19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
Showing 1 to 10 of 29 diseases
| 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 | EUCTR2018-002984-24-BE (EUCTR) | 11/02/2021 | 02/10/2020 | 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 Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosi ... | Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old - SCOB2 (Study Cystadrops® Ophthalmic for Patients Below 2 years) Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Tha ... | Nephropatic Cystinosis patients with corneal cystine crystal deposits MedDRA version: 20.0;Level: LLT;Classification code 10071112;Term: Nephropathic cystinosis;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] Nephropatic Cystinosis patients with corneal cystine crystal deposits MedDRA version: 20.0;Level: LL ... | Trade Name: Cystadrops Product Name: Cystadrops INN or Proposed INN: Cysteamine hydrochloride Other descriptive name: ß-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride Trade Name: Cystadrops Product Name: Cystadrops INN or Proposed INN: Cysteaminehydrochloride Other desc ... | Recordati Rare Diseases | NULL | Not Recruiting | Female: yes Male: yes | 5 | Phase 3 | France;Belgium;Germany;Italy;United Kingdom | ||
| 2 | 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 Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosi ... | Cystinosis | Drug: Mercaptamine | Recordati Rare Diseases | NULL | Recruiting | 6 Months | 2 Years | All | 5 | Phase 3 | Belgium;France;Germany;Italy;United Kingdom |
| 3 | 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 Multicentre, Ambispective, Observational, Real Life Study to Assess the Quality of Life Effectivenes ... | Nephropathic Cystinosis | Drug: Cysteamine Bitartrate | Chiesi SA/NV | NULL | Enrolling by invitation | N/A | N/A | All | 31 | Belgium | |
| 4 | EUCTR2018-002984-24-DE (EUCTR) | 30/06/2020 | 10/01/2020 | 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 Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosi ... | Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old - SCOB2 (Study Cystadrops® Ophthalmic for Patients Below 2 years) Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Tha ... | Nephropatic Cystinosis patients with corneal cystine crystal deposits MedDRA version: 20.0;Level: LLT;Classification code 10071112;Term: Nephropathic cystinosis;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] Nephropatic Cystinosis patients with corneal cystine crystal deposits MedDRA version: 20.0;Level: LL ... | Trade Name: Cystadrops Product Name: Cystadrops INN or Proposed INN: Cysteamine hydrochloride Other descriptive name: ß-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride Trade Name: Cystadrops Product Name: Cystadrops INN or Proposed INN: Cysteaminehydrochloride Other desc ... | Recordati Rare Diseases | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 3 | France;Germany;United Kingdom | ||
| 5 | EUCTR2018-002984-24-IT (EUCTR) | 24/06/2020 | 21/10/2020 | Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Tha ... | 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 Cystadrops® Ophthalmic for Patients Below 2 years) Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosi ... | Nephropatic Cystinosis patients with corneal cystine crystal deposits MedDRA version: 20.0;Level: LLT;Classification code 10071112;Term: Nephropathic cystinosis;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] Nephropatic Cystinosis patients with corneal cystine crystal deposits MedDRA version: 20.0;Level: LL ... | Trade Name: Cystadrops Product Name: Cystadrops Product Code: [045251016] INN or Proposed INN: Mercaptamina Other descriptive name: ß-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride Trade Name: Cystadrops Product Name: Cystadrops Product Code: [045251016] INN or Proposed INN: Mercapta ... | Recordati Rare Diseases | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 3 | France;Belgium;Germany;United Kingdom;Italy | ||
| 6 | 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 Cystinos ... | 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 Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosi ... | 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] Corneal cystine crystal deposits in pediatric patients withnephropathic cystinosis from 6 months to ... | Trade Name: Cystadrops Product Name: Cystadrops INN or Proposed INN: Mercaptamine hydrochloride Other descriptive name: Cysteamine hydrochloride Trade Name: Cystadrops Product Name: Cystadrops INN or Proposed INN: Mercaptaminehydrochloride Other de ... | RECORDATI Rare Diseases SARL | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 3 | France;United Kingdom | ||
| 7 | 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 cysteamineformulated in viscous solution in ... | Study of the preliminary efficacy and safety of topical cysteamine formulated in viscous solution in cystinosis patients - Viscou solution cysteamina Study of the preliminary efficacy and safety of topical cysteamineformulated in viscous solution in ... | 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] Cystinosis MedDRA version: 16.1;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ ... | Product Name: cysteamine (mercaptamine) viscous solution INN or Proposed INN: Mercaptamine Other descriptive name: MERCAPTAMINE HYDROCHLORIDE Product Name: cysteamine(mercaptamine) viscous solution INN or Proposed INN: Mercaptamine Other descri ... | Fundació Hospital Universitari Vall d'Hebron - Institut de Recerca (VHIR) | NULL | Not Recruiting | Female: yes Male: yes | Spain | ||||
| 8 | 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-rele ... | A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-release Capsules (RP103) in Patients withCystinosis - RP103-07 A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-rele ... | 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] Cystinosis MedDRA version: 16.1;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ ... | 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 Product Name: Cysteaminebitartrate(INN: mercaptaminebitartrate ) Product Code: RP103 INN or Proposed I ... | Raptor Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;United States;Belgium;Netherlands;Germany;United Kingdom;Italy | ||
| 9 | 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). Study comparing the effectiveness of cysteaminebitartrate delayed release capsules (RP103) to Cystag ... | 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 A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-rele ... | 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] Cystinosis MedDRA version: 20.0;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ ... | 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 Trade Name: PROCYSBI Product Name: Cysteaminebitartrate (INN: mercaptaminebitartrate ) Product Code: R ... | Horizon Pharma USA, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;United States;Belgium;Netherlands;Germany;Italy;United Kingdom | ||
| 10 | 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 CysteamineBitartrate Delayed-rele ... | A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis - RP103-07 A Long-Term Open-Label, Safety and Superior Effectiveness Study of CysteamineBitartrate Delayed-rele ... | 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] Cystinosis MedDRA version: 14.1;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ ... | 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 Product Name: Cysteaminebitartrate (INN: mercaptaminebitartrate ) Product Code: RP103 INN or Proposed ... | Raptor Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;France;Belgium;Netherlands;Germany;United Kingdom;Italy |