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 | NCT04281537 (ClinicalTrials.gov) | March 1, 2020 | 7/2/2020 | A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy. | A Multi-country Time and Motion Study to Describe the Experience of Clinicians, Patients and Their Caregivers During the Treatment of Fabry Disease With Enzyme Replacement Therapy With Agalsidase Alfa and Agalsidase Beta | Fabry Disease | Drug: Agalsidase Beta;Drug: Agalsidase Alpha | Amicus Therapeutics | NULL | Active, not recruiting | 18 Years | N/A | All | 120 | United States | |
| 2 | NCT02956954 (ClinicalTrials.gov) | March 25, 2017 | 3/11/2016 | Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease | Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease (AFD): Impact of Treatment by Agalsidase Alpha (Replagal®) | Anderson-Fabry Disease | Drug: Enzyme replacement therapy (Agalsidase alpha (Replagal®));Procedure: Magnetic Resonance Imaging | University Hospital, Rouen | NULL | Unknown status | 18 Years | N/A | All | 25 | N/A | France |
| 3 | EUCTR2007-005543-22-SI (EUCTR) | 18/03/2011 | 24/02/2011 | An investigation of 3 dosages of Replagal in adult patients with Fabry disease | A Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in Adult Patients with Fabry Disease | Fabry Disease MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Replagal Product Name: Replagal INN or Proposed INN: agalsidase alpha Other descriptive name: Replagal | Shire Human Genetic Therapies, Inc. (Shire HGT) | NULL | Not Recruiting | Female: yes Male: yes | 43 | United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom | |||
| 4 | EUCTR2007-005543-22-FI (EUCTR) | 18/05/2010 | 23/03/2010 | An investigation of 3 dosages of Replagal in adult patients with Fabry disease | A Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in Adult Patients with Fabry Disease | Fabry Disease MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Replagal Product Name: Replagal INN or Proposed INN: agalsidase alpha Other descriptive name: Replagal | Shire Human Genetic Therapies, Inc. (Shire HGT) | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 43 | Finland;United Kingdom;Czech Republic;Paraguay;Slovenia;United States;Australia | |||
| 5 | EUCTR2007-005543-22-CZ (EUCTR) | 19/06/2008 | 02/01/2008 | An investigation of 3 dosages of Replagal in adult patients with Fabry disease | A Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in Adult Patients with Fabry Disease | Fabry Disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Replagal Product Name: Replagal INN or Proposed INN: agalsidase alpha Other descriptive name: Replagal | Shire Human Genetic Therapies, Inc. (Shire HGT) | NULL | Not Recruiting | Female: yes Male: yes | 43 | United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom |