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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
24
RNA editing oligonucleotide
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
The proportion of participants with adverse events
Time frame: Up to 36 Weeks
Single Ascending Dose - Change from baseline in levels of serum M-AAT protein.
Time frame: Up to 12 Weeks
Multiple Ascending Dose - Change from baseline in levels of serum M-AAT protein
Time frame: Up to 24 Weeks
Single Ascending Dose - Area under the plasma concentration time curve for WVE-006 from time of dosing to the last measurable concentration (AUClast)
Time frame: Up to 12 Weeks
Single Ascending Dose - Maximum concentration of WVE-006 in plasma
Time frame: Up to 12 Weeks
Multiple Ascending Doses - Area under the plasma concentration time curve for WVE-006 from time of dosing to the last measurable concentration (AUClast)
Time frame: Up to 24 Weeks
Multiple Ascending Doses - Maximum concentration of WVE-006 in plasma (Cmax)
Time frame: Up to 24 Weeks
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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