Pediatric Cholestasis Clinical Trial
Official title:
An Exploratory Phase II Study to Demonstrate the Safety and Efficacy of A4250 in Children With Cholestatic Pruritus
This study will evaluate A4250 (IBATinhibitor) as a treatment option in pediatric patients with chronic cholestasis with main emphasis on safety evaluation and on effects on pruritus
The primary aims of this Phase II exploratory study in patients treated with A4250 due to cholestasis induced pruritus are to: - Assess the safety and tolerability of A4250, orally administered first as a single dose and then during a four week treatment period, as determined by the occurrence of treatment-emergent SAEs - Explore changes in serum total bile acids after a four week treatment period Secondary safety aims include assessment of the safety and tolerability of A4250, orally administered first as a single dose and then during a four week treatment period, as determined by the occurrence of treatment-emergent AEs and changes in safety parameters including laboratory tests and vital signs Secondary efficacy aims are to: - Demonstrate the efficacy of A4250, orally administered during a four week treatment period, on liver biochemistry variables and on pruritus parameters - Evaluate the pharmacokinetic properties of A4250 orally administered first as a single dose and then after a four week treatment period - Evaluate changes in VAS-itching score after a four week treatment period ;