19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| 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 | NCT03071341 (ClinicalTrials.gov) | October 2016 | 27/2/2017 | Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I | An Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Patients With Mucopolysaccharidosis I Who Were Previously Enrolled in Studies With AGT-181 | Mucopolysaccharidosis I | Drug: AGT-181 | ArmaGen, Inc | NULL | Completed | 2 Years | N/A | All | 19 | Phase 1/Phase 2 | Brazil |
| 2 | NCT02597114 (ClinicalTrials.gov) | November 2015 | 3/11/2015 | Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181 | An Extension Study of AGT-181-102 Evaluating Safety and Glycosaminoglycans (GAGs) in Adult Patients With Hurler-Scheie or Scheie Syndrome Who Have Completed 8-Weeks of Dosing With AGT-181 in Study AGT-181-102 | Mucopolysaccharidosis I | Drug: AGT-181 | ArmaGen, Inc | NULL | Completed | 18 Years | N/A | All | 3 | Phase 1 | NULL |
| 3 | NCT03053089 (ClinicalTrials.gov) | October 2015 | 9/2/2017 | Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I | A Two-Stage, Phase 1/2, Open-Label Study of the Human Insulin Receptor Monoclonal Antibody-Human Alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein, AGT-181 in Patients With Mucopolysaccharidosis I (MPS I, Hurler Syndrome) | Mucopolysaccharidosis I | Drug: AGT-181 | ArmaGen, Inc | NULL | Completed | 2 Years | N/A | All | 21 | Phase 1/Phase 2 | Brazil |
| 4 | NCT02371226 (ClinicalTrials.gov) | July 2015 | 19/2/2015 | Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I | A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein, AGT-181 in Adult Patients With Mucopolysaccharidosis I (MPS I, Hurler Syndrome) | Mucopolysaccharidosis I | Drug: AGT-181 (HIRMAb-IDUA) | ArmaGen, Inc | NULL | Active, not recruiting | 18 Years | N/A | All | 3 | Phase 1 | United States |