RestorAATion-2
Research type
Research Study
Full title
A Phase 1b/2a Open-label Single Ascending Doses (SAD) and Multiple Ascending Doses (MAD) Research Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Participants with AATD Pi*ZZ on WVE-006 (RestorAATion-2)
IRAS ID
1009561
Contact name
Mara Kochaba
Contact email
Sponsor organisation
Wave Life Sciences UK Limited
Research summary
Alpha-1 antitrypsin deficiency (AATD) is an inherited condition that may affect the liver and lungs and may cause them to not function properly over time. AATD results from several possible genetic mutations to the SERPINIA1 gene, leading to a reduction in the amount of properly functioning Alpa-1 antitrypsin (AAT) protein in the blood. This protein is important to the proper function of the liver and lungs.
Study WVE-006-002 (RestorAATion-2) is a phase 1b/2a, multicentre, open-label study to investigate the safety, tolerability (degree to which side effects of a drug can be tolerated) and effect the study drug, WVE-006, has on participants aged ≥18 and ≤70 years who present with AATD. WVE-006 is an investigational medicine that was designed to specifically edit variant SERPINA1 ribonucleic acid (RNA) back to its normal state.
The study will be split into two periods. Patients will be given a single dose of the study drug in Period 1 and will be followed up for approximately 12 weeks. The patients will then move into Period 2 where they will receive multiple doses of the study drug. Period 2 will last approximately 24 weeks .
REC name
North of Scotland Research Ethics Committee 1
REC reference
24/NS/0048
Date of REC Opinion
15 May 2024
REC opinion
Further Information Favourable Opinion