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	NCT05206773 (ClinicalTrials.gov)	March 11, 2022	20/10/2021	A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Adult Participants With Fabry Disease	A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Adults With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months	Fabry Disease	Drug: Venglustat (GZ402671);Drug: Placebo	Genzyme, a Sanofi Company	NULL	Recruiting	18 Years	N/A	All	114	Phase 3	United States
2	NCT02843035 (ClinicalTrials.gov)	January 4, 2017	20/7/2016	Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension	A 4-part, Open-label, Multicenter, Multinational Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic, and Exploratory Efficacy of Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension	Gaucher Disease Type 1;Gaucher Disease Type 3	Drug: venglustat (GZ402671);Drug: imiglucerase	Genzyme, a Sanofi Company	NULL	Active, not recruiting	18 Years	N/A	All	13	Phase 2	United States;Germany;Japan;United Kingdom