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-ganglio ... show all
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"
3 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1NCT04002830
(ClinicalTrials.gov)
November 20, 201927/6/2019A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher DiseaseA Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher DiseaseGaucher Disease, Type 3Drug: ElelysoAri ZimranPfizerNot yet recruitingN/AN/AAll15Phase 4India;Israel;Turkey
2NCT03021941
(ClinicalTrials.gov)
July 31, 201923/9/2016Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher DiseaseA MULTICENTER, OPEN LABEL, PHARMACOKINETICS, PHARMACODYNAMICS AND SAFETY STUDY OF ELELYSO(TM) (TALIGLUCERASE ALFA) IN PEDIATRIC SUBJECTS WITH TYPE 1 GAUCHER DISEASEType 1 Gaucher DiseaseDrug: Elelyso 60 units/kgPfizerNULLWithdrawnN/A12 YearsAll0Phase 4United States
3NCT04353466
(ClinicalTrials.gov)
January 1, 201713/4/2020Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTsAn Open-label, Investigator Initiated Clinical Trial to Asses Impact of Elelyso on Bone Involvement in Patients With Gaucher Disease Currently Treated With Other ERTsGaucher Disease, Type 1Procedure: quantitative chemical shift imaging (QCSI);Drug: ElelysoShaare Zedek Medical CenterPfizerActive, not recruiting18 YearsN/AAll30N/ANULL