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"
14 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2007-005668-28-FR
(EUCTR)
12/10/200916/03/2009A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose)A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) Fabry disease
MedDRA version: 9.1;Level: LLT;Classification code 10016016;Term: Fabry's disease
Trade Name: Fabrazyme
Product Name: Fabrazyme
Product Code: Agalsidase beta
INN or Proposed INN: agalsidase beta
Other descriptive name: recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL
Genzyme Europe BVNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
45Phase 3BPortugal;Czech Republic;Germany;United Kingdom;Netherlands;France
2EUCTR2007-005668-28-CZ
(EUCTR)
27/11/200810/12/2008A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose)A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) Fabry disease
MedDRA version: 9.1;Level: LLT;Classification code 10016016;Term: Fabry's disease
Trade Name: Fabrazyme
Product Name: Fabrazyme
Product Code: Agalsidase beta
INN or Proposed INN: agalsidase beta
Other descriptive name: recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL
Genzyme Europe BVNULLNot RecruitingFemale: no
Male: yes
45Phase 3BFrance;Portugal;Czech Republic;Germany;Netherlands;United Kingdom
3EUCTR2007-005668-28-DE
(EUCTR)
19/11/200804/08/2008A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose)A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) Fabry disease
MedDRA version: 9.1;Level: LLT;Classification code 10016016;Term: Fabry's disease
Trade Name: Fabrazyme 35 mg
Product Name: Fabrazyme
Product Code: Agalsidase beta
INN or Proposed INN: agalsidase beta
Other descriptive name: recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL
Trade Name: Fabrazyme 5 mg
Product Name: Fabrazyme
Product Code: Agalsidase beta
INN or Proposed INN: agalsidase beta
Other descriptive name: recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL
Genzyme Europe BVNULLNot RecruitingFemale: no
Male: yes
45Phase 3BPortugal;Czech Republic;United Kingdom;Germany;Netherlands;France
4EUCTR2007-005668-28-PT
(EUCTR)
03/10/200815/07/2008A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose)A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) Fabry disease
MedDRA version: 9.1;Level: LLT;Classification code 10016016;Term: Fabry's disease
Trade Name: Fabrazyme
Product Name: Fabrazyme
Product Code: Agalsidase beta
INN or Proposed INN: agalsidase beta
Other descriptive name: recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL
Genzyme Europe BVNULLNot RecruitingFemale: no
Male: yes
45Phase 3BPortugal;Czech Republic;Germany;United Kingdom;Netherlands;France
5EUCTR2007-005668-28-NL
(EUCTR)
04/09/200802/06/2008A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose)A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) Fabry disease
MedDRA version: 9.1;Level: LLT;Classification code 10016016;Term: Fabry's disease
Trade Name: Fabrazyme
Product Name: Fabrazyme
Product Code: Agalsidase beta
INN or Proposed INN: agalsidase beta
Other descriptive name: recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL
Genzyme Europe BVNULLNot RecruitingFemale: no
Male: yes
24Phase 3BFrance;Portugal;Czech Republic;Germany;Netherlands;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT00701415
(ClinicalTrials.gov)
September 200817/6/2008A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe SymptomsA Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (Agalsidase Beta) in Treatment-Naïve Male Pediatric Patients With Fabry Disease Without Severe SymptomsFabry DiseaseBiological: Agalsidase betaGenzyme, a Sanofi CompanyNULLCompleted5 Years18 YearsMale31Phase 3United States;Argentina;Brazil;Canada;Czech Republic;Netherlands;Norway;Poland;United Kingdom;Chile;Portugal
7EUCTR2007-005668-28-GB
(EUCTR)
29/07/200804/04/2008A study to assess two alternative dosing regimens of Fabrazyme (agalsidase beta) in boys with Fabry disease who have never received any specific treatment for this disease and who do not have severe symptoms of the disease.A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) Fabry disease
MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Fabrazyme
Product Name: Fabrazyme
Product Code: Agalsidase beta
INN or Proposed INN: agalsidase beta
Other descriptive name: recombinant human alpha-galactosidase
Genzyme Europe BVNULLNot RecruitingFemale: no
Male: yes
35Phase 3BFrance;Portugal;United States;Czech Republic;Canada;Argentina;Brazil;Poland;Germany;Netherlands;Norway;United Kingdom
8NCT00230607
(ClinicalTrials.gov)
August 30, 200629/9/2005Study of the Effects of Fabrazyme Treatment on Lactation and InfantsA Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and InfantsFabry Disease;Alpha Galactosidase A DeficiencyDrug: agalsidase betaGenzyme, a Sanofi CompanyNULLRecruitingN/AN/AAll20Phase 4United States;Austria;United Kingdom
9NCT00312767
(ClinicalTrials.gov)
April 20067/4/2006A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency.A Multicenter, Open-Label, Cross-Over Trial to Evaluate the Pharmacokinetics of Fabrazyme During Simultaneous Fabrazyme Infusion and Chronic Hemodialysis in Patients With Fabry Disease.Fabry DiseaseDrug: Fabrazyme (agalsidase beta)Genzyme, a Sanofi CompanyNULLWithdrawn18 Years65 YearsBoth0Phase 4United States
10NCT00196716
(ClinicalTrials.gov)
June 200312/9/2005A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry DiseaseA Multicenter, Open-label Study of Low Dose Maintenance Treatment of Fabrazyme (Recombinant Human Alpha-Galactosidase A (R-h Alpha-GAL)) Replacement Therapy in Patients With Fabry DiseaseFabry DiseaseBiological: Fabrazyme (agalsidase beta)Genzyme, a Sanofi CompanyNULLCompleted16 YearsN/AMale21Phase 2Czech Republic;Estonia;Poland;Slovakia
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT00837824
(ClinicalTrials.gov)
December 200223/10/2008Severe Renal Disease Study in Fabry Patients Treated With FabrazymeA Phase 2, Randomized, Open Label, Dose-Ranging, Multiple Dose Study of Fabrazyme® In Patients With Fabry Disease and With Severe Renal DiseaseFabry Disease;Chronic Kidney Disease, Stage IV (Severe)Biological: Fabrazyme (agalsidase beta)Genzyme, a Sanofi CompanyCRL/MedinetTerminated16 YearsN/AAll20Phase 2United States
12NCT00074958
(ClinicalTrials.gov)
October 200224/12/2003A Study of Fabrazyme in Pediatric Patients With Fabry DiseaseA Multi-center, Phase 2, Open-Label Study of Fabrazyme (Recombinant Human a-Galactosidase A) Replacement Therapy in Pediatric Patients With Fabry DiseaseFabry DiseaseBiological: Fabrazyme (agalsidase beta)Genzyme, a Sanofi CompanyNULLCompleted7 Years15 YearsAll16Phase 2United States;France;Poland;United Kingdom;Italy
13NCT00074984
(ClinicalTrials.gov)
February 200124/12/2003A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry DiseaseMulti-Center, Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Fabrazyme on Progression of Renal Disease and Significant Clinical Events in Patients With Fabry DiseaseFabry DiseaseBiological: Fabrazyme (agalsidase beta);Biological: PlaceboGenzyme, a Sanofi CompanyNULLCompleted16 YearsN/AAll82Phase 4United States;Canada;Czech Republic;Hungary;Poland;United Kingdom
14NCT00074971
(ClinicalTrials.gov)
October 199924/12/2003A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry DiseaseA Multi-center, Open-Label Extension Study of the Safety and Efficacy of Recombinant Human a-Galactosidase A (r-haGAL) Replacement in Patients With Fabry DiseaseFabry DiseaseDrug: Fabrazyme (agalsidase beta)Genzyme, a Sanofi CompanyNULLCompleted16 YearsN/ABoth58Phase 3United States;France;Netherlands;Puerto Rico;United Kingdom