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 | NCT01801709 (ClinicalTrials.gov) | June 2014 | 28/1/2013 | Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy | A Phase I/II, Open Labeled, Monocentric Study of Direct Intracranial Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human ARSA cDNA to Children With Metachromatic Leukodystrophy. | Metachromatic Leukodystrophy | Genetic: intracerebral administration of AAVrh.10cuARSA | Institut National de la Santé Et de la Recherche Médicale, France | European Leukodystrophy Association;Assistance Publique - Hôpitaux de Paris | Active, not recruiting | 6 Months | 5 Years | All | 5 | Phase 1/Phase 2 | France |