ARIMOCLOMOL ( DrugBank: Arimoclomol )
3 diseases
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 2 | Amyotrophic lateral sclerosis | 20 |
| 15 | Inclusion body myositis | 6 |
| 19 | Lysosomal storage disease | 9 |
2. Amyotrophic lateral sclerosis
Clinical trials : 624 / Drugs : 611 - (DrugBank : 160) / Drug target genes : 172 - Drug target pathways : 225
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2019-000374-39-DE (EUCTR) | 04/10/2019 | 28/05/2019 | Extension 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/S | NULL | Not Recruiting | Female: yes Male: yes | 231 | Phase 3 | United States;Spain;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Poland;Denmark;Germany;Netherlands;Sweden | ||
| 2 | EUCTR2019-000374-39-SE (EUCTR) | 26/09/2019 | 16/05/2019 | Extension 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/S | NULL | Not Recruiting | Female: yes Male: yes | 231 | Phase 3 | United States;Spain;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Poland;Denmark;Germany;Netherlands;Sweden | ||
| 3 | NCT03836716 (ClinicalTrials.gov) | September 19, 2019 | 7/2/2019 | Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial | Open 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 Trial | Amyotrophic Lateral Sclerosis | Drug: Arimoclomol | Orphazyme | NULL | Terminated | 18 Years | N/A | All | 120 | Phase 3 | United States;Belgium;Canada;France;Germany;Italy;Netherlands;Poland;Spain;Sweden;United Kingdom;Switzerland |
| 4 | EUCTR2019-000374-39-PL (EUCTR) | 05/09/2019 | 22/05/2019 | Extension 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/S | NULL | Not Recruiting | Female: yes Male: yes | 231 | Phase 3 | United States;Spain;Italy;Switzerland;United Kingdom;France;Canada;Poland;Belgium;Denmark;Germany;Netherlands;Sweden | ||
| 5 | EUCTR2019-000374-39-NL (EUCTR) | 27/08/2019 | 29/05/2019 | Extension 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/S | NULL | Not Recruiting | Female: yes Male: yes | 231 | Phase 3 | United States;Spain;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Poland;Denmark;Netherlands;Germany;Sweden | ||
| 6 | EUCTR2019-000374-39-ES (EUCTR) | 07/08/2019 | 11/06/2019 | Extension 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/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 231 | Phase 3 | United States;Spain;Switzerland;United Kingdom;Italy;France;Canada;Belgium;Poland;Denmark;Germany;Netherlands;Sweden | ||
| 7 | EUCTR2019-000374-39-IT (EUCTR) | 15/07/2019 | 27/01/2021 | Extension 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 - na | 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 APS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 231 | Phase 3 | United States;Spain;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Poland;Denmark;Germany;Netherlands;Sweden | ||
| 8 | EUCTR2019-000374-39-GB (EUCTR) | 15/07/2019 | 09/05/2019 | Extension 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/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 231 | Phase 3 | United States;Spain;United Kingdom;Italy;Switzerland;France;Canada;Belgium;Poland;Denmark;Germany;Netherlands;Sweden | ||
| 9 | EUCTR2018-000137-13-BE (EUCTR) | 04/12/2018 | 13/09/2018 | A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral Sclerosis | A 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/S | NULL | Not Recruiting | Female: yes Male: yes | 231 | Phase 3 | United States;France;Canada;Spain;Poland;Belgium;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | ||
| 10 | EUCTR2018-000137-13-NL (EUCTR) | 21/11/2018 | 18/09/2018 | A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral Sclerosis | A 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/S | NULL | Not Recruiting | Female: yes Male: yes | 231 | Phase 3 | France;United States;Canada;Spain;Poland;Belgium;Germany;Netherlands;United Kingdom;Switzerland;Italy;Sweden | ||
| 11 | EUCTR2018-000137-13-GB (EUCTR) | 19/11/2018 | 07/09/2018 | A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral Sclerosis | A 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/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 231 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Canada;Poland;Belgium;Spain;Netherlands;Germany;Italy;Switzerland;United Kingdom;Sweden | ||
| 12 | EUCTR2018-000137-13-SE (EUCTR) | 22/10/2018 | 07/09/2018 | A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral Sclerosis | A 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/S | NULL | Not Recruiting | Female: yes Male: yes | 231 | Phase 3 | United States;France;Canada;Spain;Poland;Belgium;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | ||
| 13 | EUCTR2018-000137-13-ES (EUCTR) | 19/10/2018 | 09/10/2018 | A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral Sclerosis | A 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/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 231 | Phase 3 | France;United States;Canada;Poland;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden | ||
| 14 | EUCTR2018-000137-13-PL (EUCTR) | 18/10/2018 | 02/10/2018 | A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral Sclerosis | A 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/S | NULL | Not Recruiting | Female: yes Male: yes | 231 | Phase 3 | United States;France;Canada;Spain;Belgium;Poland;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | ||
| 15 | EUCTR2018-000137-13-IT (EUCTR) | 08/10/2018 | 22/01/2021 | A study to compare the use of Arimoclomol with placebo in patients withAmyotrophic Lateral Sclerosis | A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis - na | 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 APS | NULL | Not Recruiting | Female: yes Male: yes | 231 | Phase 3 | France;United States;Canada;Spain;Poland;Belgium;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | ||
| 16 | EUCTR2018-000137-13-FR (EUCTR) | 05/10/2018 | 06/09/2018 | A study to compare the use of Arimoclomol with placebo in patients with Amyotrophic Lateral Sclerosis | A 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/S | NULL | Not Recruiting | Female: yes Male: yes | 231 | Phase 3 | United States;France;Canada;Spain;Poland;Belgium;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | ||
| 17 | NCT03491462 (ClinicalTrials.gov) | July 31, 2018 | 27/3/2018 | Arimoclomol in Amyotropic Lateral Sclerosis | A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral Sclerosis | Amyotrophic Lateral Sclerosis | Drug: Arimoclomol;Drug: Placebo oral capsule | Orphazyme | NULL | Completed | 18 Years | N/A | All | 245 | Phase 3 | United States;Belgium;Canada;France;Germany;Italy;Netherlands;Poland;Spain;Sweden;Switzerland;United Kingdom;Denmark |
| 18 | NCT00706147 (ClinicalTrials.gov) | January 2009 | 24/6/2008 | Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis | Phase II/III Randomized, Placebo-Controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis (ALS) | Amyotrophic Lateral Sclerosis | Drug: Arimoclomol;Drug: Placebo | University of Miami | Massachusetts General Hospital | Completed | 18 Years | N/A | All | 38 | Phase 2/Phase 3 | United States |
| 19 | NCT00561366 (ClinicalTrials.gov) | January 2008 | 16/11/2007 | A Multicenter, Double-Blind Study to Investigate the Safety and Efficacy of Arimoclomol in Volunteers With ALS | A 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 Sclerosis | Drug: Placebo;Drug: Arimoclomol | CytRx | NULL | Withdrawn | 18 Years | N/A | Both | 0 | Phase 2 | United States;Canada |
| 20 | NCT00244244 (ClinicalTrials.gov) | October 2005 | 25/10/2005 | A Multicenter, Dose Ranging Safety and Pharmacokinetics Study of Arimoclomol in ALS | A Multicenter, Dose Ranging Safety and Pharmacokinetics Study of Arimoclomol in Amyotrophic Lateral Sclerosis (ALS) | Amyotrophic Lateral Sclerosis (ALS) | Drug: arimoclomol | CytRx | NULL | Completed | 18 Years | N/A | Both | 80 | Phase 2 | United States |
15. Inclusion body myositis
Clinical trials : 42 / Drugs : 60 - (DrugBank : 16) / Drug target genes : 12 - Drug target pathways : 123
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2019-000749-11-GB (EUCTR) | 09/10/2019 | 20/06/2019 | An 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 | An 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/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 130 | Phase 3 | United States;United Kingdom | ||
| 2 | NCT04049097 (ClinicalTrials.gov) | May 20, 2019 | 1/4/2019 | Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial | An 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 | Inclusion Body Myositis | Drug: Arimoclomol | Orphazyme | University of Kansas Medical Center;University College, London | Completed | 45 Years | N/A | All | 121 | Phase 3 | United States;United Kingdom |
| 3 | EUCTR2017-004903-33-GB (EUCTR) | 18/06/2018 | 05/04/2018 | Study 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/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 150 | Phase 2;Phase 3 | United States;United Kingdom | ||
| 4 | NCT02753530 (ClinicalTrials.gov) | August 16, 2017 | 25/4/2016 | Study of Arimoclomol in Inclusion Body Myositis (IBM) | Phase II Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis (IBM) | Inclusion Body Myositis | Drug: Arimoclomol;Other: Placebo | Orphazyme | University of Kansas Medical Center;University College, London | Completed | 45 Years | N/A | All | 152 | Phase 2 | United States;United Kingdom |
| 5 | EUCTR2008-008208-42-GB (EUCTR) | 26/06/2009 | 29/05/2009 | A 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 | A 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 | Product Name: Arimoclomol Product Code: BRX-345 INN or Proposed INN: Arimoclomol Other descriptive name: Arimoclomol | UCL - The Joint UCLH/UCL Comprehensive Biomedical Research Unit | NULL | Not Recruiting | Female: yes Male: yes | 24 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 6 | NCT00769860 (ClinicalTrials.gov) | September 2008 | 8/10/2008 | Arimoclomol in Sporadic Inclusion Body Myositis | Safety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body Myositis | Inclusion Body Myositis | Drug: Arimoclomol;Other: Placebo | Richard Barohn, MD | NULL | Completed | 50 Years | N/A | All | 24 | Phase 2/Phase 3 | United States;United Kingdom |
19. Lysosomal storage disease
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04316637 (ClinicalTrials.gov) | March 9, 2022 | 18/3/2020 | Early Access Program With Arimoclomol in US Patients With NPC | Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US | Niemann-Pick Disease, Type C | Drug: Arimoclomol | Orphazyme | NULL | Available | 2 Years | N/A | All | United States | ||
| 2 | NCT03746587 (ClinicalTrials.gov) | June 6, 2018 | 1/11/2018 | Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3 | Multicentre Double-blinded, Randomized Placebo-controlled Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3 | Gaucher Disease, Type 1;Gaucher Disease, Type 3 | Drug: Arimoclomol;Drug: Placebo oral capsule | Orphazyme | NULL | Active, not recruiting | 4 Years | 60 Years | All | 39 | Phase 2 | India |
| 3 | EUCTR2015-004438-93-IT (EUCTR) | 08/11/2016 | 19/05/2016 | A 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 ApS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 46 | Phase 2;Phase 3 | Switzerland;Italy;France;United States;Poland;Spain;Denmark;Germany;United Kingdom | ||
| 4 | EUCTR2015-004438-93-DE (EUCTR) | 04/10/2016 | 10/05/2016 | A 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 (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/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 57 | Phase 2;Phase 3 | United States;France;Spain;Poland;Denmark;Germany;United Kingdom;Switzerland;Italy | ||
| 5 | EUCTR2015-004438-93-ES (EUCTR) | 20/08/2016 | 15/07/2016 | A 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 ApS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 46 | Phase 2;Phase 3 | United States;Spain;Denmark;Switzerland | ||
| 6 | NCT02612129 (ClinicalTrials.gov) | June 14, 2016 | 12/11/2015 | Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C | Arimoclomol Prospective Doubleblind, Randomised, Placebo-controlled Study in Patients Diagnosed With NiemannPick Disease Type C | Niemann-Pick Disease, Type C | Drug: arimoclomol;Drug: Placebo | Orphazyme | NULL | Active, not recruiting | 2 Years | 18 Years | All | 50 | Phase 2/Phase 3 | United States;Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom |
| 7 | EUCTR2015-004438-93-GB (EUCTR) | 16/05/2016 | 24/10/2016 | A 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/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 57 | Phase 2;Phase 3 | United States;Spain;Poland;Denmark;Germany;Italy;Switzerland;United Kingdom | ||
| 8 | EUCTR2015-004438-93-DK (EUCTR) | 09/05/2016 | 11/03/2016 | A 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/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 57 | Phase 2;Phase 3 | United States;Spain;Poland;Denmark;Germany;United Kingdom;Italy;Switzerland | ||
| 9 | EUCTR2015-004438-93-PL (EUCTR) | 31/05/2016 | A 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: _ Product Name: Arimoclomol Product Code: _ INN or Proposed INN: Arimoclomol Other descriptive name: _ Product Name: Arimoclomol Product Code: _ INN or Proposed INN: ARIMOCLOMOL | Orphazyme A/S | NULL | NA | Female: yes Male: yes | 57 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Poland;Denmark;Germany;United Kingdom;Italy;Switzerland |