A Study of Belcesiran in Patients With A1ATD-Associated Liver Disease
Status:
Recruiting
Trial end date:
2023-12-01
Target enrollment:
Participant gender:
Summary
This is a multiple dose, randomized, placebo-controlled, double-blind study of belcesiran to
evaluate the safety, tolerability, PK, and PD in adult patients with PiZZ A1ATD-associated
liver disease.
The study will be conducted in 2 parallel cohorts. A total of up to 27 participants may be
enrolled in a single cohort. The 2 cohorts are differentiated only by the duration of the
treatment period, the number of doses administered, and the timing of the end-of-treatment
(EOT) liver biopsy. Participants in Cohort 1 will undergo EOT biopsy at 24 weeks, and
participants in Cohort 2 will undergo EOT biopsy at 48 weeks.