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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
5 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
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agemax
Inclusion_
gender
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PhaseCountries
1NCT02156674
(ClinicalTrials.gov)
January 26, 20163/6/2014Naglazyme After Allo Transplant for Maroteaux-Lamy SyndromeStudy of Administration of Intravenous Naglazyme® Following Allogeneic Transplantation for Maroteaux-Lamy SyndromeMaroteaux-Lamy SyndromeDrug: Naglazyme®Masonic Cancer Center, University of MinnesotaBioMarin PharmaceuticalActive, not recruiting3 YearsN/AAll1N/AUnited States
2EUCTR2005-003512-30-PT
(EUCTR)
13/08/200724/07/2007A 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/AA 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 IncNULLNot RecruitingFemale: yes
Male: yes
4Phase 4Portugal;France
3EUCTR2007-001453-26-NL
(EUCTR)
02/07/200713/11/2009Natural 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
NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
50Netherlands
4EUCTR2005-003512-30-FR
(EUCTR)
05/04/200726/03/2007A 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/AA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
4Phase 4Portugal;France
5NCT00299000
(ClinicalTrials.gov)
May 20062/3/2006A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VIA 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 SyndromeDrug: NaglazymeBioMarin PharmaceuticalNULLCompletedN/A1 YearAll4Phase 4United States;France;Portugal