3 results
Approved WMOWill not start
The purpose of this study is to evaluate the possible risks and efficacy (improvement of disease) with an experimental oral study drug named eliglustat in pediatric patients from 2 to 18 years with Gaucher disease.
Approved WMOCompleted
Primary: To demonstrate that high (100%) stimulation of ONS therapy reduces the mean attack frequency (MAF) in MICCH by at least 35% compared to low (30%) stimulation of ONS.Secondary: To evaluate • the rate of responders (>= 50% reduction in…
Approved WMORecruiting
The objectives of this are to establish the safety, tolerability, and efficacy of VRDN-001, and the pharmacokinetic (PK) and pharmacodynamic (PD) profiles of VRDN-001 in HV and TED patients over a dose range of 3.0 to 20.0 mg/kg.