19. Lysosomal storage disease Clinical trials / Disease details


Clinical trials : 854 Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191

  
11 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1EUCTR2015-001578-17-DE
(EUCTR)
12/09/201622/12/2015An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher DiseaseAn Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase alfa on Bone-related Pathology inTreatment-naïve Patients with Type 1 Gaucher Disease Gaucher Disease
MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: VPRIV 400 Units powder for solution of infusion
INN or Proposed INN: VELAGLUCERASE ALFA
Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa
Shire Human Genetic Therapies, Inc.NULLNot RecruitingFemale: yes
Male: yes
40Phase 4United States;Spain;Turkey;Israel;Germany;Italy;United Kingdom;India
2EUCTR2015-001578-17-ES
(EUCTR)
25/02/201613/01/2016An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher DiseaseAn Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment with Velaglucerase alfa on Bone-related Pathology in Treatment-naïve Patients with Type 1 Gaucher Disease 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] - Nutritional and Metabolic Diseases [C18]
Trade Name: VPRIV 400 Units powder for solution of infusion
INN or Proposed INN: VELAGLUCERASE ALFA
Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa
Shire Human Genetic Therapies, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 4United States;Canada;Spain;Israel;Germany;Italy;United Kingdom
3EUCTR2015-001578-17-GB
(EUCTR)
17/02/201621/12/2015An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher DiseaseAn Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase alfa on Bone-related Pathology inTreatment-naïve Patients with Type 1 Gaucher Disease Gaucher Disease
MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: VPRIV 400 Units powder for solution of infusion
INN or Proposed INN: VELAGLUCERASE ALFA
Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa
Shire Human Genetic Therapies, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
19Phase 4United States;Canada;Spain;Israel;Germany;United Kingdom
4EUCTR2008-001965-27-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
5EUCTR2008-001965-27-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
6EUCTR2007-002840-21-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
7EUCTR2007-002840-21-GB
(EUCTR)
29/11/200711/10/2007A 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 - HGT-GCB-039A 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 - HGT-GCB-039 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
Trade Name: Cerezyme
Product Name: Cerezyme
INN or Proposed INN: IMIGLUCERASE
Trade Name: Cerezyme
Product Name: Cerezyme
INN or Proposed INN: IMIGLUCERASE
Shire Human Genetic Therapies, Inc.NULLNot RecruitingFemale: yes
Male: yes
32United Kingdom;Spain;Italy
8EUCTR2007-002840-21-ES
(EUCTR)
30/10/200718/07/2007A 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 DiseaseEstudio multicéntrico, aleatorizado, a doble ciego, en grupos paralelos sobre la terapia de reemplazo enzimático con Gene-Activated® glucocerebrosidasa humana (GA-GCB) en comparación con imiglucerasa en pacientes con enfermedad de Gaucher de tipo 1 - 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 DiseaseEstudio multicéntrico, aleatorizado, a doble ciego, en grupos paralelos sobre la terapia de reemplazo enzimático con Gene-Activated® glucocerebrosidasa humana (GA-GCB) en comparación con imiglucerasa en pacientes con enfermedad de Gaucher de tipo 1 - GCB039 Type I Gaucher diseaseEnfermedad de Gaucher tipo 1
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
Trade Name: Cerezyme
INN or Proposed INN: IMIGLUCERASE
Shire Human Genetic Therapies IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
32United Kingdom;Spain;Italy
9EUCTR2006-006304-11-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 IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40United Kingdom;Spain;Italy
10EUCTR2006-006304-11-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
11EUCTR2006-006304-11-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