Active not recruitingPHASE1, PHASE2INTERVENTIONAL

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

This research is looking into a new treatment called WVE-006 for a condition known as Alpha-1 Antitrypsin Deficiency (AATD). People with AATD have a genetic problem that can lead to lung and liver damage. This study is in its early stages (Phase 1b/2a), which means researchers are mainly checking if the medicine is safe and well-tolerated when given in single or multiple doses. They will also observe how the medicine affects the body and how it is processed. The goal is to gather important information to see if WVE-006 could be a helpful future treatment for individuals living with AATD.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Wave Life Sciences USA, Inc.

Enrolment target

Start

19 Jul 2024

Estimated completion

01 Sep 2026

Alpha-1 Antitrypsin Deficiency

What is this study about?

This study is investigating a new medication called WVE-006, which could potentially help people with a genetic condition called Alpha-1 Antitrypsin Deficiency (AATD). AATD happens when the body doesn't make enough of a special protein called alpha-1 antitrypsin. This protein usually protects the lungs from damage, and when it's missing or faulty, it can lead to lung problems like emphysema. It can also cause liver issues.

The main purpose of this study is to find out if WVE-006 is safe for people with AATD and how well their bodies handle it. Researchers will also look at how the medicine works in the body and how the body processes it. This is an early-stage study, meaning it's one of the first times this medicine is being tested in humans with the condition, so safety is the top priority.

By taking part, you would help doctors understand if WVE-006 could be a future treatment option. The information gathered from participants in this study is crucial for deciding if the medicine can move on to larger studies and potentially become available to more people with AATD in the future. It's a vital step in developing new ways to manage this condition.

Key takeaways

This study is testing a new medicine (WVE-006) for Alpha-1 Antitrypsin Deficiency (AATD).
Its main goal is to check how safe the medicine is and how the body handles it.
Participation involves receiving injections and attending clinic visits for monitoring.
You must be an adult between 18 and 70 years old with a specific type of AATD.
There are strict rules about who can join, including not smoking recently and limited alcohol intake.
You can always choose to leave the study at any point.

Who may be eligible?

To be considered for this study, you would need to be between 18 and 70 years old. You should have a confirmed diagnosis of Alpha-1 Antitrypsin Deficiency (AATD) linked to the specific 'Pi*ZZ' genetic type. You might be generally healthy, or have mild to moderate lung problems due to AATD (meaning your lung function test, FEV1, is 50% or better), or mild liver problems that are stable ( FibroScan score of F2 or less).

There are also some important reasons why you might not be able to join. For instance, if you've smoked in the last year, you wouldn't be eligible. You also can't have a history of serious allergies to medicines, or problems with injections under the skin. If you have any ongoing or recent infections, or if you've recently had certain vaccinations or plan to have them during the study, you wouldn't be able to participate.

Other reasons that might prevent participation include regularly drinking more than 14 alcoholic drinks per week (though you must agree to avoid alcohol for 48 hours before each dose visit). If you're currently receiving or have recently received other specific AATD treatments, or if you've been in another clinical trial recently, you might not be eligible. The study team will review your full medical history to make sure it's safe for you to take part.

Quick self-check

Am I between 18 and 70 years old?
Do I have a confirmed diagnosis of Pi*ZZ Alpha-1 Antitrypsin Deficiency?
Have I been a non-smoker for at least the last year?
Do I currently have any serious infections, or am I planning on having recent vaccinations?
Am I able to avoid more than 14 alcoholic drinks per week, and abstain 48 hours before visits?
Am I currently on, or have I recently used, other specific AATD treatments?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you would receive the new medicine, WVE-006, in either a single dose initially, or multiple doses over time. This medicine is given as an injection under the skin.

You would need to attend several visits at the study clinic. During these visits, the research team would perform various assessments. These might include blood tests to check how the medicine is working and how your body is handling it, as well as general health check-ups to monitor your safety. They might also do other tests related to your lung and liver health.

The study would involve regular contact with the research team to ensure your well-being and to collect all necessary information. The exact number of visits and the total length of your participation would be explained in detail by the study team, but typically, these studies involve a screening period, dosing period, and a follow-up period. You would have the right to withdraw from the study at any time.

Potential risks and benefits

Taking part in a study like this might offer some potential benefits, such as receiving a new investigational treatment for your Alpha-1 Antitrypsin Deficiency that is not yet widely available. You would also receive close medical attention and monitoring from the study team. However, it's important to remember that WVE-006 is an experimental medicine, and there's no guarantee it will improve your condition. There could be potential risks or side effects from taking the medicine or from the study procedures, such as discomfort from injections or reactions to the drug. All known risks would be fully explained to you before you decide to join. Remember, taking part is completely voluntary, and you are free to withdraw from the study at any time, for any reason, without affecting your usual medical care.

Locations (9)

St. Vincent's Hospital, Melbourne
Fitzroy, Australia
Dalhousie University - Queen Elizabeth II Health Sciences Centre
Halifax, Canada
Inspiration Research Limited
Toronto, Canada
Turku University Hospital
Turku, Finland
Universitaetsklinikum Aachen, AoeR
Aachen, Germany
Waikato Hospital
Hamilton, New Zealand
Queen Elizabeth Hospital, University Hospital Birmingham
Birmingham, United Kingdom
Cambridge University - Addenbrooke's Hospital
Cambridge, United Kingdom
Royal Free London NHS Foundation Trust
London, United Kingdom

Common questions

What is Alpha-1 Antitrypsin Deficiency (AATD)?

It's a genetic condition where your body doesn't make enough of a special protein that protects your lungs and liver from damage.

What is WVE-006?

WVE-006 is a new medicine being tested in this study for people with Alpha-1 Antitrypsin Deficiency. It's an investigational treatment.

What does 'Phase 1b/2a' mean for a study?

It means this is an early stage study, mainly focused on checking the safety of the new medicine and finding the right dose, rather than seeing if it cures the condition.

What are the main things I need to do if I join?

You would receive the study medicine by injection and attend several clinic visits for health checks, blood tests, and to monitor how you're doing.

Can I leave the study early if I want to?

Yes, you have the right to leave the study at any time, for any reason, without it affecting your medical care.