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 | EUCTR2007-005668-28-FR (EUCTR) | 12/10/2009 | 16/03/2009 | 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) | 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 BV | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 45 | Phase 3B | Portugal;Czech Republic;Germany;United Kingdom;Netherlands;France | ||
| 2 | EUCTR2007-005668-28-CZ (EUCTR) | 27/11/2008 | 10/12/2008 | 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) | 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 BV | NULL | Not Recruiting | Female: no Male: yes | 45 | Phase 3B | France;Portugal;Czech Republic;Germany;Netherlands;United Kingdom | ||
| 3 | EUCTR2007-005668-28-DE (EUCTR) | 19/11/2008 | 04/08/2008 | 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) | 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 BV | NULL | Not Recruiting | Female: no Male: yes | 45 | Phase 3B | Portugal;Czech Republic;United Kingdom;Germany;Netherlands;France | ||
| 4 | EUCTR2007-005668-28-PT (EUCTR) | 03/10/2008 | 15/07/2008 | 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) | 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 BV | NULL | Not Recruiting | Female: no Male: yes | 45 | Phase 3B | Portugal;Czech Republic;Germany;United Kingdom;Netherlands;France | ||
| 5 | EUCTR2007-005668-28-NL (EUCTR) | 04/09/2008 | 02/06/2008 | 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) | 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 BV | NULL | Not Recruiting | Female: no Male: yes | 24 | Phase 3B | France;Portugal;Czech Republic;Germany;Netherlands;United Kingdom | ||
| 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 |
| 6 | NCT00701415 (ClinicalTrials.gov) | September 2008 | 17/6/2008 | A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms | A 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 Symptoms | Fabry Disease | Biological: Agalsidase beta | Genzyme, a Sanofi Company | NULL | Completed | 5 Years | 18 Years | Male | 31 | Phase 3 | United States;Argentina;Brazil;Canada;Czech Republic;Netherlands;Norway;Poland;United Kingdom;Chile;Portugal |
| 7 | EUCTR2007-005668-28-GB (EUCTR) | 29/07/2008 | 04/04/2008 | A 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 BV | NULL | Not Recruiting | Female: no Male: yes | 35 | Phase 3B | France;Portugal;United States;Czech Republic;Canada;Argentina;Brazil;Poland;Germany;Netherlands;Norway;United Kingdom | ||
| 8 | NCT00230607 (ClinicalTrials.gov) | August 30, 2006 | 29/9/2005 | Study of the Effects of Fabrazyme Treatment on Lactation and Infants | A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants | Fabry Disease;Alpha Galactosidase A Deficiency | Drug: agalsidase beta | Genzyme, a Sanofi Company | NULL | Recruiting | N/A | N/A | All | 20 | Phase 4 | United States;Austria;United Kingdom |
| 9 | NCT00312767 (ClinicalTrials.gov) | April 2006 | 7/4/2006 | A 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 Disease | Drug: Fabrazyme (agalsidase beta) | Genzyme, a Sanofi Company | NULL | Withdrawn | 18 Years | 65 Years | Both | 0 | Phase 4 | United States |
| 10 | NCT00196716 (ClinicalTrials.gov) | June 2003 | 12/9/2005 | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease | A 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 Disease | Fabry Disease | Biological: Fabrazyme (agalsidase beta) | Genzyme, a Sanofi Company | NULL | Completed | 16 Years | N/A | Male | 21 | Phase 2 | Czech Republic;Estonia;Poland;Slovakia |
| 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 |
| 11 | NCT00837824 (ClinicalTrials.gov) | December 2002 | 23/10/2008 | Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme | A Phase 2, Randomized, Open Label, Dose-Ranging, Multiple Dose Study of Fabrazyme® In Patients With Fabry Disease and With Severe Renal Disease | Fabry Disease;Chronic Kidney Disease, Stage IV (Severe) | Biological: Fabrazyme (agalsidase beta) | Genzyme, a Sanofi Company | CRL/Medinet | Terminated | 16 Years | N/A | All | 20 | Phase 2 | United States |
| 12 | NCT00074958 (ClinicalTrials.gov) | October 2002 | 24/12/2003 | A Study of Fabrazyme in Pediatric Patients With Fabry Disease | A Multi-center, Phase 2, Open-Label Study of Fabrazyme (Recombinant Human a-Galactosidase A) Replacement Therapy in Pediatric Patients With Fabry Disease | Fabry Disease | Biological: Fabrazyme (agalsidase beta) | Genzyme, a Sanofi Company | NULL | Completed | 7 Years | 15 Years | All | 16 | Phase 2 | United States;France;Poland;United Kingdom;Italy |
| 13 | NCT00074984 (ClinicalTrials.gov) | February 2001 | 24/12/2003 | A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease | Multi-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 Disease | Fabry Disease | Biological: Fabrazyme (agalsidase beta);Biological: Placebo | Genzyme, a Sanofi Company | NULL | Completed | 16 Years | N/A | All | 82 | Phase 4 | United States;Canada;Czech Republic;Hungary;Poland;United Kingdom |
| 14 | NCT00074971 (ClinicalTrials.gov) | October 1999 | 24/12/2003 | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease | A Multi-center, Open-Label Extension Study of the Safety and Efficacy of Recombinant Human a-Galactosidase A (r-haGAL) Replacement in Patients With Fabry Disease | Fabry Disease | Drug: Fabrazyme (agalsidase beta) | Genzyme, a Sanofi Company | NULL | Completed | 16 Years | N/A | Both | 58 | Phase 3 | United States;France;Netherlands;Puerto Rico;United Kingdom |