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Arimoclomol citrate    (DrugBank: Arimoclomol)

3 diseases
IDDisease name (Link within this page)Number of trials
2Amyotrophic lateral sclerosis0
15Inclusion body myositis2
19Lysosomal storage disease3

2. Amyotrophic lateral sclerosis    [ 508 clinical trials,   530 drugs,   (DrugBank: 146 drugs),   170 drug target genes,   221 drug target pathways]
Searched query = "Amyotrophic lateral sclerosis", "ALS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.
0 / 508 trial found

15. Inclusion body myositis    [ 39 clinical trials,   32 drugs,   (DrugBank: 11 drugs),   11 drug target genes,   119 drug target pathways]
Searched query = "Inclusion body myositis"
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.
2 / 39 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2019-000749-11-GB
(EUCTR)		09/10/201920/06/2019An open-label, non-randomized trial to investigate the efficacy and safety of early versus delayed start of arimoclomol in patients with sporadic inclusion body myositis who have completed the IBM4809 trialAn open-label, non-randomized trial to investigate the efficacy and safety of early versus delayed start of arimoclomol in patients with sporadic inclusion body myositis who have completed the IBM4809 trial. Sporadic Inclusion Body Myositis (sIBM)
MedDRA version: 21.1;Level: LLT;Classification code 10075052;Term: Sporadic inclusion body myositis;System Organ Class: 100000004859;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]		Product Name: Arimoclomol
Product Code: BRX-345
INN or Proposed INN: ARIMOCLOMOL
Other descriptive name: Arimoclomol citrate		Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		130Phase 3United States;United Kingdom
2EUCTR2017-004903-33-GB
(EUCTR)		18/06/201805/04/2018Study of Arimoclomol in patients with Inclusion Body Myositis (IBM)Phase 2/3 Study of Arimoclomol in Inclusion Body Myositis (IBM)A Randomized, Double-blind, Placebo-Controlled Trial Sporadic Inclusion Body Myositis (sIBM)
MedDRA version: 21.1;Level: LLT;Classification code 10075052;Term: Sporadic inclusion body myositis;System Organ Class: 100000004859;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]		Product Name: Arimoclomol
Product Code: BRX-345
INN or Proposed INN: ARIMOCLOMOL
Other descriptive name: Arimoclomol citrate 		Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		150Phase 2;Phase 3United States;United Kingdom

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.
3 / 784 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2015-004438-93-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
2EUCTR2015-004438-93-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
3EUCTR2015-004438-93-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

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