298 completed. 298 Phase 3, 44 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 298 | Completed 298 |
| Phase 2 | 44 | Completed 44 |
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 |
|---|---|---|---|---|---|---|
| Phase 3 Evaluation of Efficacy and Safety of AR882 in Gout Patients (AR882-301) AR882 50 mg, AR882 75 mg, Placebo - Gout, Arthritis, Gouty | Phase 3 | Completed | Arthrosi Therapeutics | 866 | 2025-03-11 | 2026-09-28 |
| A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Clinical Study Evaluating the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia SHR-1918, SHR-1918 placebo - Homozygous Familial Hypercholesterolemia | Phase 3 | Completed | Beijing Suncadia Pharmaceuticals Co., Ltd | 55 | 2025-02-13 | 2025-12-15 |
| REVEAL - Research With I-124 EVuzamitide to Elucidate Cardiac AmyLoidosis I-124 evuzamitide - Cardiac Amyloidosis | Phase 3 | Completed | Sharmila Dorbala | 204 | 2025-01-14 | 2026-04-30 |
| A Study of the Safety of Mibavademab in Pediatric and Adult Participants Switching From Metreleptin to Mibavademab for the Treatment of Generalized Lipodystrophy (GLD) mibavademab - Generalized Lipodystrophy | Phase 3 | Completed | Regeneron Pharmaceuticals | 9 | 2024-12-16 | 2026-07-29 |
| Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency Pegzilarginase - Arginase 1 Deficiency | Phase 3 | Completed | Immedica Pharma AB | 3 | 2024-08-30 | 2026-05-22 |
| Phase 3 Evaluation of Efficacy and Safety of AR882 in Gout Patients (AR882-302) AR882 50 mg, AR882 75 mg, Placebo - Gout, Arthritis, Gouty | Phase 3 | Completed | Arthrosi Therapeutics | 818 | 2024-06-26 | 2026-10-14 |
| Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies Combination Therapy, Monotherapy obicetrapib, Monotherapy ezetimibe - Dyslipidemias, Hypercholesterolemia | Phase 3 | Completed | NewAmsterdam Pharma | 407 | 2024-03-01 | 2025-10-08 |
| Effect of Fixed Combination Citicoline Homotaurine and Pyrroloquinoline Quinone on Pattern-electroretinogram in Glaucoma Citicoline 500 mg plus Homotaurine 50 mg plus Pyrroloquinoline quinone (Neuprozin Mito® ) - Glaucoma, Neuroprotection | Phase 3 | Completed | Fondazione IRCCS Policlinico San Matteo di Pavia | 40 | 2024-01-27 | 2025-05-01 |
| A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day - Achondroplasia | Phase 3 | Completed | QED Therapeutics, a BridgeBio company | 114 | 2023-11-10 | 2026-03-17 |
| A Study to Evaluate Efficacy and Safety of Qingzhu Granules in the Treatment of Acute Gouty Arthritis Qingzhu Granules, Qingzhu Granules Placebo - Gouty Arthritis | Phase 3 | Completed | Tasly Pharmaceutical Group Co., Ltd | 472 | 2023-11-07 | 2026-02-27 |
| Phase 2b/3 Study to Assess ABP-671 a Novel URAT1 Inhibitor in Participants With Gout ABP-671, Allopurinol, Placebo - Gout | Phase 2 / Phase 3 | Completed | Atom Therapeutics Co., Ltd | 300 | 2023-08-11 | 2025-12-22 |
| A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Hypercholesterolemia (MK-0616-013) CORALreef Lipids Enlicitide Decanoate, Placebo - Hypercholesterolemia, Familial Hypercholesterolemia | Phase 3 | Completed | Merck Sharp & Dohme LLC | 2912 | 2023-08-10 | 2026-09-24 |
| A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Heterozygous Familial Hypercholesterolemia (MK-0616-017/CORALreef HeFH) Enlicitide Decanoate, Placebo - Hypercholesterolemia, Familial Hypercholesterolemia | Phase 3 | Completed | Merck Sharp & Dohme LLC | 303 | 2023-08-08 | 2026-02-24 |
| Tigulixostat, Phase 3 Study, Allopurinol Controlled in Gout Patients Tigulixostat, Allopurinol, Placebo - Gout, Hyperuricemia | Phase 3 | Completed | LG Chem | 2202 | 2023-03-30 | 2025-07-22 |
| A Therapeutic Confirmatory Study of Epaminurad Versus Febuxostat in Gout Patients Epaminurad 6 mg, Epaminurad 9 mg, Febuxostat 40 mg - Gout | Phase 3 | Completed | JW Pharmaceutical | 612 | 2023-03-21 | 2026-05-04 |
| Role of Vitamin E in Gaucher Disease Patients Vitamin E - Gaucher Disease | Phase 3 | Completed | Ain Shams University | 40 | 2023-03-03 | 2024-01-18 |
| Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia Evinacumab - Homozygous Familial Hypercholesterolemia | Phase 3 | Completed | Daniel Gaudet | 10 | 2023-02-21 | 2025-03-25 |
| Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis [18F]florbetaben - Cardiac Amyloidosis, AL Amyloidosis | Phase 3 | Completed | Lantheus Germany GmbH | 244 | 2023-01-13 | 2026-10-26 |
| A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease Velaglucerase Alfa - Gaucher Disease | Phase 3 | Completed | Takeda | 20 | 2023-01-03 | 2025-06-26 |
| Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease Recombinant human alpha galactosidase A (agalsidase beta), Recombinant human alpha-galactosidase A (agalsidase beta) - Fabry Disease | Phase 3 | Completed | Bio Sidus SA | 20 | 2022-12-13 | 2026-02-18 |
Showing 20 of 298. Sign in to see all of them.
Hyperlipoproteinemia Type II (38) · Gout (36) · Hypercholesterolemia (24) · Homozygous Familial Hypercholesterolemia (23) · Gaucher Disease (21) · Fabry Disease (18) · Amyloid Neuropathies, Familial (14) · Phenylketonurias (13) · Amyloidosis (11) · Hyperlipidemias (11) · Metabolic Diseases (11) · Genetic Diseases, Inborn (10) · Lysosomal Storage Diseases (10) · Rare Diseases (10)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (298) · Nutritional and Metabolic Diseases (298) · Genetic Diseases, Inborn (288) · Metabolic Diseases (287) · Lipid Metabolism Disorders (118) · Lipid Metabolism, Inborn Errors (115) · Nervous System Diseases (115) · Brain Diseases (93) · Central Nervous System Diseases (93) · Brain Diseases, Metabolic (87) · Brain Diseases, Metabolic, Inborn (87) · Lysosomal Storage Diseases (73) · Dyslipidemias (62) · Hyperlipoproteinemias (58)
Allopurinol (12) · Ezetimibe (11) · Febuxostat (11) · Atorvastatin (8) · sapropterin (8) · Glucosylceramidase (7) · Rosuvastatin Calcium (7) · burosumab (6) · lesinurad (6) · migalastat (6) · GAA protein, human (5) · agalsidase alfa (5) · agalsidase beta (5) · canakinumab (5) · eliglustat (5)
BioMarin Pharmaceutical (15) · Genzyme, a Sanofi Company (14) · Pfizer (13) · Shire (13) · Amgen (11) · Merck Sharp & Dohme LLC (9) · Novartis Pharmaceuticals (9) · Organon and Co (9) · Regeneron Pharmaceuticals (9) · Alnylam Pharmaceuticals (8) · Takeda (8) · Amicus Therapeutics (7) · Ardea Biosciences, Inc. (6) · AstraZeneca (5) · Chiesi Farmaceutici S.p.A. (4)
United States (184) · Canada (87) · United Kingdom (83) · France (82) · Germany (68) · Italy (68) · Spain (64) · Netherlands (52) · Australia (51) · Brazil (39) · Japan (38) · Belgium (36) · South Africa (29) · Argentina (28) · Taiwan (28)
Chris Storgard, MD (4) · Robert Evans, PharmD (3) · Allan M Lund, MD (2) · Debra Lounsbury (2) · Heda Amarante, M.D (2) · Hyungjin Cho, MD (2) · Jeff Packman (2)
PMID 33874971, PMID 21353107, PMID 22052584, PMID 24144944, PMID 27578134, PMID 27839979, PMID 33990512, PMID 35835008, PMID 39151033, PMID 39847314, PMID 40411344, PMID 18600509, PMID 19775921, PMID 21401833, PMID 22087637, PMID 22500948, PMID 24014831, PMID 24122883, PMID 24286509, PMID 24588936, PMID 25614280, PMID 26053270, PMID 27494299, PMID 27657681, PMID 30558645, PMID 31203212, PMID 31353964, PMID 31889231, PMID 32063397, PMID 32107748, PMID 32524297, PMID 32609313, PMID 32994552, PMID 33070419, PMID 33527139, PMID 34355354, PMID 34826353, PMID 34923848, PMID 35799473, PMID 35916348, PMID 35961250, PMID 36520008, PMID 36715498, PMID 36854566, PMID 37415957, PMID 37434378, PMID 37915038, PMID 37943223, PMID 38332115, PMID 38548524
2015 (7), 2018 (7), 2017 (6), 2008 (5), 2009 (5), 2014 (5), 2019 (4), 2023 (4), 2005 (3), 2016 (3), 2004 (1), P50AR054086 (1), R01NS051306 (1)