diet - GM1 Gangliosidoses, GM2 Gangliosidoses | Phase 4 | Terminated | University of Minnesota | 16 | 2015-12-22 | 2021-04-14 |
| Pharmacogenetics of SGLT2 Inhibitors Canagliflozin - Diabetes Mellitus, Gout | Phase 4 | Terminated | University of Maryland, Baltimore | 30 | 2015-06-01 | 2022-08-02 |
| A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease alglucosidase alfa - Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II) | Phase 4 | Terminated | Genzyme, a Sanofi Company | 4 | 2012-08-01 | 2016-01-18 |
| A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease Alglucosidase alfa - Pompe Disease | Phase 4 | Terminated | Genzyme, a Sanofi Company | 113 | 2012-03-01 | 2015-12-08 |
| A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants Laronidase - Mucopolysaccharidosis I, Hurler's Syndrome | Phase 4 | Terminated | Genzyme, a Sanofi Company | 2 | 2010-10-22 | 2024-02-20 |
| Immune Tolerance Induction Study Myozyme® (alglucosidase alfa) - Pompe Disease, Glycogen Storage Disease Type II (GSD-II) | Phase 4 | Terminated | Genzyme, a Sanofi Company | 4 | 2008-12-14 | 2022-04-07 |
| Study of the Effects of Fabrazyme Treatment on Lactation and Infants agalsidase beta - Fabry Disease, Alpha Galactosidase A Deficiency | Phase 4 | Terminated | Genzyme, a Sanofi Company | 7 | 2006-05-28 | 2024-04-10 |
| Morquio's Syndrome: a Case Study physiotherapeutic treatment - Morquio's Disease | Phase 4 | Terminated | Clínica de Hidroterapia e Recuperacao Funcional | 1 | 2006-01-01 | 2008-10-20 |
Glycogen Storage Disease Type II (3) · Amyloid Neuropathies, Familial (2) · Gout (2) · Diabetes Mellitus (1) · Fabry Disease (1) · Gangliosidoses (1) · Gangliosidoses, GM2 (1) · Gangliosidosis, GM1 (1) · Glucosephosphate Dehydrogenase Deficiency (1) · Hypercholesterolemia (1) · Hyperlipidemias (1) · Hyperlipoproteinemia Type II (1) · Hyperuricemia (1) · Lysosomal Storage Diseases (1) · Malaria, Vivax (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (13) · Genetic Diseases, Inborn (13) · Metabolic Diseases (13) · Nutritional and Metabolic Diseases (13) · Nervous System Diseases (7) · Carbohydrate Metabolism, Inborn Errors (6) · Lysosomal Storage Diseases (6) · Brain Diseases (5) · Brain Diseases, Metabolic (5) · Brain Diseases, Metabolic, Inborn (5) · Central Nervous System Diseases (5) · Lysosomal Storage Diseases, Nervous System (5) · Glycogen Storage Disease (3) · Lipid Metabolism Disorders (3)
GAA protein, human (3) · florbetapir (2) · Adrenal Cortex Hormones (1) · Allopurinol (1) · Canagliflozin (1) · Chloroquine (1) · Colchicine (1) · Diet, Ketogenic (1) · Febuxostat (1) · Iduronidase (1) · Primaquine (1) · agalsidase beta (1) · evolocumab (1) · lesinurad (1) · miglustat (1)
Genzyme, a Sanofi Company (5) · Clínica de Hidroterapia e Recuperacao Funcional (1) · Columbia University (1) · Ironwood Pharmaceuticals, Inc. (1) · Medical College of Wisconsin (1) · PATH (1) · The Cleveland Clinic (1) · University of Maryland, Baltimore (1) · University of Minnesota (1)
United States (9) · Austria (1) · Czechia (1) · Germany (1) · Hungary (1) · Israel (1) · Italy (1) · Poland (1) · Taiwan (1) · Thailand (1) · United Kingdom (1)
Chris Mueller, MD (1) · Henry Ginsberg, MD (1) · Jeanine R. Jarnes, PharmD (1) · Nan Wang, PhD (1) · Simeon I Taylor, MD, PhD (1) · Steven Nissen, MD (1) · Wael Jaber, MD (1)
PMID 25864295, PMID 28476546, PMID 29565424, PMID 31086307, PMID 34420548, PMID 36214004, PMID 36945579, PMID 36993363
2015 (1), R21DK105401 (1), U54NS065768 (1)