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 | NCT00258011 (ClinicalTrials.gov) | December 2005 | 22/11/2005 | Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease | A Safety Confirmatory Study of JC0498 (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients | Mucopolysaccharidosis I;Hurler Syndrome;Hurler-Scheie Syndrome;Scheie Syndrome | Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | Genzyme, a Sanofi Company | BioMarin/Genzyme LLC | Completed | N/A | N/A | All | 3 | Phase 3 | Japan |
| 2 | NCT00144781 (ClinicalTrials.gov) | December 2004 | 2/9/2005 | A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease | A Multicenter, Multinational, Randomized, Dose-Optimization Study of the Safety and Pharmacodynamic Response of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I | Mucopolysaccharidosis I;Hurler's Syndrome;Hurler-Scheie Syndrome;Scheie Syndrome | Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | Genzyme, a Sanofi Company | BioMarin/Genzyme LLC | Completed | N/A | N/A | All | 34 | Phase 4 | Brazil;Canada |
| 3 | NCT00146757 (ClinicalTrials.gov) | October 2002 | 2/9/2005 | A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old | A Phase II Open-Label Clinical Trial of Recombinant Human Alpha-L-iduronidase (Aldurazyme®) to Evaluate the Safety and Pharmacokinetics in Mucopolysaccharidosis I (MPS I) Patients Less Than 5 Years Old | Mucopolysaccharidosis I;Hurler Syndrome;Hurler-Scheie Syndrome;Scheie Syndrome | Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | Genzyme, a Sanofi Company | BioMarin/Genzyme LLC | Completed | N/A | 5 Years | All | 20 | Phase 2 | France;Germany;Netherlands;United Kingdom |