19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| 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 | EUCTR2005-003512-30-PT (EUCTR) | 13/08/2007 | 24/07/2007 | A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI) - N/A | A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI) - N/A | Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome) MedDRA version: 9.1;Level: LLT;Classification code 10056892;Term: Mucopolysaccharidosis VI | Trade Name: Naglazyme (galsulfase) | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 4 | Phase 4 | Portugal;France | ||
| 2 | EUCTR2007-001453-26-NL (EUCTR) | 02/07/2007 | 13/11/2009 | Natural course, effects of enzyme therapy and health economic aspects in patients with mucopolysaccharidosis type I, II and VI. Long-term folloe-up of untreated patients and patients receiving commercially available Aldurazyme, Elaprase and Naglazyme. | Natural course, effects of enzyme therapy and health economic aspects in patients with mucopolysaccharidosis type I, II and VI. Long-term folloe-up of untreated patients and patients receiving commercially available Aldurazyme, Elaprase and Naglazyme. | Mucopolysaccharidosis type I, II and VI. MedDRA version: 12.0;Level: LLT;Classification code 10028095;Term: Mucopolysaccharidosis IV MedDRA version: 12.0;Classification code 10056886;Term: Mucopolysaccharidosis I MedDRA version: 12.0;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II | Trade Name: Elaprase Product Name: idursulfase INN or Proposed INN: IDURSULFASE Other descriptive name: Elaprase Trade Name: Naglazyme Product Name: Naglazyme INN or Proposed INN: GALSULFASE Trade Name: Aldurazyme Product Name: Aldurazyme INN or Proposed INN: LARONIDASE Other descriptive name: Aldurazyme | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Netherlands | ||||
| 3 | EUCTR2005-003512-30-FR (EUCTR) | 05/04/2007 | 26/03/2007 | A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI) - N/A | A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI) - N/A | Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome) MedDRA version: 6.0;Level: PT;Classification code 10056892 | Trade Name: Naglazyme Product Name: Naglazyme INN or Proposed INN: galsulfase Other descriptive name: recombinant human Arysulfatase B, recombinant human N-acetylgalactosamine 4-sulfatase | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 4 | Phase 4 | Portugal;France | ||
| 4 | NCT00299000 (ClinicalTrials.gov) | May 2006 | 2/3/2006 | A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI | A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With Maroteaux-Lamy Syndrome (MPS VI) | Mucopolysaccharidosis VI;Maroteaux-Lamy Syndrome | Drug: Naglazyme | BioMarin Pharmaceutical | NULL | Completed | N/A | 1 Year | All | 4 | Phase 4 | United States;France;Portugal |