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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"
11 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2015-000891-85-GB
(EUCTR)		05/09/201706/03/2017Phase 2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2A Phase 2, Open-Label, Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 years of age with CLN2 Disease Neuronal Ceroid Lipofuscinosis type 2 (CLN2) disease
MedDRA version: 20.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		BioMarin Pharmaceutical IncNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes		10Phase 2United States;Germany;Italy;United Kingdom
2NCT02678689
(ClinicalTrials.gov)		February 201615/1/2016A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 DiseaseA Phase 2, Open-Label, Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 DiseaseJansky-Bielschowsky Disease;Batten Disease;Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2;CLN2 Disease;CLN2 DisorderBiological: BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1);Device: Intraventricular access deviceBioMarin PharmaceuticalNULLActive, not recruitingN/A17 YearsAll14Phase 2United States;Germany;Italy;United Kingdom
3EUCTR2015-000891-85-DE
(EUCTR)		19/01/201604/11/2015Phase 2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2A Phase 2 Open-Label Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 years of age with CLN2 Disease Neuronal Ceroid Lipofuscinosis type 2 (CLN2) disease
MedDRA version: 20.0;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000157084;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		Trade Name: Brineura
Product Name: cerliponase alfa
Product Code: BMN 190
INN or Proposed INN: cerliponase alfa
Other descriptive name: recombinant human tripeptidyl peptidase-1		BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		10Phase 2Germany;Italy;United Kingdom
4EUCTR2014-003480-37-IT
(EUCTR)		06/05/201507/01/2015An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 DiseaseA Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2).
MedDRA version: 17.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		Product Code: BMN 190
INN or Proposed INN: TBD
Other descriptive name: RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1)		BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		22United States;Germany;United Kingdom;Japan;Italy
5EUCTR2014-003480-37-GB
(EUCTR)		10/03/201520/10/2014An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 DiseaseA Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease Late-Infantile Neuronal Ceroid Lipofuscinosis type 2 (CLN2).
MedDRA version: 20.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes		23Phase 2United States;Germany;Italy;United Kingdom
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
6NCT02485899
(ClinicalTrials.gov)		February 201524/4/2015A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 DiseaseA Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 DiseaseJansky-Bielschowsky Disease;Batten Disease;Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2;CLN2 Disease;CLN2 DisorderBiological: BMN 190;Device: Intraventricular Access DeviceBioMarin PharmaceuticalNULLActive, not recruiting3 Years16 YearsAll23Phase 1;Phase 2United States;Germany;Italy;United Kingdom
7EUCTR2014-003480-37-DE
(EUCTR)		23/01/201503/11/2014An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 DiseaseA Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease Late-Infantile Neuronal Ceroid Lipofuscinosis type 2(CLN2).
MedDRA version: 20.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes		23Phase 2United States;Germany;Italy;United Kingdom
8EUCTR2012-005430-11-IT
(EUCTR)		14/05/201420/01/2014Phase 1/2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease Neuronal Ceroid Lipofuscinosis Type 2 (CLN2).
MedDRA version: 16.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		Product Name: N/A
Product Code: BMN 190
INN or Proposed INN: TBD
Other descriptive name: recombinant human tripeptidyl peptidase-1		BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes		22Phase 1;Phase 2Germany;United Kingdom;Italy
9EUCTR2012-005430-11-DE
(EUCTR)		11/09/201310/04/2013Phase 1/2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients with Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2)
MedDRA version: 17.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		Product Name: N/A
Product Code: BMN 190
INN or Proposed INN: TBD
Other descriptive name: recombinant human tripeptidyl peptidase-1		BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes		22Phase 1;Phase 2Germany;United Kingdom
10NCT01907087
(ClinicalTrials.gov)		September 201319/7/2013A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) DiseaseA Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) DiseaseJansky-Bielschowsky Disease;Batten Disease;Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2;CLN2 DiseaseBiological: BMN 190BioMarin PharmaceuticalNULLCompleted3 Years15 YearsAll24Phase 1;Phase 2United States;Germany;Italy;United Kingdom
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
11EUCTR2012-005430-11-GB
(EUCTR)		15/08/201308/04/2013Phase 1/2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients with Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2).
MedDRA version: 18.0;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes		24Phase 1;Phase 2United States;Germany;Italy;United Kingdom

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