307. カナバン病 Canavan disease Clinical trials / Disease details
臨床試験数 : 5 / 薬物数 : 9 - (DrugBank : 3) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 2
| 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 | NCT04998396 (ClinicalTrials.gov) | September 8, 2021 | 5/8/2021 | A Study of AAV9 Gene Therapy in Participants With Canavan Disease | A Phase 1/2 Open-Label Study of the Safety and Clinical Activity of Gene Therapy for Canavan Disease Through Administration of an Adeno-Associated Virus (AAV) Serotype 9-Based Recombinant Vector Encoding the Human ASPA Gene | Canavan Disease;Demyelinating Diseases;Genetic Diseases, Inborn;Metabolism, Inborn Errors;Metabolic Disease;Neurological Diseases or Conditions;Leukodystrophy;Leukoencephalopathies;Neurodevelopmental Disorders;Neurodegenerative Diseases | Biological: AAV9 BBP-812 | Aspa Therapeutics | NULL | Recruiting | N/A | 30 Months | All | 18 | Phase 1/Phase 2 | United States |