1 active not recruiting, 1 completed, 1 no longer available. 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Completed 1 |
| Phase 2 | 1 | Completed 1 |
Follow Mucopolysaccharidosis Type 7: new trials by email Literature Review of Mucopolysaccharidosis Type 7 trials Research Report on Mucopolysaccharidosis Type 7
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 |
|---|---|---|---|---|---|---|
| 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 | |
| An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) UX003 - Mucopolysaccharidosis Type 7 | Phase 1 / Phase 2 | Completed | Ultragenyx Pharmaceutical Inc | 3 | 2013-11-01 | 2019-01-04 |
| An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient UX003 - Mucopolysaccharidosis Type 7 | No longer available | Joyce Fox | 2017-02-06 |
Mucopolysaccharidosis VII (3) · Lysosomal Storage Diseases (2) · Metabolic Diseases (2) · Rare Diseases (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)
Carbohydrate Metabolism, Inborn Errors (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Connective Tissue Diseases (3) · Disease Attributes (3) · Genetic Diseases, Inborn (3) · Metabolism, Inborn Errors (3) · Mucinoses (3) · Mucopolysaccharidoses (3) · Nutritional and Metabolic Diseases (3) · Pathologic Processes (3) · Pathological Conditions, Signs and Symptoms (3) · Skin and Connective Tissue Diseases (3) · Abnormalities, Multiple (1) · Albinism (1) · Albinism, Oculocutaneous (1)
Joyce Fox (1) · RTI International (1) · Ultragenyx Pharmaceutical Inc (1)
United States (2) · United Kingdom (1)
Curt Scharfe, MD, PhD (1) · Simon Jones, MD (1)