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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"
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
1NCT04281537
(ClinicalTrials.gov)		March 1, 20207/2/2020A 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 BetaFabry DiseaseDrug: Agalsidase Beta;Drug: Agalsidase AlphaAmicus TherapeuticsNULLActive, not recruiting18 YearsN/AAll120United States
2NCT02956954
(ClinicalTrials.gov)		March 25, 20173/11/2016Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry DiseaseFollow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease (AFD): Impact of Treatment by Agalsidase Alpha (Replagal®)Anderson-Fabry DiseaseDrug: Enzyme replacement therapy (Agalsidase alpha (Replagal®));Procedure: Magnetic Resonance ImagingUniversity Hospital, RouenNULLUnknown status18 YearsN/AAll25N/AFrance
3EUCTR2007-005543-22-SI
(EUCTR)		18/03/201124/02/2011An investigation of 3 dosages of Replagal in adult patients with Fabry diseaseA 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)NULLNot RecruitingFemale: yes
Male: yes		43United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom
4EUCTR2007-005543-22-FI
(EUCTR)		18/05/201023/03/2010An investigation of 3 dosages of Replagal in adult patients with Fabry diseaseA 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)NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		43Finland;United Kingdom;Czech Republic;Paraguay;Slovenia;United States;Australia
5EUCTR2007-005543-22-CZ
(EUCTR)		19/06/200802/01/2008An investigation of 3 dosages of Replagal in adult patients with Fabry diseaseA 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)NULLNot RecruitingFemale: yes
Male: yes		43United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom

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