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 | EUCTR2005-004384-33-GB (EUCTR) | 19/12/2005 | 17/10/2005 | A Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients with Fabry Disease - Phase 2 study of AT1001 in Fabry Disease | A Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients with Fabry Disease - Phase 2 study of AT1001 in Fabry Disease | Fabry Disease MedDRA version: 8.1;Level: LLT;Classification code 10016016 | Product Name: 1- Deoxygalactonojirimycin Product Code: AT1001 INN or Proposed INN: N/A Other descriptive name: Migalastat hydrochloride | Amicus Therapeutics | NULL | Not Recruiting | Female: no Male: yes | 8 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom |