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 | NCT03300453 (ClinicalTrials.gov) | September 17, 2013 | 15/6/2016 | Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome | Protocol AMT110-CD-001: A Phase I/II, Open-label, Study of Intracerebral Administration of Adeno-associated Viral Vector Containing the Human Alpha-N-acetylglucosaminidase cDNA in Children With Sanfilippo Type B Syndrome | Sanfilippo Syndrome B | Drug: rAAV2/5-hNAGLU | UniQure Biopharma B.V. | Venn Life Sciences;Institut Pasteur | Completed | 18 Months | 60 Months | All | 4 | Phase 1/Phase 2 | France |
| 2 | EUCTR2012-000856-33-FR (EUCTR) | 15/05/2014 | Intracerebral Gene therapy in children with Sanfilippo type B syndrome | A phase I/II, open-label, study of intracerebral administration of adeno-associated viral vector containing the human alpha-N-acetylglucosaminidase cDNA in children with Sanfilippo type B syndrome | Mucopolysaccharidosis III B MedDRA version: 17.0;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: rAAV2/5-hNaGlu | Institut Pasteur | NULL | NA | Female: yes Male: yes | Phase 1;Phase 2 | France |