3 results
Approved WMOCompleted
To investigate the effect of cholic acid supplementation on the clinical and biochemical parameters of Zellweger spectrum disorder
Approved WMOCompleted
Primary objectives:Safety for Dose Selection• To assess the safety and tolerability of PQ912Efficacy • To evaluate the efficacy of PQ912 on working memory and attentionSecondary Objectives:Safety• To assess the safety and tolerability of long-term…
Approved WMORecruiting
Investigating the long-term safety of personalized cholic acid treatment in patients with defects in bile acid synthesis based on clinical and biochemical parameters.