- authorisedTherapeutic confirmatory (Phase III)
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients with Gaucher Disease Type 1
This clinical trial is testing an experimental gene therapy called FLT201 for adults with type 1 Gaucher disease. Researchers want to see if a single dose can safely help the body produce a vital missing enzyme, keeping blood counts, energy levels, and organ sizes stable for at least one year.
Spain - recruitingPHASE3
A Gaucher Disease Gene Therapy Trial With FLT201
This study is testing a new gene therapy called FLT201 for adults with Gaucher disease type 1. It aims to see if patients can stop their current treatment after receiving FLT201 while remaining well. This is the final stage of testing before it might become available.
United States · Argentina · Australia - active not recruitingPHASE1, PHASE2
Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)
This study is checking on the long-term health of people with Type 1 Gaucher disease who previously received a treatment called FLT201 in an earlier trial. We'll follow them for five years to see how they're doing.
United States · Brazil · Spain - 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.
United States · Germany · Japan - recruitingPHASE1, PHASE2
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
This study is testing a new medicine, LY3884961, for adults with Gaucher disease, type 1. It aims to find the right dose and see how safe and effective it is for symptoms affecting the body, but not the brain.
United States · Australia · Brazil