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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
1EUCTR2017-003083-13-GB
(EUCTR)		02/03/201811/01/2018Phase 2 study to evaluate Long-Term Safety and Efficacy of AX 250 in Patients with MPS Type IIIBA Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPSIIIB)
MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		Product Name: N/A
Product Code: AX 250
INN or Proposed INN: Not available
Other descriptive name: RHNAGLU-IGF2		Allievex CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		33Phase 2United States;Taiwan;Spain;Turkey;Germany;Colombia;United Kingdom
2EUCTR2017-003083-13-ES
(EUCTR)		27/12/201727/11/2017Phase 2 study to evaluate Long-Term Safety and Efficacy of BMN 250 in Patients with MPS Type IIIBA Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular BMN 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPSIIIB)
MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		Product Name: N/A
Product Code: BMN 250
INN or Proposed INN: Not available
Other descriptive name: RHNAGLU-IGF2		BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes		33Phase 2Germany;Colombia;United Kingdom;United States;Taiwan;Argentina;Spain;Turkey;Australia
3EUCTR2015-001985-25-DE
(EUCTR)		22/03/201616/11/2015Phase 1/2 Study to Evaluate the Safety and Efficacy of AX 250 in Patients with MPS IIIBA Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB)
MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		Product Name: N/A
Product Code: AX 250
INN or Proposed INN: Not available
Other descriptive name: RHNAGLU-IGF2		Allievex CorporationNULLNot RecruitingFemale: yes
Male: yes		33Phase 1;Phase 2Taiwan;Spain;Turkey;Australia;Colombia;Germany;United Kingdom
4EUCTR2015-001985-25-ES
(EUCTR)		02/03/201613/01/2016Phase 1/2 Study to Evaluate the Safety and Efficacy of BMN 250 in Patients with MPS IIIBA Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB)
MedDRA version: 18.1;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		Product Name: N/A
Product Code: BMN 250
INN or Proposed INN: Not available
Other descriptive name: RHNAGLU-IGF2		BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes		33Phase 1;Phase 2Taiwan;Brazil;Spain;Turkey;Australia;Germany;Colombia;United Kingdom
5EUCTR2015-001985-25-GB
(EUCTR)		06/11/201530/09/2015Phase 1/2 Study to Evaluate the Safety and Efficacy of AX 250 in Patients with MPS IIIBA Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB)
MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		Product Name: N/A
Product Code: AX 250
INN or Proposed INN: Not available
Other descriptive name: RHNAGLU-IGF2		Allievex CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		33Phase 1;Phase 2Taiwan;Spain;Turkey;Australia;Germany;Colombia;United Kingdom

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