A Study of Belcesiran in Patients With AATLD

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 AATD-associated liver disease (AATLD). The study will be conducted in 3 separate cohorts. A total of up to 16 participants may be enrolled in Cohort 1 and 2. A total number of 30 subjects will be enrolled in cohort 3. The 3 cohorts are differentiated by the duration of the treatment period, the number of doses administered, and the timing of the second liver biopsy.

Timeline

Start

2021-02-12

Primary completion

2023-12-08

Completion

2024-05-29

Outcome

Outcome not reported

Stopped: “Development project was discontinued”

Drugs

Indications