19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

  
12 trials found
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1EUCTR2019-000667-24-IT
(EUCTR)
28/03/202223/09/2021A Phase I/II Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects with Fabry DiseaseA Phase I/II, Multicenter, Open-Label, SingleDose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease - STAAR Fabry Disease (X-linked lysosomal storage disease)
MedDRA version: 20.0;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]
Product Name: Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A
Product Code: [ST-920]
Other descriptive name: Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA
Trade Name: PARACETAMOLO DOC GENERICI - 1000 MG COMPRESSA 16 COMPRESSE
Product Name: Paracetamolo
Product Code: [Paracetamolo]
INN or Proposed INN: PARACETAMOLO
Trade Name: ALISERIN - 25 MG GRANULATO EFFERVESCENTE 20 BUSTINE
Product Name: difenidramina cloridrato
Product Code: [difenidramina cloridrato]
INN or Proposed INN: DIFENIDRAMINA CLORIDRATO
Trade Name: PREDNISONE DOC GENERICI - 5 MG COMPRESSE 10 COMPRESSE IN BLISTER PVC-PVDC/ALU
Product Name: Prednisone
Product Code: [Prednisone]
INN or Proposed INN: PREDNISONE
Sangamo Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
48Phase 1;Phase 2United States;Taiwan;Canada;Australia;Germany;United Kingdom;Italy
2EUCTR2019-000667-24-GB
(EUCTR)
12/11/201919/03/2019A Phase I/II Study to Assess the Safety and Tolerability of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects with Fabry DiseaseA Phase I/II, Multicenter, Open-Label, SingleDose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease Fabry Disease (X-linked lysosomal storage disease)
MedDRA version: 20.0;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]
Product Name: Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A
Product Code: ST-920
INN or Proposed INN: Not yet assigned
Other descriptive name: Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA
Sangamo Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
30 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom
3NCT02995993
(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
4NCT02800070
(ClinicalTrials.gov)
July 201615/4/2016Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry DiseaseClinical 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 DiseaseFabry DiseaseBiological: Lentivirus Alpha-gal A transduced stem cellsUniversity Health Network, TorontoOzmosis Research Inc.Active, not recruiting18 Years50 YearsMale5Phase 1Canada
5EUCTR2007-005668-28-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 BVNULLNot RecruitingFemale: no
Male: yes
45Phase 3Portugal;France;Czech Republic;Germany;Netherlands;United Kingdom
6EUCTR2007-005668-28-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 3France;Portugal;Czech Republic;Germany;Netherlands;United Kingdom
7EUCTR2007-005668-28-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 3Portugal;Czech Republic;United Kingdom;Germany;Netherlands;France
8EUCTR2007-005668-28-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 3Portugal;Czech Republic;Germany;United Kingdom;Netherlands;France
9EUCTR2007-005668-28-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 3France;Portugal;Czech Republic;Germany;Netherlands;United Kingdom
10NCT00487630
(ClinicalTrials.gov)
June 200515/6/2007Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry DiseaseA 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 DiseaseFabry DiseaseDrug: recombinant alpha-galactosidase AAssistance Publique - Hôpitaux de ParisNULLRecruiting15 YearsN/AFemale34Phase 4France
11NCT00196716
(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
12NCT00048906
(ClinicalTrials.gov)
November 20028/11/2002Alpha-Galactosidase A Replacement Therapy for Fabry DiseaseA Safety and Pharmacokinetic Study of Replagal Enzyme Replacement Therapy in Patients With Fabry DiseaseFabry DiseaseDrug: DRX005BNational Institute of Neurological Disorders and Stroke (NINDS)NULLCompletedN/AN/ABoth3Phase 2United States