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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
1NCT00813865
(ClinicalTrials.gov)		May 11, 200922/12/2008A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher PatientsAn Open-Label, Multicenter, Long-Term Extension Study To Assess The Safety, Efficacy, And Pharmacodynamics Of AT2101 In Adult Patients With Type 1 Gaucher DiseaseGaucher Disease;Type 1 Gaucher Disease;Gaucher Disease, Type 1Drug: afegostat tartrateAmicus TherapeuticsNULLCompleted18 YearsN/AAll8Phase 2United States;United Kingdom;Paraguay
2NCT00875160
(ClinicalTrials.gov)		April 20092/4/2009A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101An Open-Label Study to Assess the Pharmacokinetics, Safety, and Pharmacodynamics of Repeated Doses of Orally Administered AT2101 in Adult Patients With Type 1 Gaucher DiseaseType 1 Gaucher DiseaseDrug: AT2101Amicus TherapeuticsNULLTerminated18 Years65 YearsBoth1Phase 1United States;United Kingdom
3EUCTR2008-007158-36-GB
(EUCTR)		25/02/200929/04/2009An open-label, multicentre, long-term extension study to assess the safety, efficacy and pharmacodynamics of AT2101 in adult patients with Type I Gaucher disease.An open-label, multicentre, long-term extension study to assess the safety, efficacy and pharmacodynamics of AT2101 in adult patients with Type I Gaucher disease. Type I Gaucher Disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease		Product Name: afegostat tartrate
Product Code: AT2101
INN or Proposed INN: afegostat tartrate		Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes		16United Kingdom
4NCT00446550
(ClinicalTrials.gov)		June 11, 20089/3/2007A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher DiseaseA Randomized, Open-label Study To Assess the Safety and Tolerability of AT2101 in Treatment-naive Adult Patients With Type 1 Gaucher DiseaseGaucher Disease;Type 1 Gaucher Disease;Gaucher Disease, Type 1Drug: afegostat tartrateAmicus TherapeuticsNULLCompleted18 Years74 YearsAll19Phase 2United States;Israel;South Africa;United Kingdom;Germany
5EUCTR2007-003359-35-DE
(EUCTR)		28/12/200722/12/2008A RANDOMIZED, OPEN-LABEL STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF AT2101 IN TREATMENT-NAIVE ADULT PATIENTS WITH TYPE 1 GAUCHER DISEASEA RANDOMIZED, OPEN-LABEL STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF AT2101 IN TREATMENT-NAIVE ADULT PATIENTS WITH TYPE 1 GAUCHER DISEASE Type 1 Gaucher DiseaseProduct Name: Isofagamine tartrate
Product Code: AT2101		Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes		16United Kingdom;Germany
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
6EUCTR2007-003359-35-GB
(EUCTR)		19/12/200714/09/2007A RANDOMIZED, OPEN-LABEL STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF AT2101 IN TREATMENT-NAIVE ADULT PATIENTS WITH TYPE 1 GAUCHER DISEASEA RANDOMIZED, OPEN-LABEL STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF AT2101 IN TREATMENT-NAIVE ADULT PATIENTS WITH TYPE 1 GAUCHER DISEASE Type 1 Gaucher DiseaseProduct Name: Isofagamine tartrate
Product Code: AT2101		Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes		16Germany;United Kingdom
7NCT00433147
(ClinicalTrials.gov)		March 23, 20077/2/2007A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement TherapyA Randomized, Open-label Study to Assess the Safety and Tolerability of Multiple Dose Levels and Multiple Dosing Regimens of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement TherapyGaucher Disease, Type 1;Type 1 Gaucher Disease;Gaucher DiseaseDrug: Afegostat tartrateAmicus TherapeuticsNULLCompleted18 Years74 YearsAll30Phase 2United States

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