1 active not recruiting, 1 completed. 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 1 | Completed 1 |
Follow Mucopolysaccharidosis Type 1: new trials by email Literature Review of Mucopolysaccharidosis Type 1 trials Research Report on Mucopolysaccharidosis Type 1
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 |
|---|---|---|---|---|---|---|
| Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients Laronidase, Antihistamine, Antipyretic - Mucopolysaccharidosis Type 1 | Phase 3 | Completed | Cinnagen | 12 | 2022-09-10 | 2024-08-06 |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 |
Mucopolysaccharidosis I (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1) · 3-methylcrotonyl CoA carboxylase 1 deficiency (1) · 3-methylcrotonyl CoA carboxylase 2 deficiency (1) · Acidemia, isovaleric (1) · Acrodermatitis enteropathica (1) · Adenine phosphoribosyltransferase deficiency (1) · Adrenal hyperplasia, congenital, type 5 (1) · Adrenocorticotropic hormone deficiency (1) · Amaurosis congenita of Leber, type 2 (1) · Anemia, Sickle Cell (1) · Angelman Syndrome (1) · Aortic Stenosis, Supravalvular (1) · Arginine-Glycine Amidinotransferase Deficiency (1)
Carbohydrate Metabolism, Inborn Errors (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Connective Tissue Diseases (2) · Genetic Diseases, Inborn (2) · Lysosomal Storage Diseases (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Mucinoses (2) · Mucopolysaccharidoses (2) · Nutritional and Metabolic Diseases (2) · Skin and Connective Tissue Diseases (2) · Abnormalities, Multiple (1) · Albinism (1) · Albinism, Oculocutaneous (1) · Amino Acid Metabolism, Inborn Errors (1)
Antipyretics (1) · Histamine Antagonists (1) · Iduronidase (1)
Cinnagen (1) · RTI International (1)
Iran (1) · United States (1)
Ali Rabani, Professor (1) · Curt Scharfe, MD, PhD (1)