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

NCT ID: NCT06405633

Last Updated: 2025-10-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-07-19

Study Completion Date

2026-09-30

Brief Summary

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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.

Detailed Description

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Conditions

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Alpha-1 Antitrypsin Deficiency

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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WVE-006 (Dose 1)

Group Type EXPERIMENTAL

WVE-006

Intervention Type DRUG

RNA editing oligonucleotide

WVE-006 (Dose 2)

Group Type EXPERIMENTAL

WVE-006

Intervention Type DRUG

RNA editing oligonucleotide

WVE-006 (Dose 3)

Group Type EXPERIMENTAL

WVE-006

Intervention Type DRUG

RNA editing oligonucleotide

Interventions

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WVE-006

RNA editing oligonucleotide

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Wave Life Sciences Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Cynthia Caracta, MD

Role: STUDY_DIRECTOR

Wave Life Sciences

Locations

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St. Vincent's Hospital, Melbourne

Fitzroy, Victoria, Australia

Site Status RECRUITING

Dalhousie University - Queen Elizabeth II Health Sciences Centre

Halifax, Nova Scotia, Canada

Site Status RECRUITING

Inspiration Research Limited

Toronto, Ontario, Canada

Site Status RECRUITING

Turku University Hospital

Turku, , Finland

Site Status RECRUITING

Universitaetsklinikum Aachen, AoeR

Aachen, , Germany

Site Status RECRUITING

Waikato Hospital

Hamilton, , New Zealand

Site Status RECRUITING

Queen Elizabeth Hospital, University Hospital Birmingham

Birmingham, , United Kingdom

Site Status RECRUITING

Cambridge University - Addenbrooke's Hospital

Cambridge, , United Kingdom

Site Status RECRUITING

Royal Free London NHS Foundation Trust

London, , United Kingdom

Site Status RECRUITING

Countries

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Australia Canada Finland Germany New Zealand United Kingdom

Central Contacts

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Clinical Operations

Role: CONTACT

855-215-4687

Facility Contacts

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Sue Brenton

Role: primary

Scott Fulton

Role: primary

Kayla Cardoso

Role: primary

Dhwani Patel

Role: backup

Terhi Yla-Outinen

Role: primary

Sarah Henke

Role: primary

Chris Tuffery

Role: primary

Emma Bruce

Role: primary

Abi Ford

Role: primary

Cristina Ma

Role: primary

Other Identifiers

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WVE-006-002

Identifier Type: -

Identifier Source: org_study_id

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