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-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 | 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) | 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] | Trade Name: Cystadrops Product Name: Cystadrops Product Code: [045251016] INN or Proposed INN: Mercaptamina Other descriptive name: ß-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride | Recordati Rare Diseases | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 3 | France;Belgium;Germany;United Kingdom;Italy |