Alpha-1 Antitrypsin DeficiencyPhase 1/2NCT06405633
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD
A Phase 1/2 study testing WVE-006 in people with Alpha-1 Antitrypsin Deficiency, run by Wave Life Sciences USA, Inc. at 9 sites in 6 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Alpha-1 Antitrypsin Deficiency
- What is being tested?
- WVE-006
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 9 sitesin 6 countries
- Study dates
- 19 Jul 2024to Sep 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- • 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.
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- 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.
Trial locations and contacts
9 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
9 sites in 6 countries
United Kingdom3 sites
- Active, not recruiting
Queen Elizabeth Hospital, University Hospital Birmingham
Birmingham, B15 2GW
- Active, not recruiting
Cambridge University - Addenbrooke's Hospital
Cambridge, CB2 0QQ
- Active, not recruiting
Royal Free London NHS Foundation Trust
London, SW8 3RN
Canada2 sites, 1 recruiting
- Active, not recruiting
Dalhousie University - Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia, B3H 1V7
- Recruiting
Inspiration Research Limited
Toronto, Ontario, M5T 3A9
Kayla Cardoso, kcardoso@inspirationresearch.ca
Dhwani Patel, dpatel@inspirationresearch.ca
Australia1 site
- Active, not recruiting
St. Vincent's Hospital, Melbourne
Fitzroy, Victoria, 3065
Germany1 site
- Active, not recruiting
Universitaetsklinikum Aachen, AoeR
Aachen, 52074
About this study
Official title: A Phase 1b/2a Open-label Single Ascending Doses (SAD) and Multiple Ascending Doses (MAD) Research Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Participants With AATD Pi*ZZ on WVE-006 (RestorAATion-2)
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
- Being tested
- WVE-006
- Medical subject headings
- alpha 1-Antitrypsin Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
WVE-006Drug
RNA editing oligonucleotide
| Study group | Role | What they receive |
|---|---|---|
| WVE-006 (Dose 1) | Experimental | Not described Receives: Drug: WVE-006 |
| WVE-006 (Dose 2) | Experimental | Not described Receives: Drug: WVE-006 |
| WVE-006 (Dose 3) | Experimental | Not described Receives: Drug: WVE-006 |
| WVE-006 (Dose 4) | Experimental | Not described Receives: Drug: WVE-006 |
| WVE-006 (Dose 5) | Experimental | Not described Receives: Drug: WVE-006 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of participants with adverse events
Measured: Up to 36 Weeks
Secondary outcomes (6)
Single Ascending Dose - Change from baseline in levels of serum M-AAT protein.
Measured: Up to 12 Weeks
Multiple Ascending Dose - Change from baseline in levels of serum M-AAT protein
Measured: 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)
Measured: Up to 12 Weeks
Single Ascending Dose - Maximum concentration of WVE-006 in plasma
Measured: 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)
Measured: Up to 24 Weeks
Multiple Ascending Doses - Maximum concentration of WVE-006 in plasma (Cmax)
Measured: Up to 24 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2024
- Study start19 Jul 2024
- Primary completionSep 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha-1 Antitrypsin DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Wave Life Sciences USA, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, Wave Life Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06405633
- Sponsor's protocol ID
- WVE-006-002
- Status last verified
- Oct 2026
- First submitted
- 3 May 2024
- Last updated
- 19 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b/2a Open-label Single Ascending Doses (SAD) and Multiple Ascending Doses (MAD) Research Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Participants With AATD Pi*ZZ on WVE-006 (RestorAATion-2)
- ClinicalTrials.gov ID
- NCT06405633
- Acronym / sponsor ID
- WVE-006-002
- Conditions
- Alpha-1 Antitrypsin Deficiency
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Wave Life Sciences USA, Inc.Industry (pharma, biotech, devices)
- Study start
- 19 Jul 2024 (actual)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 8 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| WVE-006 (Dose 1) | Experimental | Drug: WVE-006 |
| WVE-006 (Dose 2) | Experimental | Drug: WVE-006 |
| WVE-006 (Dose 3) | Experimental | Drug: WVE-006 |
| WVE-006 (Dose 4) | Experimental | Drug: WVE-006 |
| WVE-006 (Dose 5) | Experimental | Drug: WVE-006 |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (11 listed)
- • Participant has a history of multiple drug allergies or of allergic reaction to an oligonucleotide or to N-acetylgalactosamine (GalNAc).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| WVE-006RNA editing oligonucleotide | Drug | WVE-006 (Dose 1), WVE-006 (Dose 2), WVE-006 (Dose 3), WVE-006 (Dose 4), WVE-006 (Dose 5) |
Primary outcome
The proportion of participants with adverse events
Time frame: Up to 36 Weeks
6 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director, MD study director
Wave Life Sciences
9 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing WVE-006 in people with Alpha-1 Antitrypsin Deficiency, run by Wave Life Sciences USA, Inc. at 9 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United Kingdom, Canada, Australia, Finland, Germany and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Operations (855-215-4687, Clinicaltrials@wavelifesci.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The proportion of participants with adverse events (time frame: Up to 36 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2026.