19. ライソゾーム病
［臨床試験数：784，薬物数：673（DrugBank：101），標的遺伝子数：68，標的パスウェイ数：184］
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 | NCT02995993 (ClinicalTrials.gov) | November 2016 | 14/12/2016 | Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease | An Open-Label, Multi-Center Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGal in Patients With Fabry Disease | Fabry Disease | Drug: Moss-aGal (recombinant human alpha-galactosidase A produced in moss) | Greenovation Biotech GmbH | FGK Clinical Research GmbH | Completed | 18 Years | 65 Years | All | 6 | Phase 1 | Germany |
| 2 | NCT02800070 (ClinicalTrials.gov) | July 2016 | 15/4/2016 | Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease | Clinical Pilot Study of Autologous Stem Cell Transplantation of Cluster of Differentiation 34 Positive (CD34+) Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease | Fabry Disease | Biological: Lentivirus Alpha-gal A transduced stem cells | University Health Network, Toronto | Ozmosis Research Inc. | Active, not recruiting | 18 Years | 50 Years | Male | 5 | Phase 1 | Canada |
| 3 | 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 | ||
| 4 | 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 | ||
| 5 | 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 | ||
| 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 | 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 | ||
| 7 | 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 | ||
| 8 | NCT00487630 (ClinicalTrials.gov) | June 2005 | 15/6/2007 | Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease | A Multicenter, Phase 4, Randomized, Controlled Study to Evaluate the Efficacy and Safety of Recombinant Alpha-Galactosidase A (Agalsidase Beta, FABRAZYME) in Heterozygous Females for Fabry Disease | Fabry Disease | Drug: recombinant alpha-galactosidase A | Assistance Publique - Hôpitaux de Paris | NULL | Recruiting | 15 Years | N/A | Female | 34 | Phase 4 | France |
| 9 | 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 |
| 10 | NCT00048906 (ClinicalTrials.gov) | November 2002 | 8/11/2002 | Alpha-Galactosidase A Replacement Therapy for Fabry Disease | A Safety and Pharmacokinetic Study of Replagal Enzyme Replacement Therapy in Patients With Fabry Disease | Fabry Disease | Drug: DRX005B | National Institute of Neurological Disorders and Stroke (NINDS) | NULL | Completed | N/A | N/A | Both | 3 | Phase 2 | United States |