19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| 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 | NCT05039866 (ClinicalTrials.gov) | August 16, 2021 | 20/8/2021 | Long-Term Follow-up of Subjects Who Were Treated With ST-920 | Long-Term Follow-up of Fabry Disease Subjects Who Were Treated With ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy | Fabry Disease;Fabry Disease, Cardiac Variant | Biological: ST-920 | Sangamo Therapeutics | NULL | Enrolling by invitation | 18 Years | N/A | All | 48 | United States | |
| 2 | EUCTR2019-000667-24-GB (EUCTR) | 12/11/2019 | 19/03/2019 | A Phase I/II Study to Assess the Safety and Tolerability of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects with Fabry Disease | A Phase I/II, Multicenter, Open-Label, SingleDose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease | Fabry Disease (X-linked lysosomal storage disease) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A Product Code: ST-920 INN or Proposed INN: Not yet assigned Other descriptive name: Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA | Sangamo Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 30 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Kingdom | ||
| 3 | NCT04046224 (ClinicalTrials.gov) | July 23, 2019 | 1/8/2019 | Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease | A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects With Fabry Disease | Fabry Disease | Biological: ST-920 | Sangamo Therapeutics | NULL | Recruiting | 18 Years | N/A | All | 48 | Phase 1/Phase 2 | United States;United Kingdom |