- Active not recruitingPHASE2
Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension
This study is looking at a new medication, venglustat, for adults with a rare genetic condition called Gaucher disease, specifically types 1 and 3. Researchers want to see if venglustat, alone or with another drug called Cerezyme, is safe and helps improve symptoms.
For: Gaucher Disease Type 1 · Gaucher Disease Type 3United States · Germany · Japan - Active not recruitingPHASE3
Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3
This study looks at a new oral medicine, venglustat, for people aged 12 and over with Gaucher disease type 3. It compares it to the usual IV treatment (imiglucerase) to see how well it helps with brain-related symptoms and keeps the disease stable.
For: Gaucher's Disease Type IIIUnited States · Argentina · Canada