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 | EUCTR2012-002773-64-BE (EUCTR) | 21/01/2014 | 19/04/2013 | A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis | A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis - RP103-07 | Cystinosis MedDRA version: 14.1;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate ) Product Code: RP103 INN or Proposed INN: mercaptamine bitartrate Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate ) Product Code: RP103 INN or Proposed INN: mercaptamine bitartrate Trade Name: Cystagon 50mg Product Name: Cystagon INN or Proposed INN: MERCAPTAMINE BITARTRATE Trade Name: Cystagon 150mg Product Name: Cystagon INN or Proposed INN: MERCAPTAMINE BITARTRATE | Raptor Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;France;Belgium;Netherlands;Germany;United Kingdom;Italy | ||
| 2 | EUCTR2012-002773-64-NL (EUCTR) | 15/08/2013 | 01/11/2013 | Study comparing the effectiveness of cysteamine bitartrate delayed-release capsules (RP103) in Patients with Cystinosis (Adult and Children 12 years and older) | A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis - Superiority, Safety and Efficacy Study of RP103 in Cystinosis | Cystinosis MedDRA version: 20.0;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: PROCYSBI Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate) Product Code: RP103 INN or Proposed INN: mercaptamine bitartrate Trade Name: PROCYSBI Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate) Product Code: RP103 INN or Proposed INN: mercaptamine bitartrate Trade Name: Cystagon 50mg Product Name: Cystagon INN or Proposed INN: mercaptamine bitartrate Trade Name: Cystagon 150mg Product Name: Cystagon INN or Proposed INN: mercaptamine bitartrate | Horizon Pharma USA, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;United States;Belgium;Germany;Netherlands;Italy;United Kingdom | ||
| 3 | NCT01733316 (ClinicalTrials.gov) | January 31, 2013 | 16/11/2012 | Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis | A Long-Term, Open-Label, Safety, Tolerability and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients With Cystinosis | Cystinosis | Drug: RP103;Drug: Cystagon® | Horizon Pharma USA, Inc. | NULL | Completed | 12 Years | N/A | All | 41 | Phase 3 | United States;Belgium;France;Italy;Netherlands;United Kingdom |
| 4 | NCT01744782 (ClinicalTrials.gov) | December 20, 2012 | 5/12/2012 | Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis | An Open-Label, Safety and Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naïve Patients With Cystinosis | Cystinosis | Drug: RP103 | Horizon Pharma USA, Inc. | NULL | Completed | N/A | 6 Years | All | 17 | Phase 3 | United States;Brazil |
| 5 | EUCTR2010-018365-34-NL (EUCTR) | 14/12/2010 | 06/07/2010 | A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis | A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis - RP103-04 | Cystinosis MedDRA version: 20.0;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: PROCYSBI Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate ) Product Code: RP103 INN or Proposed INN: mercaptamine bitartrate Trade Name: PROCYSBI Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate ) Product Code: RP103 INN or Proposed INN: mercaptamine bitartrate | Horizon Pharma USA, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;United States;Netherlands | ||
| 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 | NCT01197378 (ClinicalTrials.gov) | August 27, 2010 | 3/9/2010 | Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) | A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients With Cystinosis | Cystinosis | Drug: Cysteamine Bitartrate Delayed-release Capsules | Horizon Pharma USA, Inc. | NULL | Completed | 1 Year | N/A | All | 60 | Phase 3 | United States;France;Netherlands |
| 7 | NCT01000961 (ClinicalTrials.gov) | June 2010 | 22/10/2009 | Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis | A Randomized, Crossover Pharmacokinetic and Pharmacodynamic Study to Determine the Safety and Efficacy of Cysteamine Bitartrate Delayed-release Capsules (RP103), Compared to Cystagon® in Patients With Nephropathic Cystinosis | Cystinosis | Drug: Cystagon® (Cysteamine Bitartrate);Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103) | Horizon Pharma USA, Inc. | NULL | Completed | 6 Years | N/A | All | 43 | Phase 3 | United States;France;Netherlands |
| 8 | NCT00872729 (ClinicalTrials.gov) | May 2009 | 27/3/2009 | Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis | A Pilot Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Cysteamine Bitartrate Delayed-release Capsules (RP103), Compared to Cysteamine Bitartrate (Cystagon®) in Patients With Nephropathic Cystinosis | Cystinosis | Drug: Cystagon®;Drug: RP103 | Horizon Pharma USA, Inc. | NULL | Completed | N/A | N/A | All | 9 | Phase 1;Phase 2 | United States |
| 9 | EUCTR2010-018365-34-FR (EUCTR) | 18/06/2010 | A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis - RP103-04 | A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis - RP103-04 | Cystinosis MedDRA version: 12.1;Level: LLT;Classification code 10011777;Term: Cystinosis | Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate ) Product Code: RP103 INN or Proposed INN: mercaptamine bitartrate | Raptor Therapeutics Inc. | NULL | NA | Female: yes Male: yes | 20 | Phase 3 | France;Netherlands |