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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
8 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02995993
(ClinicalTrials.gov)
November 201614/12/2016Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry DiseaseAn Open-Label, Multi-Center Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGal in Patients With Fabry DiseaseFabry DiseaseDrug: Moss-aGal (recombinant human alpha-galactosidase A produced in moss)Greenovation Biotech GmbHFGK Clinical Research GmbHCompleted18 Years65 YearsAll6Phase 1Germany
2EUCTR2007-005668-28-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
3EUCTR2007-005668-28-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
4EUCTR2007-005668-28-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
5EUCTR2007-005668-28-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2007-005668-28-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
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
8NCT00196716
(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