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.
Complete the form to learn more about the possibility of participating.
Sign up NowAlpha-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 is designed to bind to RNA in liver cells and help correct the message before it makes protein. The study will investigate whether the study drug enables the liver to produce a healthy form of the AAT protein.
If you agree to participate in the study and meet the screening criteria, 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 Australian Therapeutic Goods Agency [TGA], 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.
Thank you for your interest in the RestorAATion-2 study! Please answer the following questions to see if you may be eligible to participate in the study. In addition to these pre-screening requirements, other study requirements will apply.
If you agree, you may be contacted via email or text regarding your eligibility. If you meet these pre-screening requirements, a study team member will reach out for next steps.
Home Wound Care will collect any information that you provide to contact you and ask some further questions to assess whether you may be eligible to participate in this study. Without this information Home Wound Care will not be able to undertake these tasks. Submitting your contact details does not mean you have to join the study. The decision to participate in research is always yours to make.
If you do not want Home Wound Care to contact you about the study, please do not provide your information.
Home Wound Care will share your contact information with Clicksend for the purposes of sending SMS communications to you.
Two weeks after an SMS is sent to you, the body of the message will be replaced with unreadable symbols to ensure that your information is de-identified. Emails between you and Home Wound Care will be deleted four-months after the study has completed recruiting participants.
If Home Wound Care assesses that you may be eligible to participate in the study, they will share your information with Medpace (who is assisting Wave Life Sciences to conduct the study), the study doctor and staff at the relevant site. This is to find out more information and determine if the study is right for you. Only de-identified information about you will be shared with the sponsor, Wave Life Sciences. Your information may also be shared with regulators who are overseeing the study (e.g. the Therapeutic Goods Administration) and the relevant Human Research Ethics Committee. Home Wound Care is a UK based organisation, and your information may be stored in the UK [check]. Some of the other entities involved in the study may be based outside of Australia.
Additional details on how to access your information, seek deletion of your information, object to the processing of your information, or restrict processing of your information, as well as how to seek correction or make a complaint related to handling of your personal information, may be found via the Home Wound Care Data Protection Policy on website: www.homewoundcare.co.uk/privacy-policy.