Overview

A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease

Status:
Terminated
Trial end date:
2020-06-25
Target enrollment:
Participant gender:
Summary
The purpose of this study is to evaluate the safety and tolerability of single or multiple doses of ALN-AAT02. The study will be conducted in 2 sequential phases in which Part A will be a single-ascending dose (SAD) phase in healthy participants, and Part B will be a multiple-ascending dose (MAD) phase in participants with ZZ type alpha-1 antitrypsin deficiency (PiZZ) and biopsy-proven alpha-1 antitrypsin (AAT) deficiency-associated liver disease.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Alnylam Pharmaceuticals
Treatments:
Alpha 1-Antitrypsin
Protein C Inhibitor