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"
14 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03910621
(ClinicalTrials.gov)
April 2, 20208/3/2019Safety and Efficacy of Miglustat in Chinese NPC PatientsA Single Arm Uncontrolled 12 Months Clinical Study to Evaluate the Safety and Efficacy of Miglustat (Zavesca) for the Treatment of Niemann Pick Type C Disease (NPC) in Chinese SubjectsNiemann-Pick Disease, Type CDrug: MiglustatActelionNULLRecruiting4 YearsN/AAll18Phase 4China
2EUCTR2014-005194-37-DE
(EUCTR)
19/10/201528/04/2015A non-therapeutic study in patients with Niemann-Pick disease type C in order to characterise the individual patient disease profile and historic signo-symptomatology progression patternA prospective non-therapeutic study in patients diagnosed with Niemann-Pick disease type C in order to characterise the individual patient disease profile and historic signo-symptomatology progression pattern - Characterisation of Niemann-Pick disease type C Niemann-Pick disease - type C
MedDRA version: 18.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: MIglustat
Product Name: Zavesca
Product Code: A16AX06
INN or Proposed INN: MIGLUSTAT
Orphazyme ApSNULLNot RecruitingFemale: yes
Male: yes
40Phase 1Spain;Germany;Switzerland
3EUCTR2014-005194-37-ES
(EUCTR)
13/07/201508/05/2015A non-therapeutic study in patients with Niemann-Pick disease type C in order to characterise the individual patient disease profile and historic signo-symptomatology progression patternA prospective non-therapeutic study in patients diagnosed with Niemann-Pick disease type C in order to characterise the individual patient disease profile and historic signo-symptomatology progression pattern - Characterisation of Niemann-Pick disease type C Niemann-Pick disease - type C
MedDRA version: 18.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: MIglustat
Product Name: Zavesca
Product Code: A16AX06
INN or Proposed INN: MIGLUSTAT
Orphazyme ApSNULLNot RecruitingFemale: yes
Male: yes
40Phase 1Spain;Germany;Switzerland
4EUCTR2007-005516-61-IT
(EUCTR)
06/02/200923/03/2009Multicenter randomized study to assess the efficacy and the safety of two therapeutic regimens(high dose of imiglucerase versus co-administration of imiglucerase and miglustat) in type I Gaucher disease patients who have not responded to previous treatment with low dose imiglucerase - NDMulticenter randomized study to assess the efficacy and the safety of two therapeutic regimens(high dose of imiglucerase versus co-administration of imiglucerase and miglustat) in type I Gaucher disease patients who have not responded to previous treatment with low dose imiglucerase - ND GAUCHER disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: CEREZYME
INN or Proposed INN: Imiglucerase
Trade Name: ZAVESCA
INN or Proposed INN: Miglustat
ISTITUTO GIANNINA GASLININULLNot RecruitingFemale: yes
Male: yes
Italy
5EUCTR2007-007164-20-ES
(EUCTR)
20/05/200821/02/2008Uso racional de los tratamientos por inhibición de sustrato y enzimático sustitutivo en pacientes con Enfermedad de Gaucher tipo 1Uso racional de los tratamientos por inhibición de sustrato y enzimático sustitutivo en pacientes con Enfermedad de Gaucher tipo 1 Tratamiento de la enfermedad de Gaucher
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: CEREZYME
INN or Proposed INN: IMIGLUCERASA
Trade Name: MIGLUSTAT (ZAVESCA)
INN or Proposed INN: MIGLUSTAT
INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUDNULLNot RecruitingFemale: yes
Male: yes
Spain
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2005-001651-37-HU
(EUCTR)
10/12/200704/10/2007Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease.Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 18 of the protocol:1BACKGROUND AND RATIONALE1.1Disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: Zavesca
Product Name: Miglustat
Product Code: OGT918
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot RecruitingFemale: yes
Male: yes
50Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy
7EUCTR2006-004661-34-FR
(EUCTR)
08/12/200618/10/2006Evaluation d’un traitement par miglustat (Zavesca®) chez les patients atteints de mucopolysaccharidose de type III (maladie de Sanfilippo).Essai thérapeutique de phase IIb randomisé en aveugle contre placebo. - ZAV-MPSIII 2006Evaluation d’un traitement par miglustat (Zavesca®) chez les patients atteints de mucopolysaccharidose de type III (maladie de Sanfilippo).Essai thérapeutique de phase IIb randomisé en aveugle contre placebo. - ZAV-MPSIII 2006 Mucopolysaccharidose de type III
MedDRA version: 8.1;Level: LLT;Classification code 10056890;Term: Mucopolysaccharidosis III
Trade Name: ZAVESCA
Product Name: ZAVESCA
INN or Proposed INN: miglustat
Other descriptive name: MIGLUSTAT
HOSPICES CIVILS DE LYONNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 2bFrance
8EUCTR2006-005842-35-IT
(EUCTR)
22/11/200628/10/2009Efficacy and safety of treatment with N-butyl-deoxynojirimycin (NB-DNJ-miglustat) in patients with Niemann-Pick disease type C. - NDEfficacy and safety of treatment with N-butyl-deoxynojirimycin (NB-DNJ-miglustat) in patients with Niemann-Pick disease type C. - ND NIEMANN-PICK DISEASE TYPE C
MedDRA version: 9.1;Level: LLT;Classification code 10029403
Trade Name: ZAVESCA
INN or Proposed INN: Miglustat
Dipartimento di Pediatria Universita` di Napoli Federico IINULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Italy
9EUCTR2005-001651-37-CZ
(EUCTR)
02/10/200617/08/2006Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. - MAINTENANCEOpen-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. - MAINTENANCE Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol:1BACKGROUND AND RATIONALE1.1Disease
MedDRA version: 8.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: Zavesca
Product Name: Miglustat
Product Code: OGT918
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot RecruitingFemale: yes
Male: yes
50Hungary;Germany;United Kingdom;Czech Republic;Spain;Italy
10EUCTR2005-001651-37-ES
(EUCTR)
12/04/200625/01/2006A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher DiseaseA phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol:1BACKGROUND AND RATIONALE1.1Disease Trade Name: Zavesca
Product Name: Miglustat
Product Code: OGT918
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot RecruitingFemale: yes
Male: yes
50Phase 4Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2005-001651-37-DE
(EUCTR)
28/02/200616/05/2006Open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance therapy after a switch from Enzyme Replacement Therapy in adult patients with stable Type 1 Gaucher disease - MAINTENANCEOpen-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance therapy after a switch from Enzyme Replacement Therapy in adult patients with stable Type 1 Gaucher disease - MAINTENANCE Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 18 of the protocol:1BACKGROUND AND RATIONALE1.1Disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: ZAVESCA
Product Name: Miglustat
Product Code: OGT918
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot RecruitingFemale: yes
Male: yes
50Hungary;Czech Republic;Spain;Germany;Italy;United Kingdom
12EUCTR2005-001651-37-IT
(EUCTR)
06/02/200614/03/2006A phase IV, open label, non comparative, multicenter study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch form enzyme replacement therapy ERT in adult patients with stable type 1 Gaucher disease - NDA phase IV, open label, non comparative, multicenter study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch form enzyme replacement therapy ERT in adult patients with stable type 1 Gaucher disease - ND Gaucher disease is the most common of the glycosphingolipids storage disease and has autosomal recessive inheritance.
MedDRA version: 6.1;Level: PT;Classification code 10018048
Trade Name: ZAVESCA 84CPS 100MG
INN or Proposed INN: Miglustat
Actelion Registration LtdNULLNot RecruitingFemale: yes
Male: yes
50Phase 4Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy
13NCT00418847
(ClinicalTrials.gov)
July 20044/1/2007Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 GangliosidosisPharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis: Single and Multiple Oral DosesGangliosidoses GM2Drug: miglustatThe Hospital for Sick ChildrenActelionCompleted6 Years20 YearsBoth5Phase 2Canada
14NCT00672022
(ClinicalTrials.gov)
July 20042/5/2008Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral DosesPharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset GM2 Gangliosidosis: Single and Steady State Oral DosesGM2 Gangliosidoses;Tay-Sachs;Sandhoff DiseaseDrug: Zavesca (Miglustat)Children's Research InstituteActelionCompleted6 Months5 YearsBoth10Phase 3United States