The RestorAATion-2 clinical research study is for people who have an AAT protein deficiency caused by Pi*Z mutations. While alpha-1 antitrypsin deficiency (AATD, also known as alpha-1) mainly affects lung function, patients with the Pi*ZZ genotype may also experience liver disease. The study will evaluate how well the investigational study drug works as a potential treatment for protecting the lungs and liver in patients diagnosed with Pi*ZZ Alpha-1.
To join the study, a participant must meet the study enrollment criteria, including certain pre-screening requirements.
If you have or are at risk of liver and/or lung disease, you may be eligible to be screened further to join the study if you meet the following pre-screening requirements:
18-70 years old
Weigh at least 50kg
Confirmed diagnosis of AATD Pi*ZZ genotype
Have been a non-smoker for at least 1 year and agree to abstain from tobacco and nicotine containing products (including vaping) for the duration of the study
Other study requirements will apply for enrollment.
If you are unsure whether you meet the pre-screening requirements, please talk to your doctor.
Study participants can expect the following:
*Some participants may skip Period 1. Ask the study doctor if you do not remember whether you are participating in Period 1 or not.
Participation in a clinical study is voluntary. You can ask any questions you have and may leave the study at any time, for any reason.
Take the pre-screener to see if you may be eligible.
Take the Pre-ScreenerAlpha-1 is an inherited disease (you are born with it) that may lead to symptoms such as shortness of breath, frequent lung infections, and fatigue. Symptoms tend to worsen over time, although life expectancy with Alpha-1 tends to be normal.
Your Alpha-1 is caused by an abnormality in your DNA. This makes your liver produce a non-working version of a protein important for lung health. This abnormal protein becomes trapped in the liver and does not reach the lungs, which can gradually lead to liver and lung damage. There is no cure for Alpha-1, although therapies are available in some countries. Available therapies often require weekly infusions to slow lung damage caused by Alpha-1.
The investigational study drug, WVE-006, is designed to bind to your RNA in your liver cells and help correct the message before it makes protein. This could help your liver cells start producing the normal form of the AAT protein, M-AAT, replacing the faulty Z-AAT protein that leads to AATD. Ultimately, WVE-006 could protect both the lungs and liver from damage caused by Alpha-1.
Yes. During the RestorAATion-2 study, you will receive at least one dose (injected via needle under the skin) of the study drug. Your dose level may change during the study, which the study doctor will explain to you.
“Investigational” means that the medicine has not been approved by any Regulatory Agency (such as the US Food and Drug Administration [FDA] or the European Medicines Agency [EMA]) anywhere for use (treatment) as a prescription or over-the-counter medicine, and it can only be used in clinical research studies like RestorAATion-2.
Here are some common questions and answers about study participation.
Clinical research studies, or trials, help scientists and doctors explore whether a medical strategy, device, or medication is safe and effective for people. Before any medication can be approved and made available to the public, it must go through several phases of clinical research.
Before enrolling in a clinical trial, you must review, understand and sign an Informed Consent Form (ICF). The ICF contains information about the study, including study goals, how long the study will last, advantages and disadvantages of participating in the study, risks, and the tests and procedures you will receive. This information is meant to allow you to make an informed decision on whether it is right for you to participate in the study.
Study participation usually involves visiting a clinic regularly, taking or receiving an investigational medicine, and having assessments to monitor your health. You can still see your regular doctor, but you should let them know that you are participating in a study.
Participation in clinical research studies is your choice, and you may stop at any time for any reason.
Use the filters, map, and listing to find the study clinic closest to you.