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19. Lysosomal storage disease
784 clinical trials,   673 drugs   (DrugBank: 101 drugs),   68 drug target genes,   184 drug target pathways

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"
7 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-IT
(EUCTR)		26/07/200510/08/2005An 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 INCNULLNot RecruitingFemale: no
Male: yes		94United Kingdom;Germany;Spain;Italy;Sweden
2EUCTR2004-002743-27-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
3EUCTR2004-002743-27-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
4EUCTR2004-002743-27-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
5EUCTR2004-002743-27-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
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
6NCT00630747
(ClinicalTrials.gov)		September 200428/2/2008Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving IdursulfaseAn Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement TherapyHunter Syndrome;Mucopolysaccharidosis II (MPS II)Biological: IdursulfaseShireNULLCompleted5 YearsN/AMale94Phase 2;Phase 3United States;Brazil;Canada;France;Germany;Italy;Romania;Spain;Sweden;United Kingdom
7NCT00069641
(ClinicalTrials.gov)		September 200329/9/2003Iduronate-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 IIMucopolysaccharidosis IIBiological: Iduronate-2-sulfatase enzyme replacement therapy;Biological: iduronate-2-sulfatase enzyme replacement therapy;Biological: PlaceboShireNULLCompleted5 Years25 YearsMale96Phase 2;Phase 3United Kingdom;Brazil;Germany;United States

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