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Active, not recruiting Phase 1/2

A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD

NCT06405633 · tracked via the Priya Life Science Australia tracker
Phase
Phase 1/2
Started
2024-07-19
Last updated
2026-04-14

Condition(s) studied

Alpha-1 Antitrypsin Deficiency

Investigational drug(s) / intervention(s)

WVE-006

WVE-006: RNA editing oligonucleotide

Study 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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
70 Years
Healthy volunteers
No
Inclusion Criteria: * Healthy as determined by the Investigator, based on a medical evaluation, or has mild to moderate AATD-induced lung disease (FEV1 ≥50%) and/or stable mild AATD-induced liver disease (≤F2 (≤10 kPa) on FibroScan. * Genetic testing confirming Pi\*ZZ. * Participant has been a non-smoker for at least 1 year prior to screening. Exclusion Criteria: * • Participant has a history of multiple drug allergies or of allergic reaction to an oligonucleotide or to N-acetylgalactosamine (GalNAc). * Participant has a history of intolerance or any medical condition that might interfere with subcutaneous injections. * Any ongoing or recent infections. * Any recent or planned vaccinations during the study. * Participant has a history of regular alcohol consumption exceeding 14 standard drinks/week. * Unwilling to abstain from alcohol for 48 hours prior to dosing at each of the dosing visits. * Any recent or planned major surgery during the study. * Participant has any medical condition or social circumstance that, in the opinion of the Investigator, would make the participant unsuitable for participation in the study or for dosing on Day 1, or could interfere with the assessments of safety, pharmacodynamics, or pharmacokinetics, or completion of the study. * Participant currently on AAT augmentation therapy, planned to be on augmentation therapy anytime during the study, or has been on augmentation therapy within 30 days prior to Screening Visit. * Donation of blood or blood products in excess of 500 mL within 12 weeks prior to Screening Visit and/or unwilling to refrain from blood donation for the duration of the study. * Participant has received an investigational agent within 3 months of the Screening Visit.

Primary outcome measure(s)

  • The proportion of participants with adverse events — Up to 36 Weeks

Trial sites (9)

FacilityCityRegionStatus
St. Vincent's Hospital, Melbourne Fitzroy Victoria
Dalhousie University - Queen Elizabeth II Health Sciences Centre Halifax Nova Scotia
Inspiration Research Limited Toronto Ontario
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
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06405633 on ClinicalTrials.gov ↗ ← All trials in Australia