Recruiting
Phase 1
Phase 2

AATD

Sponsor:

Wave Life Sciences USA, Inc.

Code:

NCT06405633

Conditions

Alpha-1 Antitrypsin Deficiency

Eligibility Criteria

Sex: All

Age: 18 - 70

Healthy Volunteers: Not accepted

Interventions

WVE-006

Study Details

Brief 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.

Conditions

Alpha-1 Antitrypsin Deficiency

Study ID

NCT06405633

Start date

Jul 19, 2024

Status verified date

Oct, 2026

Completion date

Sep, 2027

Anticipated

Primary completion date

Sep, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

48 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: WVE-006 (Dose 1)

experimental: WVE-006 (Dose 2)

experimental: WVE-006 (Dose 3)

experimental: WVE-006 (Dose 4)

experimental: WVE-006 (Dose 5)

Interventions

WVE-006

RNA editing oligonucleotide

Primary outcome measure

  • The proportion of participants with adverse events [ Time Frame: Up to 36 Weeks ]

Central Contacts and Locations

Central contacts

Clinical Operations

855-215-4687Clinicaltrials@wavelifesci.com

Locations

Inspiration Research Limited

Recruiting

Toronto, Ontario, Canada, M5T 3A9

Contacts

Kayla Cardoso

kcardoso@inspirationresearch.ca

Dhwani Patel

dpatel@inspirationresearch.ca

More Information

Sponsor

Wave Life Sciences USA, Inc.

Last update posted

Oct 19, 2026

Last verified

Oct, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-20. This information was provided to ClinicalTrials.gov by Wave Life Sciences USA, Inc. on 2026-10-19. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.