venglustat
venglustat: 6 clinical trials tracked.
venglustat evidence translation
What we can confirm
6 tracked trials
What it means
What to watch
Prepared research question
What is verified about venglustat, and what remains unknown?
Traceable citations · Unknowns marked
See linked entities and source evidence2 indications · 1 study sponsors
Related indications
Study sponsors
Clinical Trials (6)
A Phase 1, Multi-center, Parallel, Open-label, Pharmacokinetic, Safety, and Tolerability Study of Venglustat Given as a Single Dose in Adult Participants With Mild, Moderate, and Severe Hepatic Impairment and in Matched Participants With Normal Hepatic Function
Primary endpoint: Maximum venglustat plasma concentration observed (Cmax)
A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy
Primary endpoint: Slope of left ventricular mass index as measured by cardiac magnetic resonance imaging (MRI) (central reading)
A Phase 3, Multicenter, Multinational, Randomized, Double-blind, Double-dummy, Active-comparator Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (GD3) Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ERT)
Primary endpoint: Change in Scale for Assessment and Rating of Ataxia (SARA) modified total score
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 Participants ≥16 Years of Age With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months
Primary endpoint: Percent change from baseline at 6 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome (FD-PRO) items (neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain)
An Open-label Study of Excretion Balance and Pharmacokinetics Following a Single Oral Dose of [14C]-Venglustat (2.67 MBq) in Healthy Male Subjects
Primary endpoint: Percentage of radioactive dose excreted in urine and faeces
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
Primary endpoint: Number of participants with Treatment Emergent Adverse Events (TEAEs)