Arimoclomol    (DrugBank: Arimoclomol)

3 diseases
IDDisease name (Link within this page)Number of trials
2Amyotrophic lateral sclerosis18
15Inclusion body myositis6
19Lysosomal storage disease9

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
18 / 508 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2019-000374-39-DE
(EUCTR)
04/10/201928/05/2019Extension study of Arimoclomol in patients with amyotrophic lateral sclerosis.Open-label, Non-randomised Extension Trial to Assess the Long-Term Safety and Efficacy of 1200 mg/day Arimoclomol 400 mg Three Times a Day (t.i.d.) in Subjects with Amyotrophic Lateral Sclerosis (ALS) who have Completed the ORARIALS-01 Trial Amyotrophic Lateral Sclerosis
MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Arimoclomol
Product Code: BRX-345
INN or Proposed INN: ARIMOCLOMOL
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
231Phase 3United States;Spain;Switzerland;United Kingdom;Italy;France;Canada;Belgium;Poland;Denmark;Germany;Netherlands;Sweden
2EUCTR2019-000374-39-SE
(EUCTR)
26/09/201916/05/2019Extension study of Arimoclomol in patients with amyotrophic lateral sclerosis.Open-label, Non-randomised Extension Trial to Assess the Long-Term Safety and Efficacy of 1200 mg/day Arimoclomol 400 mg Three Times a Day (t.i.d.) in Subjects with Amyotrophic Lateral Sclerosis (ALS) who have Completed the ORARIALS-01 Trial Amyotrophic Lateral Sclerosis
MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Arimoclomol
Product Code: BRX-345
INN or Proposed INN: ARIMOCLOMOL
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
231Phase 3United States;Spain;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Poland;Denmark;Germany;Netherlands;Sweden
3NCT03836716
(ClinicalTrials.gov)
September 19, 20197/2/2019Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension TrialOpen Label, Non-randomized Extension Trial to Assess Long Term Safety and Efficacy of Arimoclomol in Subjects With Amyotropic Lateral Sclerosis Who Have Completed the ORARIALS-01 TrialAmyotrophic Lateral SclerosisDrug: ArimoclomolOrphazymeNULLEnrolling by invitation18 YearsN/AAll231Phase 3United States;Belgium;Canada;France;Germany;Italy;Netherlands;Poland;Spain;Sweden;Switzerland;United Kingdom
4EUCTR2019-000374-39-PL
(EUCTR)
05/09/201922/05/2019Extension study of Arimoclomol in patients with amyotrophic lateral sclerosis.Open-label, Non-randomised Extension Trial to Assess the Long-Term Safety and Efficacy of 1200 mg/day Arimoclomol 400 mg Three Times a Day (t.i.d.) in Subjects with Amyotrophic Lateral Sclerosis (ALS) who have Completed the ORARIALS-01 Trial Amyotrophic Lateral Sclerosis
MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Arimoclomol
Product Code: BRX-345
INN or Proposed INN: ARIMOCLOMOL
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
231Phase 3United States;Spain;Italy;Switzerland;United Kingdom;France;Canada;Poland;Belgium;Denmark;Germany;Netherlands;Sweden
5EUCTR2019-000374-39-NL
(EUCTR)
27/08/201929/05/2019Extension study of Arimoclomol in patients with amyotrophic lateral sclerosis.Open-label, Non-randomised Extension Trial to Assess the Long-Term Safety and Efficacy of 1200 mg/day Arimoclomol 400 mg Three Times a Day (t.i.d.) in Subjects with Amyotrophic Lateral Sclerosis (ALS) who have Completed the ORARIALS-01 Trial Amyotrophic Lateral Sclerosis
MedDRA version: 20.0;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Arimoclomol
Product Code: BRX-345
INN or Proposed INN: ARIMOCLOMOL
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
231Phase 3United States;Spain;Switzerland;United Kingdom;Italy;France;Canada;Belgium;Poland;Denmark;Netherlands;Germany;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2019-000374-39-ES
(EUCTR)
07/08/201911/06/2019Extension study of Arimoclomol in patients with amyotrophic lateral sclerosis.Open-label, Non-randomised Extension Trial to Assess the Long-Term Safety and Efficacy of 1200 mg/day Arimoclomol 400 mg Three Times a Day (t.i.d.) in Subjects with Amyotrophic Lateral Sclerosis (ALS) who have Completed the ORARIALS-01 Trial Amyotrophic Lateral Sclerosis
MedDRA version: 20.0;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Arimoclomol
Product Code: BRX-345
INN or Proposed INN: ARIMOCLOMOL
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
231Phase 3United States;Spain;Switzerland;United Kingdom;Italy;France;Canada;Belgium;Poland;Denmark;Germany;Netherlands;Sweden
7EUCTR2019-000374-39-GB
(EUCTR)
15/07/201909/05/2019Extension study of Arimoclomol in patients with amyotrophic lateral sclerosis.Open-label, Non-randomised Extension Trial to Assess the Long-Term Safety and Efficacy of 1200 mg/day Arimoclomol 400 mg Three Times a Day (t.i.d.) in Subjects with Amyotrophic Lateral Sclerosis (ALS) who have Completed the ORARIALS-01 Trial Amyotrophic Lateral Sclerosis
MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Arimoclomol
Product Code: BRX-345
INN or Proposed INN: ARIMOCLOMOL
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
231Phase 3United States;Spain;United Kingdom;Italy;Switzerland;France;Canada;Belgium;Poland;Denmark;Germany;Netherlands;Sweden
8EUCTR2018-000137-13-BE
(EUCTR)
04/12/201813/09/2018A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral SclerosisA Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis Amyotrophic Lateral Sclerosis
MedDRA version: 20.0;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
231Phase 3France;United States;Canada;Poland;Spain;Belgium;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden
9EUCTR2018-000137-13-NL
(EUCTR)
21/11/201818/09/2018A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral SclerosisA Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis Amyotrophic Lateral Sclerosis
MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Arimoclomol
Product Code: BRX-345
INN or Proposed INN: ARIMOCLOMOL
Orphazyme A/SNULLNot RecruitingFemale: yes
Male: yes
231Phase 3United States;France;Canada;Spain;Poland;Belgium;Germany;Netherlands;United Kingdom;Switzerland;Italy;Sweden
10EUCTR2018-000137-13-GB
(EUCTR)
19/11/201807/09/2018A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral SclerosisA Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis Amyotrophic Lateral Sclerosis
MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
231Phase 3France;United States;Canada;Poland;Belgium;Spain;Netherlands;Germany;Italy;Switzerland;United Kingdom;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2018-000137-13-SE
(EUCTR)
22/10/201807/09/2018A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral SclerosisA Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis Amyotrophic Lateral Sclerosis
MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Arimoclomol
Product Code: BRX-345
INN or Proposed INN: ARIMOCLOMOL
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
231Phase 3United States;France;Canada;Spain;Poland;Belgium;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden
12EUCTR2018-000137-13-ES
(EUCTR)
19/10/201809/10/2018A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral SclerosisA Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis Amyotrophic Lateral Sclerosis
MedDRA version: 20.0;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Arimoclomol
Product Code: BRX-345
INN or Proposed INN: ARIMOCLOMOL
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
231Phase 3France;United States;Canada;Poland;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden
13EUCTR2018-000137-13-PL
(EUCTR)
18/10/201802/10/2018A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral SclerosisA Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis Amyotrophic Lateral Sclerosis
MedDRA version: 20.0;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
231Phase 3France;United States;Canada;Belgium;Spain;Poland;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden
14EUCTR2018-000137-13-FR
(EUCTR)
05/10/201806/09/2018A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral SclerosisA Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis Amyotrophic Lateral Sclerosis
MedDRA version: 20.0;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Orphazyme A/SNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
231Phase 3United States;France;Canada;Poland;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden
15NCT03491462
(ClinicalTrials.gov)
July 31, 201827/3/2018Arimoclomol in Amyotropic Lateral SclerosisA Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral SclerosisAmyotrophic Lateral SclerosisDrug: Arimoclomol;Drug: Placebo oral capsuleOrphazymeNULLActive, not recruiting18 YearsN/AAll231Phase 3United States;Belgium;Canada;France;Germany;Italy;Netherlands;Poland;Spain;Sweden;Switzerland;United Kingdom;Denmark
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT00706147
(ClinicalTrials.gov)
January 200924/6/2008Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral SclerosisPhase II/III Randomized, Placebo-Controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis (ALS)Amyotrophic Lateral SclerosisDrug: Arimoclomol;Drug: PlaceboUniversity of MiamiMassachusetts General HospitalCompleted18 YearsN/AAll38Phase 2;Phase 3United States
17NCT00561366
(ClinicalTrials.gov)
January 200816/11/2007A Multicenter, Double-Blind Study to Investigate the Safety and Efficacy of Arimoclomol in Volunteers With ALSA Multicenter, Double-Blind, Placebo-Controlled, Phase 2b Study to Investigate the Safety and Efficacy of Arimoclomol in Volunteers With Amyotrophic Lateral Sclerosis (ALS)Amyotrophic Lateral SclerosisDrug: Placebo;Drug: ArimoclomolCytRxNULLWithdrawn18 YearsN/ABoth0Phase 2United States;Canada
18NCT00244244
(ClinicalTrials.gov)
October 200525/10/2005A Multicenter, Dose Ranging Safety and Pharmacokinetics Study of Arimoclomol in ALSA Multicenter, Dose Ranging Safety and Pharmacokinetics Study of Arimoclomol in Amyotrophic Lateral Sclerosis (ALS)Amyotrophic Lateral Sclerosis (ALS)Drug: arimoclomolCytRxNULLCompleted18 YearsN/ABoth80Phase 2United States

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.
6 / 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
2NCT04049097
(ClinicalTrials.gov)
May 20, 20191/4/2019Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension 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 TrialInclusion Body MyositisDrug: ArimoclomolOrphazymeUniversity of Kansas Medical Center;University College, LondonEnrolling by invitation45 YearsN/AAll150Phase 3United States;United Kingdom
3EUCTR2017-004903-33-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
4NCT02753530
(ClinicalTrials.gov)
August 9, 201725/4/2016Study of Arimoclomol in Inclusion Body Myositis (IBM)Phase II Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis (IBM)Inclusion Body MyositisDrug: Arimoclomol;Other: PlaceboOrphazymeUniversity of Kansas Medical Center;University College, LondonActive, not recruiting45 YearsN/AAll150Phase 2United States;United Kingdom
5EUCTR2008-008208-42-GB
(EUCTR)
26/06/200929/05/2009A Randomised, Double-Blind Placebo-Controlled Pilot Study Assessing the Safety and Tolerability of Arimoclomol in Adult with Sporadic Inclusion Body Myositis - Arimoclomol for Inclusion Body MyositisA Randomised, Double-Blind Placebo-Controlled Pilot Study Assessing the Safety and Tolerability of Arimoclomol in Adult with Sporadic Inclusion Body Myositis - Arimoclomol for Inclusion Body Myositis The research project proposes to investigate Sporadic Inclusion Body Myositis (IBM). IBM is the commonest acquired disease of muscle affecting people aged 50 years and over. It is a progressive and debilitating disease which causes both muscle weakness and wasting, characteristically of the quadriceps and finger flexors. Over time it can lead to severe disability, falls and swallowing impairment. IBM is currently without a clinically proven, effective pharmacological treatment.
MedDRA version: 9.1;Level: LLT;Classification code 10066407;Term: Inclusion body myositis
UCL - The Joint UCLH/UCL Comprehensive Biomedical Research UnitNULLNot Recruiting Female: yes
Male: yes
24Phase 2United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT00769860
(ClinicalTrials.gov)
September 20088/10/2008Arimoclomol in Sporadic Inclusion Body MyositisSafety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body MyositisInclusion Body MyositisDrug: Arimoclomol;Other: PlaceboRichard Barohn, MDNULLCompleted50 YearsN/AAll24Phase 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.
9 / 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
1NCT04316637
(ClinicalTrials.gov)
September 18, 201918/3/2020Early Access Program With Arimoclomol in US Patients With NPCEarly Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the USNiemann-Pick Disease, Type CDrug: ArimoclomolOrphazymeNULLAvailable2 YearsN/AAllUnited States
2NCT03746587
(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 capsuleOrphazymeNULLActive, not recruiting4 Years60 YearsAll39Phase 2India
3EUCTR2015-004438-93-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
4EUCTR2015-004438-93-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
5EUCTR2015-004438-93-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT02612129
(ClinicalTrials.gov)
June 14, 201612/11/2015Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type CArimoclomol Prospective Doubleblind, Randomised, Placebo-controlled Study in Patients Diagnosed With NiemannPick Disease Type CNiemann-Pick Disease, Type CDrug: arimoclomol;Drug: PlaceboOrphazymeNULLActive, not recruiting2 Years18 YearsAll50Phase 2;Phase 3United States;Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom
7EUCTR2015-004438-93-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
8EUCTR2015-004438-93-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
9EUCTR2015-004438-93-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]
Orphazyme A/SNULLNA Female: yes
Male: yes
57Phase 2;Phase 3United States;Spain;Poland;Denmark;Germany;United Kingdom;Italy;Switzerland