19. Lysosomal storage disease Clinical trials / Disease details


Clinical trials : 899 Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182

  
7 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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PhaseCountries
1NCT03746587
(ClinicalTrials.gov)
June 6, 20181/11/2018Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3Multicentre Double-blinded, Randomized Placebo-controlled Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3Gaucher Disease, Type 1;Gaucher Disease, Type 3Drug: Arimoclomol;Drug: Placebo oral capsuleKemPharm Denmark A/SNULLTerminated4 Years60 YearsAll39Phase 2India
2EUCTR2015-004438-93-IT
(EUCTR)
08/11/201619/05/2016A clinical study to investigate the study drug Arimoclomol in a double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C.Arimoclomol prospective double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C - Investigate study drug Arimoclomol with patients diagnosed with Niemann Pick disease type C. Niemann Pick disease type C
MedDRA version: 19.0;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]
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: Arimoclomol
Other descriptive name: _
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: Arimoclomol
Other descriptive name: _
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: Arimoclomol
Other descriptive name: _
Orphazyme ApSNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
46Phase 2;Phase 3Switzerland;Italy;France;United States;Poland;Spain;Denmark;Germany;United Kingdom
3EUCTR2015-004438-93-DE
(EUCTR)
04/10/201610/05/2016A clinical study to investigate the study drug Arimoclomol in a double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type CArimoclomol prospective double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C - Investigate study drug Arimoclomol with patients diagnosed with Niemann Pick disease type C. Niemann Pick disease type C
MedDRA version: 20.0;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]
Product Name: Arimoclomol (hard gelatine capsule)
Product Code: _
INN or Proposed INN: Arimoclomol citrate
Product Name: Arimoclomol (hard gelatine capsule)
Product Code: _
INN or Proposed INN: Arimoclomol citrate
Product Name: Arimoclomol (hard gelatine capsule)
INN or Proposed INN: Arimoclomol citrate
Product Name: Arimoclomol (HPMC capsule)
Product Code: _
INN or Proposed INN: Arimoclomol citrate
Product Name: Arimoclomol (HPMC capsule)
Product Code: _
INN or Proposed INN: Arimoclomol citrate
Product Name: Arimoclomol (HPMC capsule)
Product Code: _
INN or Proposed INN: Arimoclomol citrate
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
57Phase 2;Phase 3United States;France;Spain;Poland;Denmark;Germany;United Kingdom;Switzerland;Italy
4EUCTR2015-004438-93-ES
(EUCTR)
20/08/201615/07/2016A clinical study to investigate the study drug Arimoclomol in a double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C.Arimoclomol prospective double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C - Investigate study drug Arimoclomol with patients diagnosed with Niemann Pick disease type C. Niemann Pick disease type C
MedDRA version: 19.0;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]
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: Arimoclomol
Other descriptive name: _
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: Arimoclomol
Other descriptive name: _
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: Arimoclomol
Other descriptive name: _
Orphazyme ApSNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
46Phase 2;Phase 3United States;Spain;Denmark;Switzerland
5EUCTR2015-004438-93-GB
(EUCTR)
16/05/201624/10/2016A clinical study to investigate the study drug Arimoclomol in a double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C.Arimoclomol prospective double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C - Investigate study drug Arimoclomol with patients diagnosed with Niemann Pick disease type C. Niemann Pick disease type C
MedDRA version: 20.0;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]
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: Arimoclomol
Other descriptive name: ARIMOCLOMOL CITRATE
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: Arimoclomol
Other descriptive name: ARIMOCLOMOL CITRATE
Product Name: Arimoclomol
INN or Proposed INN: ARIMOCLOMOL
Other descriptive name: ARIMOCLOMOL CITRATE
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
57Phase 2;Phase 3United States;Spain;Poland;Denmark;Germany;Italy;Switzerland;United Kingdom
6EUCTR2015-004438-93-DK
(EUCTR)
09/05/201611/03/2016A clinical study to investigate the study drug Arimoclomol in a double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C.Arimoclomol prospective double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C - Investigate study drug Arimoclomol with patients diagnosed with Niemann Pick disease type C. Niemann Pick disease type C
MedDRA version: 20.0;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]
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: Arimoclomol citrate
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: Arimoclomol citrate
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: ARIMOCLOMOL
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
57Phase 2;Phase 3United States;Spain;Poland;Denmark;Germany;United Kingdom;Italy;Switzerland
7EUCTR2015-004438-93-PL
(EUCTR)
31/05/2016A clinical study to investigate the study drug Arimoclomol in a double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C.Arimoclomol prospective double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C - Investigate study drug Arimoclomol with patients diagnosed with Niemann Pick disease type C. Niemann Pick disease type C
MedDRA version: 20.0;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]
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: Arimoclomol
Other descriptive name: ARIMOCLOMOL CITRATE
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: Arimoclomol
Other descriptive name: ARIMOCLOMOL CITRATE
Product Name: Arimoclomol
Product Code: _
INN or Proposed INN: ARIMOCLOMOL
Other descriptive name: ARIMOCLOMOL CITRATE
Orphazyme A/SNULLNot RecruitingFemale: yes
Male: yes
57Phase 2;Phase 3United States;Spain;Poland;Denmark;Germany;United Kingdom;Italy;Switzerland