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RECRUITING
NCT06405633
PHASE1/PHASE2

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.

View on ClinicalTrials.gov

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

Interventions

DRUG

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