19. ライソゾーム病
［臨床試験数：784，薬物数：673（DrugBank：101），標的遺伝子数：68，標的パスウェイ数：184］
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.
| 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) | September 18, 2019 | 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 | ||
| 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 |
| 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] | Orphazyme A/S | NULL | NA | Female: yes Male: yes | 57 | Phase 2;Phase 3 | United States;Spain;Poland;Denmark;Germany;United Kingdom;Italy;Switzerland |