4 results
To investigate the effect of cholic acid supplementation on the clinical and biochemical parameters of Zellweger spectrum disorder
The primary objectives are:* to determine the effect of steady-state concentrations of TMC114 coadministered with a lowdose of ritonavir on the steady-state pharmacokinetics of ddI,* to determine the effect of steady-state concentrations of ddI on…
Dose escalation phase:Primary• Determine the RP2D and if reached, the MTD of GEN3014• Evaluate the safety and tolerability of GEN3014 Secondary:• Characterize the PK properties of GEN3014• Characterize the pharmacodynamic properties of GEN3014•…
Investigating the long-term safety of personalized cholic acid treatment in patients with defects in bile acid synthesis based on clinical and biochemical parameters.