WVE-006 for Alpha-1 antitrypsin deficiency (AATD) | Alpha-1 antitrypsin deficiency (Pi*ZZ)

Inclusion criteria

• {"criterion_text":"- 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."}
• {"criterion_text":"- Genetic testing confirming Pi*ZZ."}
• {"criterion_text":"- Participant has been a non-smoker for at least 1 year prior to screening."}

Exclusion criteria

• {"criterion_text":"- Participant has a history of multiple drug allergies or of allergic reaction to an oligonucleotide or to N-acetylgalactosamine (GalNAc)."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- Participant has received an investigational agent within 3 months of the Screening Visit."}
• {"criterion_text":"- Participant has a history of intolerance or any medical condition that might interfere with subcutaneous injections."}
• {"criterion_text":"- Any ongoing or recent infections."}
• {"criterion_text":"- Any recent or planned vaccinations during the study."}
• {"criterion_text":"- Participant has a history of regular alcohol consumption exceeding 14 standard drinks/week."}
• {"criterion_text":"- Unwilling to abstain from alcohol for 48 hours prior to dosing at each of the dosing visits."}
• {"criterion_text":"- Any recent or planned major surgery during the study."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 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."}

Primary endpoints

• {"endpoint_text":"- The proportion of participants with adverse events.","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- Single Ascending Dose - Change from baseline in levels of serum M-AAT protein.","definition_or_measurement_approach":"Change from baseline in serum M-AAT protein levels following single ascending doses."}
• {"endpoint_text":"- Multiple Ascending Dose - Change from baseline in levels of serum M-AAT protein","definition_or_measurement_approach":"Change from baseline in serum M-AAT protein levels following multiple ascending doses."}
• {"endpoint_text":"- Single Ascending Dose - Area under the plasma concentration time curve for WVE-006 from time of dosing to the last measurable concentration (AUClast)","definition_or_measurement_approach":"AUClast of WVE-006 following single ascending dose."}
• {"endpoint_text":"- Single Ascending Dose - Maximum concentration of WVE-006 in plasma","definition_or_measurement_approach":"Cmax of WVE-006 following single ascending dose."}
• {"endpoint_text":"- Multiple Ascending Doses - Area under the plasma concentration time curve for WVE-006 from time of dosing to the last measurable concentration (AUClast)","definition_or_measurement_approach":"AUClast of WVE-006 following multiple ascending doses."}
• {"endpoint_text":"- Multiple Ascending Doses - Maximum concentration of WVE-006 in plasma (Cmax)","definition_or_measurement_approach":"Cmax of WVE-006 following multiple ascending doses."}

Methods

• Finland: Website Package (K2_Recruitment material_Website Package_FI) — web-based recruitment materials and online pre-screener package to identify eligible participants (associated documents listed for Finland).
• Finland: Pre-Screener Package (K2_Recruitment material_Pre-Screener Package) — pre-screening materials to evaluate potential participant eligibility prior to site contact.
• Finland: Brochure / Flyer / Participant Journey (K2_Recruitment material_Brochure / K2_Recruitment material_Participant Journey / K2_Recruitment material_ATD Flyer) — printed/digital flyers and brochures to inform and recruit people with AATD in Finland.
• Germany: AATD Flyer / Brochure / Participant Journey / K1_Recruitment arrangements_Germany — recruitment flyers/brochures and participant journey materials for Germany.
• Privacy Policy: Privacy Policy document in recruitment materials for Finland indicating handling of personal data during recruitment.

Finland

Earliest CTIS Part Ii Submission Date

08-11-2024

Latest Decision Or Authorization Date

12-02-2026

Processing Time Days

461

Number Of Sites

1

Number Of Participants

2

Germany

Earliest CTIS Part Ii Submission Date

31-01-2025

Latest Decision Or Authorization Date

29-08-2025

Processing Time Days

210

Number Of Sites

1

Number Of Participants

5

Primary sponsor

Full Name

Wave Life Sciences Ireland Limited

Organisation Type

Pharmaceutical company

Country Of Registered Address

Ireland

Contract research organisations

Name

PPD Development LP

Responsibilities

4

Name

Pharmaceutical Product Development LLC

Responsibilities

4

Name

Medpace Finland Oy

Responsibilities

1; 12; 13; 2; 4; 5; 6

Third parties

• {"country":"United States","full_name":"Eclinical Solutions LLC","duties_or_roles":"6","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"EPL Pathology Archives LLC","duties_or_roles":"storage of biological material","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Packaging Coordinators LLC","duties_or_roles":"14","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Charles River Laboratories Edinburgh Limited","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Charles River Laboratories Inc.","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Quest Diagnostics Inc.","duties_or_roles":"4","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Cenetron Diagnostics Ltd.","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"1; 12; 13; 2; 4; 5; 6","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Transperfect Translations International Inc.","duties_or_roles":"label translations","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Yprime LLC","duties_or_roles":"3","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Denmark","full_name":"Nordic Bioscience A/S","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"GSK PLC","duties_or_roles":"Proteomics","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Illingworth Research Group Limited","duties_or_roles":"home nursing visits","organisation_type":"Hospital/Clinic/Other health care facility"}