Clinical Trial

Disease: Alpha-1 Antitrypsin Deficiency, AATD, (NCT06186492)

Disease info:

Alpha-1 antitrypsin deficiency (AATD) is an inherited genetic disorder that is most commonly caused by a G-to-A point mutation in the SERPINA1 gene. This mutation results in the production of insufficient levels of circulating M-AAT protein, which protects the lungs from proteolytic enzymes, and aggregation of misfolded Z-AAT protein in hepatocytes. This leads to lung and liver disease. Individuals with AATD have Z mutations on both alleles, known as the PiZZ genotype.

Augmentation therapy via delivery of functional AAT protein is currently the only treatment option for AATD lung disease and requires weekly intravenous infusions. There are no treatments for AATD liver disease, other than liver transplantation.

There are approximately 200,000 patients in the United States and Europe who have Z mutations on both alleles.
Official title:
A Phase 1, Randomized, Double-blind, Placebo-controlled, Safety, Tolerability, and Pharmacokinetic Study of Single Ascending Doses and Multiple Doses of WVE-006 in Healthy Participants

Name: Clinical Operations

Phone: 855-215-4687



United Kingdom, Wales
Simbec-Orion Clinical Pharmacology, Merthyr Tydfil, Wales, United Kingdom, CF48 4DR

Study start:
Nov. 14, 2023
56 participants
Gene editing method:
A-to-I RNA editing oligonucleotides (AIMers)
Type of edit:
RNA editing
Delivery method:
Subcutaneous administration of GalNAc-conjugated RNA editing oligonucleotide - In-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


This study is the first study in the RestorAATion clinical program.

The purpose of this first-in human (FIH), double-blind, randomized, placebo-controlled, single ascending dose (SAD) and multiple-dose Phase 1 study is to assess the safety, tolerability, and PK of WVE-006 compared to placebo in healthy participants following a single dose (Period 1) and multiple doses (Period 2) of WVE-006.

This information will be used to determine doses and regimes that have the potential to be pharmacologically active in patients with Alpha-1 antitrypsin deficiency in the RestorAATion 2 study, and the maximum safe and tolerable dose that may be given to these patients.

Last updated: Apr. 20, 2024
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