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 | NCT03153319 (ClinicalTrials.gov) | June 5, 2017 | 11/5/2017 | Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I and II | Phase 1/2 Study of the Effect of Adalimumab on Physical Function and Musculoskeletal Disease in Mucopolysaccharidosis Types I and II | Mucopolysaccharidosis I;Mucopolysaccharidosis II | Drug: Adalimumab Injection [Humira];Drug: Saline Solution for Injection | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | NULL | Recruiting | 5 Years | N/A | All | 14 | Phase 1/Phase 2 | United States |