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

2 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	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ALFA) IN PEDIATRIC SUBJECTS WITH TYPE 1 GAUCHER DISEASE	Type 1 Gaucher Disease	Drug: Elelyso 60 units/kg	Pfizer	NULL	Withdrawn	N/A	12 Years	All	0	Phase 4	United States
2	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	Completed	18 Years	N/A	All	30	N/A	NULL