Syneos Health (Beijing) Inc.Ltd.

Primary Objective: To demonstrate the sustained effect of odevixibat on native liver survival in children with biliary atresia (BA) who have completed Study A4250-011 (BOLD)

The primary objectives of the study are to evaluate the safety and tolerability of IDX-1197 and to determine the maximum tolerated dose (MTD) and recommended phase II dose (RP2D) of IDX-1197 in combination with capecitabine and oxaliplatin (Xelox) or irinotecan for the treatment of patients with advanced gastric cancer. Secondary objectives of the study are to evaluate the efficacy of IDX-1197 in combination with Xelox or irinotecan, as defined by objective response rate (ORR), progression-free survival (PFS), disease control rate (DCR), duration of response (DOR), time to response (TTR), best overall response (BOR), time to disease progression (TTP), lowest repeatable effective dose (MRAD), and overall survival (OS) as assessed by response evaluation criteria in solid tumors (RECIST) v1.1. To evaluate the pharmacokinetics (PK) of IDX-1197 in combination with Xelox or irinotecan as a single ascending dose (SAD) in patients with advanced gastric cancer (dose-finding portion only). Population PK models of IDX-1197 and irinotecan were evaluated in combination with Xelox or irinotecan in patients with advanced gastric cancer. The effects of IDX-1197 plus irinotecan on cardiac repolarization were evaluated by assessing the corrected QT interval. The risk of QT prolongation was assessed by ECG recordings and time-matched PK measurements.

The primary objective was to evaluate the effectiveness of maralixibat for biliary drainage after hepatoportoenterostomy (HPE) in patients with biliary atresia (BA). Secondary objectives were to evaluate changes in sBA (serum bile acid) levels after maralixibat treatment, to evaluate the rate of bilirubin normalization after maralixibat treatment, to evaluate transplant-free survival after maralixibat treatment, to evaluate biochemical markers of cholestasis and liver disease after maralixibat treatment, and to evaluate the safety, tolerability, and pharmacokinetics of maralixibat.