19. Lysosomal storage disease Clinical trials / Disease details

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

3 trials found

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	NCT04002830 (ClinicalTrials.gov)	November 20, 2020	27/6/2019	A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease	A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease	Gaucher Disease, Type 3	Drug: Elelyso	Ari Zimran	Pfizer	Recruiting	N/A	N/A	All	15	Phase 4	India;Israel;Turkey
2	NCT03021941 (ClinicalTrials.gov)	July 31, 2019	23/9/2016	Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease	A MULTICENTER, OPEN LABEL, PHARMACOKINETICS, PHARMACODYNAMICS AND SAFETY STUDY OF ELELYSO(TM) (TALIGLUCERASE ALFA) IN PEDIATRIC SUBJECTS WITH TYPE 1 GAUCHER DISEASE	Type 1 Gaucher Disease	Drug: Elelyso 60 units/kg	Pfizer	NULL	Withdrawn	N/A	12 Years	All	0	Phase 4	United States
3	NCT04353466 (ClinicalTrials.gov)	January 1, 2017	13/4/2020	Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs	An Open-label, Investigator Initiated Clinical Trial to Asses Impact of Elelyso on Bone Involvement in Patients With Gaucher Disease Currently Treated With Other ERTs	Gaucher Disease, Type 1	Procedure: quantitative chemical shift imaging (QCSI);Drug: Elelyso	Shaare Zedek Medical Center	Pfizer	Active, not recruiting	18 Years	N/A	All	30	N/A	NULL