19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 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 | NCT04273269 (ClinicalTrials.gov) | May 11, 2021 | 21/1/2020 | A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis | An Open-Label Adaptive-Design Study of Intracisternal Adenoassociated Viral Vector Serotype rh.10 Carrying the Human ß-Galactosidase cDNA for Treatment of GM1 Gangliosidosis | GM1 Gangliosidosis | Genetic: LYS-GM101 | LYSOGENE | NULL | Recruiting | N/A | 3 Years | All | 16 | Phase 1/Phase 2 | United States;United Kingdom;France |
| 2 | EUCTR2019-004949-32-FR (EUCTR) | 09/03/2020 | LYS-GM101 gene therapy trial in patients with GM1 gangliosidosis | An open-label adaptive-design study of intracisternal administration of adeno-associated viral vector serotype rh.10 carrying the human ß-galactosidase cDNA for the treatment of GM1 gangliosidosis | GM1 gangliosidosis MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: LYS-GM101 Product Code: LYS-GM101 INN or Proposed INN: adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-ßgal) Other descriptive name: Adeno-associated virus serotype rh.10 expressing beta-galactosidase | Lysogene | NULL | NA | Female: yes Male: yes | 18 | Phase 1;Phase 2 | United States;France;United Kingdom |