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 | NCT04125927 (ClinicalTrials.gov) | September 1, 2020 | 3/10/2019 | Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2) | Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients From 6 Months to Less Than 2 Years Old | Cystinosis | Drug: Mercaptamine | Recordati Rare Diseases | NULL | Recruiting | 6 Months | 2 Years | All | 5 | Phase 3 | Belgium;France;Germany;Italy;United Kingdom |
| 2 | EUCTR2018-002984-24-GB (EUCTR) | 25/10/2019 | 15/07/2019 | Open-label, Single-arm, Multicenter Study to Assess the Safety of, Cystadrops® in Pediatric Cystinosis Patients,from 6 Months to Less Than 2 Years Old [SCOB2 Study] | Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old [SCOB2 Study] - SCOB2 | Corneal cystine crystal deposits in pediatric patients withnephropathic cystinosis from 6 months to less than 2 years old;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Trade Name: Cystadrops Product Name: Cystadrops INN or Proposed INN: Mercaptamine hydrochloride Other descriptive name: Cysteamine hydrochloride | RECORDATI Rare Diseases SARL | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 3 | France;United Kingdom | ||
| 3 | EUCTR2013-003228-35-ES (EUCTR) | 16/07/2014 | 12/11/2013 | Study of the preliminary efficacy and safety of topical cysteamine formulated in viscous solution in cystinosis patients | Study of the preliminary efficacy and safety of topical cysteamine formulated in viscous solution in cystinosis patients - Viscou solution cysteamina | 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: cysteamine (mercaptamine) viscous solution INN or Proposed INN: Mercaptamine Other descriptive name: MERCAPTAMINE HYDROCHLORIDE | Fundació Hospital Universitari Vall d'Hebron - Institut de Recerca (VHIR) | NULL | Not Recruiting | Female: yes Male: yes | Spain | ||||
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | EUCTR2009-012564-13-FR (EUCTR) | 03/01/2013 | 19/06/2013 | Cysteamine Hydrochloride for nephrOpathic Cystinosis, open-label Phase III pivotal study | Cysteamine Hydrochloride for nephropathic Cystinosis, open-label Phase III pivotal study - CYSTADROPS CHOC study | Nephropatic cystinosis patients with cystine corneal deposits MedDRA version: 16.0;Level: LLT;Classification code 10071112;Term: Nephropathic cystinosis;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: CYSTADROPS 0.55% eye drops, solution INN or Proposed INN: CYSTEAMINE HYDROCHLORIDE Other descriptive name: Mercaptamine hydrochloride | Orphan Europe SARL | NULL | Not Recruiting | Female: yes Male: yes | Phase 3 | France | |||
| 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 | EUCTR2010-020098-18-FR (EUCTR) | 09/09/2010 | 12/07/2010 | CrYSTobs A cohort of patients with cystinosis : compliance to cysteamine and neurological complicationsAn auxiliary study to Raptor RP103 03 and RP103 04 - CrYSTobs | CrYSTobs A cohort of patients with cystinosis : compliance to cysteamine and neurological complicationsAn auxiliary study to Raptor RP103 03 and RP103 04 - CrYSTobs | nephropatic cystinosis MedDRA version: 12.1;Level: LLT;Classification code 10011777;Term: Cystinosis | Trade Name: CYSTAGON Product Name: CYSTAGON INN or Proposed INN: MERCAPTAMINE BITARTRATE Other descriptive name: CYSTEAMINE Product Name: RP 103 INN or Proposed INN: MERCAPTAMINE BITARTRATE Other descriptive name: CYSTEAMINE Product Name: RP 103 INN or Proposed INN: MERCAPTAMINE BITARTRATE Other descriptive name: CYSTEAMINE | HOSPICES CIVILS DE LYON | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | France | ||||
| 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 | EUCTR2007-006024-35-FR (EUCTR) | 04/02/2008 | 12/12/2007 | Adaptive dose regimen of Cystadrops for cOrneal Crystal deposiTs and ocular manifestations in nephropathic cystinosis : an open label, dose-response pilot study - CYSTADROPS OCT-1 pilot study | Adaptive dose regimen of Cystadrops for cOrneal Crystal deposiTs and ocular manifestations in nephropathic cystinosis : an open label, dose-response pilot study - CYSTADROPS OCT-1 pilot study | Nephropathic cystinotic patients with cystine crystals corneal deposits MedDRA version: 9.1;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders | Product Name: CYSTADROPS INN or Proposed INN: cysteamine hydrochloride Other descriptive name: mercaptamine, mercamine, 2-aminoethanethiol | Orphan Europe SARL | NULL | Not Recruiting | Female: yes Male: yes | France | ||||
| 15 | 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 |