19. Lysosomal storage disease Clinical trials / Disease details

Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191

2 trials found

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
2	NCT02437253 (ClinicalTrials.gov)	May 2015	5/5/2015	Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI	Pilot Study of the Effect of Adalimumab on Physical Function and Musculoskeletal Disease in Mucopolysaccharidosis Types I, II and VI	Mucopolysaccharidosis Type I;Mucopolysaccharidosis Type II;Mucopolysaccharidosis Type VI	Drug: Adalimumab;Other: Placebo	Los Angeles Biomedical Research Institute	NULL	Completed	5 Years	N/A	All	2	Phase 1/Phase 2	United States