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
RECRUITINGDalhousie University - Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia, Canada
RECRUITINGThe 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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Inspiration Research Limited
Toronto, Ontario, Canada
Turku University Hospital
Turku, Finland
RECRUITINGUniversitaetsklinikum Aachen, AoeR
Aachen, Germany
RECRUITINGWaikato Hospital
Hamilton, New Zealand
RECRUITINGQueen Elizabeth Hospital, University Hospital Birmingham
Birmingham, United Kingdom
RECRUITINGCambridge University - Addenbrooke's Hospital
Cambridge, United Kingdom
RECRUITINGRoyal Free London NHS Foundation Trust
London, United Kingdom
RECRUITING