19. Lysosomal storage disease
784 clinical trials, 673 drugs (DrugBank: 101 drugs), 68 drug target genes, 184 drug target pathways
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | NCT02156674 (ClinicalTrials.gov) | January 26, 2016 | 3/6/2014 | Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome | Study of Administration of Intravenous Naglazyme® Following Allogeneic Transplantation for Maroteaux-Lamy Syndrome | Maroteaux-Lamy Syndrome | Drug: Naglazyme® | Masonic Cancer Center, University of Minnesota | BioMarin Pharmaceutical | Active, not recruiting | 3 Years | N/A | All | 1 | N/A | United States |
| 2 | 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 | ||
| 3 | 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 | ||||
| 4 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 4 | Phase 4 | Portugal;France | ||
| 5 | 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 |