Abstract
Recombinant human serum albumin (rHA) is a promising alternative to human serum albumin (HSA) for managing ascites in cirrhotic patients. This phase Ib study aims to assess the safety, tolerability, and pharmacokinetics/pharmacodynamics (PK/PD) profiles of rHA in this population.
This randomized, open-label, phase Ib trial was conducted between December 2019 and September 2020 at 3 medical centers in China. Patients with cirrhotic ascites were randomly assigned to receive rHA or HSA at 10 g/day, 20 g/day, or 30 g/day. Each group had 12 participants (nine receiving rHA and three receiving HSA as positive control). Treatment lasted up to 14 days or until serum albumin levels reached 35 g/L, followed by a 28-day follow-up. Adverse events monitored assessed safety and tolerability, while PK/PD was evaluated by tracking serum albumin levels and plasma colloid osmotic pressure (PCOP) before and after each dose (ClinicalTrials.gov No. NCT04701697).
Thirty-six Chinese participants were enrolled, with 32 completing the study. The incidence of adverse events was similar between the rHA and HSA groups (44.4% vs. 44.4%, p > 0.05). Serum albumin concentration increases were comparable between groups during treatment and follow-up. While most participants experienced weight and abdominal circumference decreases, no significant dose effect was observed (p > 0.05). No anti-drug antibodies were detected.
In this study, rHA demonstrated similar safety and PK/PD to HSA in cirrhotic patients with ascites. rHA was well-tolerated, supporting the need to evaluate its safety and efficacy in a phase II clinical study.