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-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 | 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 INN or Proposed INN: Cysteamine hydrochloride Other descriptive name: ß-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride | Recordati Rare Diseases | NULL | Not Recruiting | Female: yes Male: yes | 5 | Phase 3 | France;Belgium;Germany;Italy;United Kingdom | ||
| 2 | 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 | 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 INN or Proposed INN: Cysteamine hydrochloride 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;Germany;United Kingdom | ||
| 3 | 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 | ||
| 4 | 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 |