19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| 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 | NCT04246060 (ClinicalTrials.gov) | July 31, 2020 | 27/1/2020 | Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients | Multicentre, Ambispective, Observational, Real Life Study to Assess the Quality of Life Effectiveness of Extended Release (ER) Oral Cysteamine Therapy (Procysbi) in Belgian Patients Suffering From Nephropathic Cystinosis | Nephropathic Cystinosis | Drug: Cysteamine Bitartrate | Chiesi SA/NV | NULL | Enrolling by invitation | N/A | N/A | All | 31 | Belgium | |
| 2 | EUCTR2012-002773-64-IT (EUCTR) | 12/02/2014 | 21/11/2013 | A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-release Capsules (RP103) in Patients withCystinosis | A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-release Capsules (RP103) in Patients withCystinosis - RP103-07 | Cystinosis MedDRA version: 16.1;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 Product Name: Cystagon INN or Proposed INN: Cystagon Other descriptive name: Cysteamine bitartrate Trade Name: CYSTAGON Product Name: Cystagon INN or Proposed INN: Cystagon Other descriptive name: Cysteamine bitartrate | Raptor Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;United States;Belgium;Netherlands;Germany;United Kingdom;Italy | ||
| 3 | EUCTR2012-002773-64-GB (EUCTR) | 22/01/2014 | 26/02/2013 | Study comparing the effectiveness of cysteamine bitartrate delayed release capsules (RP103) to Cystagon in Patients with Cystinosis(Adults and Children 12 years and older). | A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine 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;Netherlands;Germany;Italy;United Kingdom | ||
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | EUCTR2012-002773-64-FR (EUCTR) | 03/06/2013 | 28/09/2015 | A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-release Capsules (RP103) in Patients withCystinosis | A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-release Capsules (RP103) in Patients withCystinosis - RP103-07 | Cystinosis MedDRA version: 18.0;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] | Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate ) Product Code: RP103 INN or Proposed INN: mercaptamine bitartrate Trade Name: Cystagon 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;Italy;United Kingdom | ||
| 7 | 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 |
| 8 | 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 |
| 9 | NCT01432561 (ClinicalTrials.gov) | September 2011 | 9/9/2011 | Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™ | Food-Effect on Bioavailability of Cystagon™ in Normal, Healthy Adults | Cystinosis;Nephropathic Cystinosis | Drug: Cysteamine bitartrate | University of California, San Diego | Raptor Pharmaceuticals Corp. | Completed | 18 Years | N/A | All | 8 | N/A | United States |
| 10 | 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 | ||
| 11 | EUCTR2009-017882-42-NL (EUCTR) | 19/10/2010 | 25/06/2010 | A Randomized, Crossover, Pharmacokinetic and PharmacodynamicStudy to Determine the Safety and Efficacy of Cysteamine BitartrateDelayed-release Capsules (RP103), Compared to Cystagon® in Patientswith Nephropathic Cystinosis | A Randomized, Crossover, Pharmacokinetic and PharmacodynamicStudy to Determine the Safety and Efficacy of Cysteamine BitartrateDelayed-release Capsules (RP103), Compared to Cystagon® in Patientswith Nephropathic Cystinosis | 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 Trade Name: Cystagon | Raptor Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Netherlands;France | |||
| 12 | 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 |
| 13 | EUCTR2009-017882-42-FR (EUCTR) | 12/08/2010 | 14/06/2010 | A Randomized, Crossover, Pharmacokinetic and PharmacodynamicStudy to Determine the Safety and Efficacy of Cysteamine BitartrateDelayed-release Capsules (RP103), Compared to Cystagon® in Patientswith Nephropathic Cystinosis | A Randomized, Crossover, Pharmacokinetic and PharmacodynamicStudy to Determine the Safety and Efficacy of Cysteamine BitartrateDelayed-release Capsules (RP103), Compared to Cystagon® in Patientswith Nephropathic Cystinosis | 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 INN or Proposed INN: mercaptamine bitartrate Trade Name: Cystagon | Raptor Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | Phase 3 | France;Netherlands | |||
| 14 | 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 |
| 15 | 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 |
| 16 | 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 |