Rhgus (DrugBank: -)
1 disease| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 19 | Lysosomal storage disease | 11 |
| 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 | EUCTR2015-000104-26-PT (EUCTR) | 03/10/2016 | 03/05/2016 | A safety, tolerability and efficacy study in MPS 7 patients less than 5 years of age 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] | Ultragenyx Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | United States;Portugal;Spain | |||
| 2 | EUCTR2015-001875-32-PT (EUCTR) | 18/04/2016 | 19/01/2016 | 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] | Ultragenyx Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 3 | United States;Portugal;Mexico;Brazil | |||
| 3 | EUCTR2015-000104-26-ES (EUCTR) | 05/04/2016 | 03/02/2016 | A safety, tolerability and efficacy study in MPS 7 patients less than 5 years of age 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] | Ultragenyx Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | Portugal;United States;Spain | |||
| 4 | NCT02432144 (ClinicalTrials.gov) | November 10, 2015 | 22/4/2015 | A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) | A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7 | Sly Syndrome;MPS VII;Mucopolysaccharidosis;Mucopolysaccharidosis VII | Drug: UX003 | Ultragenyx Pharmaceutical Inc | NULL | Completed | 5 Years | N/A | All | 12 | Phase 3 | United States;Brazil;Mexico;Portugal |
| 5 | NCT02418455 (ClinicalTrials.gov) | July 21, 2015 | 12/4/2015 | Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age | An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less Than 5 Years Old | Sly Syndrome;MPS VII;Mucopolysaccharidosis;Mucopolysaccharidosis VII | Drug: UX003 | Ultragenyx Pharmaceutical Inc | NULL | Completed | N/A | 5 Years | All | 8 | Phase 2 | United States;Portugal;Spain |
| 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 |
| 6 | NCT02230566 (ClinicalTrials.gov) | December 2014 | 22/8/2014 | A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) | 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 | MPS 7;Sly Syndrome;Mucopolysaccharidosis;MPS VII | Drug: UX003;Other: Placebo | Ultragenyx Pharmaceutical Inc | NULL | Completed | 5 Years | 35 Years | All | 12 | Phase 3 | United States |
| 7 | 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 | ||
| 8 | EUCTR2013-001152-35-GB (EUCTR) | 02/08/2013 | 25/06/2013 | 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] | Ultragenyx Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 5 | Phase 1;Phase 2 | Spain;United Kingdom | |||
| 9 | 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 | |||
| 10 | 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 | ||||
| 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 |
| 11 | 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 |