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"
12 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04429984
(ClinicalTrials.gov)
January 31, 20219/6/2020Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in IndiaA Post Marketing Surveillance (PMS) Study for VPRIV (Velaglucerase Alfa) in IndiaGaucher DiseaseDrug: Velaglucerase alfa (VPRIV)ShireNULLNot yet recruitingN/AN/AAll35NULL
2NCT03702361
(ClinicalTrials.gov)
September 4, 20186/8/2018Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher DiseaseRapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease: An Investigator-initiated StudyPrimary DiseaseDrug: VPRIVShaare Zedek Medical CenterNULLUnknown status6 Years75 YearsAll15Phase 4Israel
3EUCTR2015-001578-17-IT
(EUCTR)
18/07/201804/11/2020An 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 - SHP-GCB-402 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 U - POLVERE PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO(VETRO) 1 FLACONCINO
Product Name: Velaglucerase alfa
Product Code: [-]
INN or Proposed INN: velaglucerasi alfa
Other descriptive name: GLUCOCEREBROSIDASE UMANO GENE-ATTIVATO
SHIRE HUMAN GENETIC THERAPIES, INCNULLNot RecruitingFemale: yes
Male: yes
40Phase 4United States;Spain;Israel;Germany;United Kingdom;Italy
4EUCTR2015-001578-17-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
5NCT02574286
(ClinicalTrials.gov)
June 29, 20169/10/2015Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants 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 DiseaseGaucher DiseaseDrug: Velaglucerase alfa;Dietary Supplement: Vitamin DShireNULLCompleted18 Years70 YearsAll21Phase 4United States;Israel;Spain;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2015-001578-17-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
7EUCTR2015-001578-17-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
8NCT04120506
(ClinicalTrials.gov)
January 10, 201610/8/2018Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Adult Patients With Type 1 Gaucher Disease, Previously on a Stable Dose of VPRIV for at Least 3 Months: an Extension of the Investigator-initiated StudyGaucher Disease, Type 1Drug: VPRIVShaare Zedek Medical CenterShireActive, not recruiting6 Years75 YearsAll15Phase 4NULL
9NCT02528617
(ClinicalTrials.gov)
July 201528/7/2015The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher DiseaseThe Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher DiseaseGaucher Disease Type 1;Gaucher Disease Type 3Drug: Velaglucerase alfaBaylor Research InstituteTexas Scottish Rite Hospital for ChildrenWithdrawn4 Years14 YearsAll0Phase 4United States
10NCT00635427
(ClinicalTrials.gov)
May 20086/3/2008An Open-Label Extension Study of GA-GCB ERT 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 DiseaseGaucher Disease, Type 1Biological: VPRIV®ShireNULLCompleted2 YearsN/AAll95Phase 3United States;Argentina;India;Israel;Korea, Republic of;Paraguay;Poland;Russian Federation;Spain;Tunisia;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT00430625
(ClinicalTrials.gov)
January 20071/2/2007A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher DiseaseA Multicenter, Randomized, Double-Blind, Parallel Group, Two-Dose Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher DiseaseGaucher Disease, Type 1Biological: VPRIV ®,ShireNULLCompleted2 YearsN/AAll25Phase 3Argentina;Israel;Paraguay;Russian Federation;Tunisia;United States
12EUCTR2012-003427-38-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