NCT03767829

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

A Phase 1/2, Randomized, Double-blind, Placebo-controlled, Single-ascending and Multiple-dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT02 in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 32 (actual)
• Sponsor: Alnylam Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Accepted

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.

Conditions

• ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease

Interventions

Type	Name	Description
DRUG	ALN-AAT02	ALN-AAT02 will be administered subcutaneously (SC) at dose levels planned for Part A.
DRUG	Placebo	Sterile normal saline (0.9% NaCl) matching volume of ALN-AAT02 doses will be administered SC.
DRUG	ALN-AAT02	ALN-AAT02 will be administered subcutaneously (SC). Part B dose levels to be determined upon review of data from Part A.

Timeline

• Start date: 2018-12-05
• Primary completion: 2020-06-25
• Completion: 2020-06-25
• First posted: 2018-12-07
• Last updated: 2021-04-27

Locations

1 site across 1 country: United Kingdom