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-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
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 |
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
1 | NCT04429984 (ClinicalTrials.gov) | January 31, 2021 | 9/6/2020 | Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India | A Post Marketing Surveillance (PMS) Study for VPRIV (Velaglucerase Alfa) in India | Gaucher Disease | Drug: Velaglucerase alfa (VPRIV) | Shire | NULL | Not yet recruiting | N/A | N/A | All | 35 | NULL | |
2 | NCT03702361 (ClinicalTrials.gov) | September 4, 2018 | 6/8/2018 | Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease | Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease: An Investigator-initiated Study | Primary Disease | Drug: VPRIV | Shaare Zedek Medical Center | NULL | Unknown status | 6 Years | 75 Years | All | 15 | Phase 4 | Israel |
3 | 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 Type 1 Gaucher Disease, Previously on a Stable Dose of VPRIV for at Least 3 Months: an Extension of the Investigator-initiated Study | Gaucher Disease, Type 1 | Drug: VPRIV | Shaare Zedek Medical Center | Shire | Active, not recruiting | 6 Years | 75 Years | All | 15 | Phase 4 | NULL |
4 | 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 |