19. Lysosomal storage disease Clinical trials / Disease details

Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182

12 trials found

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No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT05529992 (ClinicalTrials.gov)	January 3, 2023	2/9/2022	A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher ...	A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucer ...	Gaucher Disease	Drug: Velaglucerase Alfa	Takeda	NULL	Recruiting	2 Years	N/A	All	20	Phase 3	China
2	NCT03702361 (ClinicalTrials.gov)	September 4, 2018	6/8/2018	Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gau ...	Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gau ...	Primary Disease	Drug: VPRIV	Shaare Zedek Medical Center	NULL	Completed	6 Years	75 Years	All	15	Phase 4	Israel
3	EUCTR2015-001578-17-IT (EUCTR)	18/07/2018	04/11/2020	An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Na ...	An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase a ...	Gaucher Disease MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease ...	Trade Name: VPRIV - 400 U - POLVERE PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO(VETRO) ...	SHIRE HUMAN GENETIC THERAPIES, INC	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 4	United States;Spain;Israel;Germany;United Kingdom;Italy
4	EUCTR2015-001578-17-DE (EUCTR)	12/09/2016	22/12/2015	An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Na ...	An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase a ...	Gaucher Disease MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease ...	Trade Name: VPRIV 400 Units powder for solution of infusion INN or Proposed INN: VELAGLUCERASE ALFA Oth ...	Shire Human Genetic Therapies, Inc.	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 4	United States;Spain;Turkey;Israel;Germany;Italy;United Kingdom;India
5	NCT02574286 (ClinicalTrials.gov)	June 29, 2016	9/10/2015	Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Part ...	An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment With Velaglucerase ...	Gaucher Disease	Drug: Velaglucerase alfa;Dietary Supplement: Vitamin D	Shire	NULL	Completed	18 Years	70 Years	All	21	Phase 4	United States;Israel;Spain;United Kingdom
6	EUCTR2015-001578-17-ES (EUCTR)	25/02/2016	13/01/2016	An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Na ...	An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment with Velaglucerase ...	Gaucher Disease MedDRA version: 18.1;Level: PT;Classification code 10075697;Term: Gaucher's disease ...	Trade Name: VPRIV 400 Units powder for solution of infusion INN or Proposed INN: VELAGLUCERASE ALFA Oth ...	Shire Human Genetic Therapies, Inc.	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 4	United States;Canada;Spain;Israel;Germany;United Kingdom;Italy
7	EUCTR2015-001578-17-GB (EUCTR)	17/02/2016	21/12/2015	An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Na ...	An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase a ...	Gaucher Disease MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease ...	Trade Name: VPRIV 400 Units powder for solution of infusion INN or Proposed INN: VELAGLUCERASE ALFA Oth ...	Shire Human Genetic Therapies, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	19	Phase 4	United States;Canada;Spain;Israel;Germany;United Kingdom
8	NCT04120506 (ClinicalTrials.gov)	January 10, 2016	10/8/2018	Long 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 ...	Gaucher Disease, Type 1	Drug: VPRIV	Shaare Zedek Medical Center	Shire	Completed	6 Years	75 Years	All	15	Phase 4	NULL
9	NCT02528617 (ClinicalTrials.gov)	July 2015	28/7/2015	The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease	The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease	Gaucher Disease Type 1;Gaucher Disease Type 3	Drug: Velaglucerase alfa	Baylor Research Institute	Texas Scottish Rite Hospital for Children	Withdrawn	4 Years	14 Years	All	0	Phase 4	United States
10	NCT00635427 (ClinicalTrials.gov)	May 2008	6/3/2008	An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease	An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacemen ...	Gaucher Disease, Type 1	Biological: VPRIV ®	Shire	NULL	Completed	2 Years	N/A	All	95	Phase 3	United States;Argentina;India;Israel;Korea, Republic of;Paraguay;Poland;Russian Federation;Spain;Tun ...

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