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)
Facility
City
Region
Status
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.
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