RecruitingPHASE1INTERVENTIONAL

A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)

This research study is looking into a new experimental medicine, AIR-001, for adults who have a specific genetic lung condition called Alpha-1 Antitrypsin Deficiency (AATD). People with AATD don't produce enough of a protein that protects the lungs from damage. This first stage of the study, called Phase 1, aims to find out how safe the medicine is when given in different amounts. Researchers will also check how the body absorbs, uses, and gets rid of AIR-001. The study is open to both men and women between 18 and 74 years old who meet certain health criteria. It's an important step to see if AIR-001 could be a helpful treatment in the future.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

AIRNA Corporation

Enrolment target

Start

17 Mar 2026

Estimated completion

01 Jan 2029

Alpha 1 Antitrypsin Deficiency

What is this study about?

This study is all about a new medicine, AIR-001, for a condition called Alpha-1 Antitrypsin Deficiency, or AATD. People with AATD have a genetic problem that means their body doesn't make enough alpha-1 antitrypsin, which is a protein that protects the lungs. Without enough of this protection, the lungs can get damaged more easily, leading to breathing problems.

Because AIR-001 is a new medicine, researchers need to carefully check if it's safe for people to use. This kind of study, called a Phase 1 trial, is specifically designed to understand how the medicine behaves in the human body. They will be looking at different doses – from small amounts given once (single dose) to repeated doses over time (multiple doses) – to see what kind of effects it has.

The main goal is to check for any side effects and to understand how the body handles AIR-001, including how much of it gets into the blood and how long it stays there. The information gathered from this study will help doctors decide if AIR-001 is promising enough to continue testing in larger studies, with the hope that it could eventually become a new treatment option for people with AATD.

Key takeaways

This study is testing a brand-new medicine, AIR-001, for Alpha-1 Antitrypsin Deficiency (AATD).
It's a very early-stage trial (Phase 1) focused on checking the safety of the medicine.
Researchers will also see how the body handles different doses of AIR-001.
You need to be an adult with AATD and meet specific health requirements to join.
Participation involves clinic visits, taking the study medicine, and health checks.
Your involvement helps advance research for AATD, but a direct personal benefit isn't guaranteed.

Who may be eligible?

To be part of this study, you need to be an adult between 18 and 74 years old. You must have been diagnosed with Alpha-1 Antitrypsin Deficiency (AATD) because of a specific gene pattern (PiZZ genotype), and your alpha-1 antitrypsin protein levels must be low. Your lung function, measured by a breathing test, should also be at a certain level, and you shouldn't have smoked (including vaping) for at least six months.

There are also some health checks you'd need to pass, like your weight being within a certain range and having no acute chest infections recently. Women who could become pregnant would need to use effective contraception throughout the study and have a negative pregnancy test at the start.

However, you can't join if you are breastfeeding or have recently received a common AATD treatment called augmentation therapy. If you're allergic to AIR-001, have a complicated lung disease requiring continuous oxygen, or have certain liver issues, you also wouldn't be able to take part. Recent COVID-19 or flu infections would also mean you can't join at that time.

Quick self-check

Are you between 18 and 74 years old?
Do you have Alpha-1 Antitrypsin Deficiency (AATD) with the PiZZ gene type?
Have you stopped smoking or vaping for at least 6 months?
Do you not currently use, or plan to use, other AATD treatments alongside this study?
Are you able to attend study visits and follow all instructions?

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

What does participation involve?

If you join this study, you will receive doses of the new medicine, AIR-001. The study involves giving either a single dose at one time or multiple doses over a period. Depending on which group you are in, you might visit the clinic several times for monitoring and to receive medication.

During these visits, doctors and nurses will carry out various checks and tests. These include blood tests, urine tests, and likely breathing tests to see how your lungs are doing. They will also carefully watch for any side effects and check your overall health. You'll be asked if you've noticed any changes or feel unwell. You'll also need to follow specific instructions about contraception, if applicable.

The exact number of visits, tests, and the total duration of your participation will be explained in detail by the study team. It's important to understand this fully before you agree to take part.

Potential risks and benefits

Taking part in a research study has potential benefits and risks. You might not directly benefit from receiving the study medicine, as the main goal of this early-stage trial is to gather information about safety rather than treat your condition. However, you would be helping doctors learn more about AATD and a potential new treatment, which could help future patients. Possible risks include side effects from the medicine, which are not yet fully known, or discomfort from the study procedures like blood tests. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

Clinical Study Center
Melbourne, Australia· Recruiting
Clinical Study Center 1
Tbilisi, Georgia· Recruiting
Clinical Study Center 2
Tbilisi, Georgia· Recruiting
Clinical Study Center
London, United Kingdom· Recruiting

Common questions

What is Alpha-1 Antitrypsin Deficiency (AATD)?

It's a genetic condition where your body doesn't make enough of a protein that protects your lungs from damage, which can lead to breathing problems.

What is a Phase 1 study?

It's the first stage of testing a new medicine in people. The main goals are to check if it's safe, find the right dose, and see how the body handles it.

Will AIR-001 cure my AATD?

This study is focused on safety and how the medicine works in the body. It's too early to know if AIR-001 will cure AATD or how effective it will be as a treatment.

What does 'PiZZ genotype' mean?

It's a specific genetic marker that confirms you have a particular type of Alpha-1 Antitrypsin Deficiency caused by a change in your DNA.

How long will I be in the study if I participate?

The exact length of time will be explained by the study team, as it depends on whether you receive a single dose or multiple doses of the medicine.

How to find out more

AIRNA Corporation Clinical Trials Information

patients@airna.com 617-609-8790 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.