9 withdrawn. 9 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 4 | 9 | Withdrawn 9 |
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 |
|---|---|---|---|---|---|---|
| Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia methotrexate, rituximab, bortezomib - Hypophosphatasia | Phase 4 | Withdrawn | Alexion Pharmaceuticals, Inc. | 2026-07-29 | 2025-12-18 | |
| A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II) ELAPRASE, Rituximab, Methotrexate - Mucopolysaccharidosis (MPS), Hunter Syndrome | Phase 4 | Withdrawn | Takeda | 2023-02-28 | 2025-12-09 | |
| To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease agalsidase beta (GZ419828), agalsidase alfa - Fabry Disease | Phase 4 | Withdrawn | Sanofi | 2020-09-01 | 2023-04-07 | |
| Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic Disease asfotase alfa - Hypophosphatasia | Phase 4 | Withdrawn | Alexion Pharmaceuticals, Inc. | 2020-04-01 | 2020-06-04 | |
| Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease Elelyso 60 units/kg - Type 1 Gaucher Disease | Phase 4 | Withdrawn | Pfizer | 2019-07-31 | 2019-09-24 | |
| The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease Velaglucerase alfa - Gaucher Disease Type 1, Gaucher Disease Type 3 | Phase 4 | Withdrawn | Baylor Research Institute | 2015-07-01 | 2018-03-02 | |
| Efficacy Study of Daily Pro-Omega LDL for Low-Density Lipoprotein Cholesterol and Triglyceride Reduction Pro-Omega LDL, Placebo - Mixed Hyperlipoproteinemia | Phase 4 | Withdrawn | Nordic Pharma, USA | 2014-02-01 | 2015-12-14 | |
| CSPPT- Chronic Kidney Diseases Study Enalapril maleate and folic acid tablets, Enalapril maleate - Hypertension, Hyperhomocysteinemia | Phase 4 | Withdrawn | Shenzhen Ausa Pharmed Co.,Ltd | 2008-05-01 | 2016-01-20 | |
| A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency. Fabrazyme (agalsidase beta) - Fabry Disease | Phase 4 | Withdrawn | Genzyme, a Sanofi Company | 2006-04-01 | 2014-02-05 |
Fabry Disease (2) · Gaucher Disease (2) · Hypophosphatasia (2) · Hyperhomocysteinemia (1) · Hyperlipoproteinemia Type II (1) · Hypertension (1) · Mucopolysaccharidoses (1) · Mucopolysaccharidosis II (1) · Renal Insufficiency, Chronic (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (9) · Metabolic Diseases (9) · Nutritional and Metabolic Diseases (9) · Lipid Metabolism Disorders (5) · Lipid Metabolism, Inborn Errors (5) · Lysosomal Storage Diseases (5) · Nervous System Diseases (5) · Brain Diseases (4) · Brain Diseases, Metabolic (4) · Brain Diseases, Metabolic, Inborn (4) · Central Nervous System Diseases (4) · Lipidoses (4) · Lysosomal Storage Diseases, Nervous System (4)
Folic Acid (2) · Immunoglobulins, Intravenous (2) · Methotrexate (2) · Rituximab (2) · agalsidase beta (2) · Bortezomib (1) · Enalapril (1) · Glucosylceramidase (1) · agalsidase alfa (1) · asfotase alfa (1) · idursulfase (1)
Alexion Pharmaceuticals, Inc. (2) · Baylor Research Institute (1) · Genzyme, a Sanofi Company (1) · Nordic Pharma, USA (1) · Pfizer (1) · Sanofi (1) · Shenzhen Ausa Pharmed Co.,Ltd (1) · Takeda (1)
United States (4) · China (1)
David Becker, MD (1) · Fanfan Hou, MD (1) · Raphael Schiffmann, M.D.,M.H.Sc. (1) · Xin Xu, MD (1)
2019 (1)