19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 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 | NCT01414985 (ClinicalTrials.gov) | April 15, 2010 | 13/12/2010 | AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis | Direct CNS Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human CLN2 cDNA to Children With LINCL With Uncommon Genotypes and/or Moderate to Severe Impairment | Late Infantile Neuronal Ceroid Lipofuscinosis;Batten Disease | Biological: AAVrh.10CUCLN2 | Weill Medical College of Cornell University | NULL | Completed | 3 Years | 18 Years | All | 8 | Phase 1/Phase 2 | United States |