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A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD
Sponsor: Wave Life Sciences Ltd.
Summary
The purpose of this open-label study is to assess the safety, tolerability, pharmacodynamics, and pharmacokinetics of WVE-006 in participants with alpha-1 antitrypsin deficiency (AATD) following Period 1 single ascending dose (SAD) and Period 2 multiple ascending doses (MAD), respectively.
Official 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)
Key Details
Gender
All
Age Range
18 Years - 70 Years
Study Type
INTERVENTIONAL
Enrollment
24
Start Date
2024-07-19
Completion Date
2026-09
Last Updated
2025-10-07
Healthy Volunteers
No
Conditions
Interventions
WVE-006
RNA editing oligonucleotide
Locations (9)
St. Vincent's Hospital, Melbourne
Fitzroy, Victoria, Australia
Dalhousie University - Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia, Canada
Inspiration Research Limited
Toronto, Ontario, Canada
Turku University Hospital
Turku, Finland
Universitaetsklinikum Aachen, AoeR
Aachen, Germany
Waikato Hospital
Hamilton, New Zealand
Queen Elizabeth Hospital, University Hospital Birmingham
Birmingham, United Kingdom
Cambridge University - Addenbrooke's Hospital
Cambridge, United Kingdom
Royal Free London NHS Foundation Trust
London, United Kingdom