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 | EUCTR2004-002743-27-IT (EUCTR) | 26/07/2005 | 10/08/2005 | An open-label extension of study TKT024 evaluating long-term safety and clinical outcomes in MPS II patients receiving iduronate-2-sulfatase enzyme replacement therapy. | An open-label extension of study TKT024 evaluating long-term safety and clinical outcomes in MPS II patients receiving iduronate-2-sulfatase enzyme replacement therapy. | Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) MedDRA version: 6.1;Level: PT;Classification code 10056889 | Product Name: Idursulfase Product Code: I2S | TKT INC | NULL | Not Recruiting | Female: no Male: yes | 94 | United Kingdom;Germany;Spain;Italy;Sweden | |||
| 2 | EUCTR2004-002743-27-SE (EUCTR) | 11/05/2005 | 17/03/2005 | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) MedDRA version: 6.1;Level: PT;Classification code 10056889 | Product Name: Idursulfase (I2S) Other descriptive name: Idursulfase | Shire Human Genetic Therapies INC. | NULL | Not Recruiting | Female: no Male: yes | 96 | United Kingdom;Germany;Spain;Italy;Sweden | |||
| 3 | EUCTR2004-002743-27-ES (EUCTR) | 21/04/2005 | 04/04/2006 | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) MedDRA version: 6.1;Level: PT;Classification code 10056889 | Product Name: Idursulfase (I2S) Other descriptive name: Idursulfase | TKT Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 94 | United Kingdom;Germany;Spain;Italy;Sweden | |||
| 4 | EUCTR2004-002743-27-GB (EUCTR) | 31/01/2005 | 23/02/2005 | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) MedDRA version: 6.1;Level: PT;Classification code 10056889 | Product Name: Idursulfase (I2S) Other descriptive name: Idursulfase | TKT Inc. | NULL | Not Recruiting | Female: no Male: yes | 94 | Phase 3 | Spain;Germany;Italy;United Kingdom;Sweden | ||
| 5 | EUCTR2004-002743-27-DE (EUCTR) | 23/12/2004 | 30/11/2004 | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) MedDRA version: 6.1;Level: PT;Classification code 10056889 | Product Name: Idursulfase (I2S) Other descriptive name: Idursulfase | Shire Human Genetic Therapies INC. | NULL | Not Recruiting | Female: no Male: yes | 96 | United Kingdom;Germany;Spain;Italy;Sweden | |||
| 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 |
| 6 | NCT00630747 (ClinicalTrials.gov) | September 2004 | 28/2/2008 | Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | Hunter Syndrome;Mucopolysaccharidosis II (MPS II) | Biological: Idursulfase | Shire | NULL | Completed | 5 Years | N/A | Male | 94 | Phase 2;Phase 3 | United States;Brazil;Canada;France;Germany;Italy;Romania;Spain;Sweden;United Kingdom |
| 7 | NCT00069641 (ClinicalTrials.gov) | September 2003 | 29/9/2003 | Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) | A Phase II/III, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Weekly and Every Other Week Dosing Regimens of Iduronate-2-Sulfatase Enzyme Replacement Therapy in Patients With MPS II | Mucopolysaccharidosis II | Biological: Iduronate-2-sulfatase enzyme replacement therapy;Biological: iduronate-2-sulfatase enzyme replacement therapy;Biological: Placebo | Shire | NULL | Completed | 5 Years | 25 Years | Male | 96 | Phase 2;Phase 3 | United Kingdom;Brazil;Germany;United States |