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

  
8 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1EUCTR2008-001965-27-ES
(EUCTR)
23/12/200817/09/2008Estudio de extensión abierto de terapia de sustitución enzimática con glucocerebrosidasa humana activada genéticamente® (GA-GCB) en pacientes con enfermedad de Gaucher de tipo IAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher DiseaseEstudio de extensión abierto de terapia de sustitución enzimática con glucocerebrosidasa humana activada genéticamente® (GA-GCB) en pacientes con enfermedad de Gaucher de tipo IAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease Enfermedad de Gaucher de tipo IType I Gaucher disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Product Name: Gene-Activated Human Glucocerebrosidase
Product Code: GA-GCB
INN or Proposed INN: velaglucerase alfa
Other descriptive name: Gene activated human glucocerebrosidase
Shire Human Genetic Therapies, Inc.NULLNot RecruitingFemale: yes
Male: yes
102Spain;United Kingdom
2EUCTR2008-001965-27-GB
(EUCTR)
29/10/200815/07/2008An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher DiseaseAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease Type I Gaucher disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Product Name: Gene-Activated Human Glucocerebrosidase 400U/vial
Product Code: GA-GCB
INN or Proposed INN: velaglucerase alfa
Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa
Product Name: Gene-Activated Human Glucocerebrosidase 200U/vial
Product Code: GA-GCB
INN or Proposed INN: velaglucerase alfa
Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa
Shire Human Genetic Therapies, Inc.NULLNot Recruiting Female: yes
Male: yes
102 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noSpain;United Kingdom
3EUCTR2006-006304-11-IT
(EUCTR)
14/04/200804/08/2008A Multicenter Open-Label Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease Previously Treated with Imiglucerase - NDA Multicenter Open-Label Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease Previously Treated with Imiglucerase - ND Patients with type 1 Gaucher disease who previously received imiglucerase
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Product Name: Gene-Activated Human Glucocerebrosidase
Product Code: GA-GCB
SHIRE HUMAN GENETIC THERAPIES, INC.NULLNot RecruitingFemale: yes
Male: yes
40United Kingdom;Spain;Italy
4EUCTR2007-002840-21-IT
(EUCTR)
25/02/200815/04/2008A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - GCB039A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - GCB039 Patients with Gaucher disease of type 1
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Product Name: Gene Activated Human Glucocerebrosidase
Product Code: GA-GCB
Product Name: imiglucerasi
INN or Proposed INN: Imiglucerase
SHIRE HUMAN GENETIC THERAPIES, INC.NULLNot RecruitingFemale: yes
Male: yes
32United Kingdom;Spain;Italy
5EUCTR2006-006304-11-ES
(EUCTR)
27/08/200704/03/2010A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase.Estudio abierto multicéntrico sobre la terapia de sustitución enzimática con glucocerebrosidasa humana genéticamente activada (Gene-Activated®, GA-GCB) en pacientes con enfermedad de Gaucher de tipo 1 previamente tratados con imiglucerasa - TKT034A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase.Estudio abierto multicéntrico sobre la terapia de sustitución enzimática con glucocerebrosidasa humana genéticamente activada (Gene-Activated®, GA-GCB) en pacientes con enfermedad de Gaucher de tipo 1 previamente tratados con imiglucerasa - TKT034 Type I Gaucher DiseaseEnfermedad de Gaucher de Tipo I
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Product Name: Gene-Activated Human Glucocerebrosidase
Product Code: GA-GCB
Other descriptive name: Gene activated human glucocerebrosidase
Shire Human Genetic Therapies IncNULLNot RecruitingFemale: yes
Male: yes
40Phase 2;Phase 3Spain;Germany;Italy;United Kingdom
6EUCTR2006-006304-11-GB
(EUCTR)
03/07/200712/02/2007A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase - TKT034A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase - TKT034 Type I Gaucher Disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Product Name: Gene-Activated Human Glucocerebrosidase
Product Code: GA-GCB
Other descriptive name: Gene activated human glucocerebrosidase
Shire Human Genetic Therapies IncNULLNot Recruiting Female: yes
Male: yes
40 Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4):Spain;Germany;Italy;United Kingdom
7EUCTR2012-003427-38-Outside-EU/EEA
(EUCTR)
07/04/2016Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher DiseaseA Multi-center, Open-label, Efficacy and Safety Study of Velaglucerase Alfa Enzyme Replacement Therapy in Children and Adolescents With Type 3 Gaucher Disease Type 3 Gaucher disease
MedDRA version: 18.1;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: VPRIV
Product Name: Gene-Activated Human Glucocerebrosidase 400U/vial
Product Code: GA-GCB
INN or Proposed INN: velaglucerase alfa
Other descriptive name: VELAGLUCERASE ALFA
Shire Human Genetic TherapiesNULLNAFemale: yes
Male: yes
6Tunisia;Egypt;India
8EUCTR2006-006304-11-DE
(EUCTR)
02/11/2007A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase. - TKT034A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase. - TKT034 Type I Gaucher Disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Product Name: Gene-Activated Human Glucocerebrosidase
Product Code: GA-GCB
INN or Proposed INN: velaglucerase alfa
Other descriptive name: Gene activated human glucocerebrosidase
Shire Human Genetic Therapies IncNULLNot RecruitingFemale: yes
Male: yes
40Phase 2;Phase 3Spain;Germany;Italy;United Kingdom