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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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
5 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2004-002743-27-SE
(EUCTR)		11/05/200517/03/2005An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement TherapyAn 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.NULLNot RecruitingFemale: no
Male: yes		96United Kingdom;Germany;Spain;Italy;Sweden
2EUCTR2004-002743-27-ES
(EUCTR)		21/04/200504/04/2006An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement TherapyAn 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes		94United Kingdom;Germany;Spain;Italy;Sweden
3EUCTR2004-002743-27-GB
(EUCTR)		31/01/200523/02/2005An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement TherapyAn 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.NULLNot RecruitingFemale: no
Male: yes		94Phase 3Spain;Germany;Italy;United Kingdom;Sweden
4EUCTR2004-002743-27-DE
(EUCTR)		23/12/200430/11/2004An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement TherapyAn 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.NULLNot RecruitingFemale: no
Male: yes		96United Kingdom;Germany;Spain;Italy;Sweden
5EUCTR2007-006044-22-PL
(EUCTR)		21/04/2008A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy - not availableA Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy - not available Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome)
MedDRA version: 10.1;Level: PT;Classification code 10056889;Term: 		Trade Name: Elaprase 2mg/ml
Product Name: Idursulfase (I2S)
Product Code: I2S
INN or Proposed INN: Idursulfase
Other descriptive name: Idursulfase		Shire Human Genetic Therapies, Inc. (Shire HGT)NULLNot RecruitingFemale: no
Male: yes		30Phase 4Poland

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