NCT03767829

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

Terminated Phase 1, PHASE2 Last updated 27 April 2021

What this trial tests

Phase 1, PHASE2 trial testing ALN-AAT02 in ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease in 32 participants. Terminated before completion.

Timeline

5 December 2018

Primary endpoint
25 June 2020

25 June 2020

Quick facts

Lead sponsor	Alnylam Pharmaceuticals
Phase	Phase 1, PHASE2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	treatment
Enrollment	32
Start date	5 December 2018
Primary completion	25 June 2020
Estimated completion	25 June 2020
Sites	1 location across United Kingdom

Drugs / interventions tested

• ALN-AAT02 — full drug profile →
• Placebo
• ALN-AAT02 — full drug profile →

Conditions studied

• ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease — all drugs for ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease →

Sponsor

Alnylam Pharmaceuticals — full company profile →

Who can join

Adults 18 to 65, any sex, with ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Therapeutic siRNA: state of the art.
   Hu B, Zhong L, Weng Y, Peng L, et al · · 2020 · cited 991× · PMID 32561705 · DOI 10.1038/s41392-020-0207-x
2. Paving the Road for RNA Therapeutics.
   Dammes N, Peer D. · · 2020 · cited 159× · PMID 32893005 · DOI 10.1016/j.tips.2020.08.004
3. The landscape of nanoparticle-based siRNA delivery and therapeutic development.
   Moazzam M, Zhang M, Hussain A, Yu X, et al · · 2024 · cited 116× · PMID 38204162 · DOI 10.1016/j.ymthe.2024.01.005
4. Improved Nucleic Acid Therapy with Advanced Nanoscale Biotechnology.
   Weng Y, Huang Q, Li C, Yang Y, et al · · 2020 · cited 72× · PMID 31927331 · DOI 10.1016/j.omtn.2019.12.004
5. RNA delivery biomaterials for the treatment of genetic and rare diseases.
   Zhao W, Hou X, Vick OG, Dong Y. · · 2019 · cited 65× · PMID 31255978 · DOI 10.1016/j.biomaterials.2019.119291
6. A Review of Alpha-1 Antitrypsin Binding Partners for Immune Regulation and Potential Therapeutic Application.
   O'Brien ME, Murray G, Gogoi D, Yusuf A, et al · · 2022 · cited 57× · PMID 35269582 · DOI 10.3390/ijms23052441
7. Alpha-1 antitrypsin deficiency: an update on clinical aspects of diagnosis and management.
   Santos G, Turner AM. · · 2020 · cited 17× · PMID 33659933 · DOI 10.12703/b/9-1