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.
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 |
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1 | NCT04002830 (ClinicalTrials.gov) | November 20, 2019 | 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 | Not yet 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PEDIATRIC SUBJECTS WITH TYPE 1 GAUCHER DISEASE | Type 1 Gaucher Disease | Drug: Elelyso 60 units/kg | Pfizer | NULL | Withdrawn | N/A | 12 Years | All | 0 | Phase 4 | United States |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 |