Research.Patient.info
Gaucher's disease type I research hub
2 indexed studies · 1 currently recruiting
- recruitingTherapeutic confirmatory (Phase III)
Open label, two cohort (with and without imiglucerase), multicenter study to evaluate pharmacokinetics, safety, and efficacy of eliglustat in pediatric patients with Gaucher disease type 1 and type 3
This study is looking at a new medicine called eliglustat for children with Gaucher disease types 1 and 3. It wants to understand how the medicine works in their bodies, if it's safe, and if it helps improve their symptoms, sometimes alongside another treatment called Cerezyme.
Spain · France · Italy - 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.
United States · Argentina · Canada