326 completed. 326 Phase 1, 109 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 326 | Completed 326 |
| Phase 2 | 109 | Completed 109 |
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 |
|---|---|---|---|---|---|---|
| The Food Effect on Pharmacokinetics of Fixed Dose Combination of Gemigliptin/Dapagliflozin/Metformin in Healthy Volunteers Fixed-dose combination of gemigliptin/dapagliflozin/metformin 50/10/1000 mg - Healthy, Fasting | Phase 1 | Completed | LG Chem | 28 | 2026-06-11 | 2026-09-10 |
| Test-retest Study With [18F]FBB in Cardiac Amyloidosis Florbetaben F18 - Cardiac Amyloidosis | Phase 1 | Completed | Lantheus Germany GmbH | 15 | 2025-10-21 | 2026-10-23 |
| A Study to Investigate the Effect of Food on Balcinrenone/Dapagliflozin Pharmacokinetics in Fed and Fasted State and Pharmacokinetics of Balcinrenone When Dosed With a P-gp Inhibitor in Healthy Participants. balcinrenone/ dapagliflozin, quinidine - Healthy Participants | Phase 1 | Completed | AstraZeneca | 14 | 2025-05-20 | 2025-08-12 |
| A Bioequivalence Study of Two Different Dosage Form of Genakumab Genakumab Injection (T, Solution for injection), Genakumab for Injection (R, Powder for solution for injection) - Gouty Arthritis (GA) | Phase 1 | Completed | Changchun GeneScience Pharmaceutical Co., Ltd. | 178 | 2024-06-24 | 2025-02-11 |
| Recapturing Immune Tolerance to Pegloticase for the Management of Tophaceous Gout Rituximab - Tophaceous Gout | Phase 1 | Completed | University of California, Los Angeles | 2 | 2024-04-10 | 2025-11-20 |
| A Study to Investigate the Pharmacokinetic/Pharmacodynamic Characteristics of IBI128(A New Xanthine Oxidase Inhibitor) dose-5 group, dose-1 group, dose-2 group - Gout Arthritis | Phase 1 | Completed | Innovent Biologics (Suzhou) Co. Ltd. | 30 | 2024-03-05 | 2024-03-22 |
| A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Oral ABP-745 Administration in Healthy Volunteers ABP-745, Placebo - Gout, Acute Gout Flare | Phase 1 | Completed | Atom Therapeutics Co., Ltd | 90 | 2024-01-23 | 2025-08-17 |
| Study Comparing Once Daily Dose of 900mg of TETA 4HCL Against Cuprior® (450mg Trientine Base, Twice Daily). 900mg TETA 4HCl Once Daily Formulation, 900mg TETA 4HCl Cuprior® - Wilson's Disease | Phase 1 | Completed | Orphalan | 26 | 2024-01-16 | 2026-08-26 |
| PK Effects of SHR4640 on Repaglinide and Midazolam, and the Impact of SHR4640 on QT Interval repaglinide; midazolam; SHR4640, repaglinide; midazolam; SHR4640; SHR4640 placebo, repaglinide; midazolam; SHR4640 placebo - Gout and Hyperuricemia | Phase 1 | Completed | Jiangsu HengRui Medicine Co., Ltd. | 24 | 2024-01-04 | 2024-03-06 |
| Effect of Tigulixostat on the Pharmacokinetics of Theophylline Tigulixostat, Theophylline - Gout, Hyperuricemia | Phase 1 | Completed | LG Chem | 24 | 2023-10-31 | 2024-01-03 |
| PK and PD Study of NPI-001 and Cysteamine Bitartrate Cysteamine Bitartrate, N-Acetylcysteine Amide - Cystinosis | Phase 1 / Phase 2 | Completed | Nacuity Pharmaceuticals, Inc. | 8 | 2023-10-29 | 2026-10-13 |
| Open Label PK, PD and DDI of Dotinurad and Allopurinol in Gout Patients With Hyperuricemia dotinurad, dotinurad + allopurinol, allopurinol - Gout, Hyperuricemia | Phase 1 / Phase 2 | Completed | Urica Therapeutics Inc. | 20 | 2023-10-04 | 2026-06-10 |
| Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD). Promoting Resilience in Stress Management (PRISM), Clinical-focused narrative (CFN) - Mitochondrial Diseases, Mitochondrial Myopathies | Phase 1 / Phase 2 | Completed | Children's Hospital of Philadelphia | 16 | 2023-09-22 | 2024-11-29 |
| A First-in-Human Phase 1 Study of Plasmalogen Precursor PPI-1011 in Healthy Adult Volunteers to Assess Safety, Tolerability, and Pharmacokinetics PPI-1011, Placebo - Rhizomelic Chondrodysplasia Punctata | Phase 1 | Completed | MED-LIFE DISCOVERIES LP | 56 | 2023-06-28 | 2026-10-09 |
| A VSA003 Phase 1 Study in Chinese Adult Healthy Volunteers VSA003, 0.9% NaCl - Dyslipidemias, Familial Hypercholesterolemia | Phase 1 | Completed | Visirna Therapeutics HK Limited | 36 | 2023-06-01 | 2025-09-12 |
| SAD,MAD and Food Effect Study of SAP-001 Tablets in Chinese Subjects SAP-001, Placebo - Gout, Hyperuricemia | Phase 1 | Completed | Shanton Pharma Co., Ltd. | 70 | 2023-05-29 | 2024-11-12 |
| A Study to Compare the Relative Bioavailability of Two Iberdomide (CC-220) Formulations and to Assess The Effect Of Food on the Drug Levels of Iberdomide in Healthy Adult Participants Iberdomide - Healthy Volunteers | Phase 1 | Completed | Bristol-Myers Squibb | 32 | 2023-05-18 | 2024-07-26 |
| Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia Ilofotase Alfa, 0.8 mg/kg, Ilofotase Alfa, 3.2 mg/kg - Hypophosphatasia | Phase 1 / Phase 2 | Completed | AM-Pharma | 12 | 2023-05-15 | 2025-02-27 |
| A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers verenafusp alfa 6 mg/kg, verenafusp alfa 9 mg/kg, verenafusp alfa 12 mg/kg - Mucopolysaccharidosis Type II, Metabolic Diseases | Phase 1 | Completed | AO GENERIUM | 18 | 2023-04-06 | 2024-07-03 |
| A Study to Evaluate the Safety, Tolerability, PK, and PD Properties of PRX-115 in Adult Volunteers With Elevated Uric Acid Levels PRX-115, Placebo - Gout | Phase 1 | Completed | Protalix | 64 | 2023-03-23 | 2025-02-11 |
Showing 20 of 326. Sign in to see all of them.
Lecithin Cholesterol Acyltransferase Deficiency (47) · Gout (44) · Hyperuricemia (22) · Gaucher Disease (18) · Mucopolysaccharidosis I (17) · Lysosomal Storage Diseases (15) · Fabry Disease (14) · Glycogen Storage Disease Type II (12) · Hyperlipoproteinemia Type II (12) · Mucopolysaccharidosis II (12) · Multiple Acyl Coenzyme A Dehydrogenase Deficiency (11) · Acquired Immunodeficiency Syndrome (9) · Fasting (9) · HIV Infections (9)
Nutritional and Metabolic Diseases (326) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (325) · Metabolic Diseases (320) · Genetic Diseases, Inborn (318) · Nervous System Diseases (134) · Lipid Metabolism Disorders (125) · Lipid Metabolism, Inborn Errors (125) · Brain Diseases (102) · Central Nervous System Diseases (102) · Brain Diseases, Metabolic (99) · Brain Diseases, Metabolic, Inborn (99) · Lysosomal Storage Diseases (92) · Carbohydrate Metabolism, Inborn Errors (69) · Musculoskeletal Diseases (65)
Ganciclovir (9) · eliglustat (6) · ruzinurad (6) · verinurad (6) · burosumab (5) · migalastat (5) · nitisinone (5) · 2-Hydroxypropyl-beta-cyclodextrin (4) · Acetaminophen (4) · larazotide acetate (4) · valanafusp alpha (4) · Allopurinol (3) · Cysteamine (3) · Doxycycline (3) · Hydrocortisone (3)
IPCA Laboratories Ltd. (12) · Par Pharmaceutical, Inc. (11) · Alexion Pharmaceuticals, Inc. (9) · Ardea Biosciences, Inc. (8) · Shire (8) · BioMarin Pharmaceutical (7) · Dr. Reddy's Laboratories Limited (7) · Genzyme, a Sanofi Company (7) · LG Chem (7) · National Institute of Allergy and Infectious Diseases (NIAID) (7) · Amicus Therapeutics (6) · National Center for Research Resources (NCRR) (6) · ArmaGen, Inc (5) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (5) · Protalix (5)
United States (173) · United Kingdom (44) · Canada (26) · Germany (24) · France (20) · Australia (18) · India (16) · China (13) · Brazil (10) · Netherlands (10) · Spain (10) · Italy (9) · Japan (8) · Israel (7) · New Zealand (7)
J. Hall, MD (4) · Forbes D Porter, M.D. (3) · Karl Elmo Anderson (3) · Patrice Rioux, MD PhD (3) · Amy Zhang, PhD (2) · Celeste Decker, MD (2) · Dr. Hardik Dave, M.B.B.S. (2) · Dr. Rupesh Vala, M.B.B.S. (2) · Dr. Suhas Khandave, M.D. (2)
PMID 25919461, PMID 29688815, PMID 29976218, PMID 33332761, PMID 33980287, PMID 35022448, PMID 35835061, PMID 37171121, PMID 38101904, PMID 38776275, PMID 41388232, PMID 14605787, PMID 14662594, PMID 15923132, PMID 16278898, PMID 17482283, PMID 18201934, PMID 18256395, PMID 18473686, PMID 18752978, PMID 19194885, PMID 19277123, PMID 19775921, PMID 20299511, PMID 20497190, PMID 20512995, PMID 20652413, PMID 22134099, PMID 22397652, PMID 22547151, PMID 22775199, PMID 22794786, PMID 23348766, PMID 23494656, PMID 23497636, PMID 23570273, PMID 23581634, PMID 23713767, PMID 23860343, PMID 24094767, PMID 24206527, PMID 24296492, PMID 24443373, PMID 24569459, PMID 25281031, PMID 25421091, PMID 25758611, PMID 25889044, PMID 26000324, PMID 26048034
2015 (3), 2016 (2), U54NS078059 (2), 2009 (1), 2014 (1), 2017 (1), 2018 (1), K23NS087151 (1), M01RR001070 (1), P01HL059412 (1), R21DK077415 (1), U01HD090845 (1), U54DK083908 (1), U54NS065768 (1), UL1TR000457-06 (1)