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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		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2016-000301-37-SE
(EUCTR)		09/10/201805/06/2018Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		Trade Name: Cerdelga
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Trade Name: Cerezyme
Product Name: Imiglucerase
INN or Proposed INN: IMIGLUCERASE
Other descriptive name: Cerezyme
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE		Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		120Phase 3France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;United Kingdom;Italy;Sweden
2EUCTR2016-000301-37-ES
(EUCTR)		21/06/201806/07/2018Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		Trade Name: Cerdelga
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Trade Name: Cerezyme
Product Name: Imiglucerase
INN or Proposed INN: IMIGLUCERASE
Other descriptive name: Cerezyme
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE		Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		120Phase 3France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;Italy;United Kingdom;Sweden
3NCT03485677
(ClinicalTrials.gov)		April 11, 201823/3/2018Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3Open Label, Two Cohort (With and Without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients With Gaucher Disease Type 1 and Type 3Gaucher's Disease Type I;Gaucher's Disease Type IIIDrug: Eliglustat GZ385660;Drug: Imiglucerase GZ437843SanofiNULLRecruiting2 Years17 YearsAll60Phase 3Argentina;Canada;France;Italy;Japan;Russian Federation;Spain;Sweden;Turkey;United Kingdom
4NCT02536755
(ClinicalTrials.gov)		October 28, 201527/8/2015Study of Skeletal Response to Eliglustat in Patients With Gaucher DiseaseOpen Label Interventional Multicenter Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Successfully Completed the Phase 2 or Phase 3 StudiesGaucher's DiseaseDrug: eliglustat GZ385660Genzyme, a Sanofi CompanyNULLActive, not recruiting18 YearsN/AAll32Phase 3Canada;Russian Federation
5EUCTR2016-000301-37-IT
(EUCTR)		20/06/2018Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		Trade Name: Cerdelga
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Trade Name: Cerezyme
Product Name: Imiglucerase
INN or Proposed INN: IMIGLUCERASE
Other descriptive name: Cerezyme
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE		Genzyme CorporationNULLNAFemale: yes
Male: yes		120Phase 3France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;United Kingdom;Italy;Sweden

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