19. Lysosomal storage disease Clinical trials / Disease details
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 |