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 | 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 | ||
| 2 | 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 | ||||
| 3 | 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 |