Recombinant human beta glucuronidase (DrugBank: -)
1 disease| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 19 | Lysosomal storage disease | 4 |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2013-001152-35-ES (EUCTR) | 21/08/2014 | 04/07/2014 | A safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injection | An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7 | Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: Not available Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS | Ultragenyx Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 5 | Phase 1;Phase 2 | Spain;United Kingdom | ||
| 2 | EUCTR2015-001875-32-Outside-EU/EEA (EUCTR) | 12/11/2015 | A long-term safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injection | A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects with MPS 7 | Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: pending Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS | Ultragenyx Pharmaceutical Inc. | NULL | NA | Female: yes Male: yes | 12 | United States | ||||
| 3 | EUCTR2014-005638-71-Outside-EU/EEA (EUCTR) | 22/12/2014 | An efficacy and safety study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injection | A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7 | Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: Not available Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS | Ultragenyx Pharmaceutical Inc. | NULL | NA | Female: yes Male: yes | 12 | Phase 3 | United States | |||
| 4 | EUCTR2015-000104-26-Outside-EU/EEA (EUCTR) | 23/07/2015 | A safety and efficacy study in young MPS 7 patients (less than 5 years old) receiving enzyme (UX003) replacement by intravenous injection | An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 years old | Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human betaglucuronidase Product Code: UX003 INN or Proposed INN: pending Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS | Ultragenyx Pharmaceutical Inc. | NULL | NA | Female: yes Male: yes | 7 | United States |