AuthorisedHuman Pharmacology (Phase I)- First administration to humansInterventional

A Phase 1 Study of AIR-001 in Adults with AATD.

This study, called AIR-001, is a new clinical trial for adults living with a condition called Alpha-1 Antitrypsin Deficiency, often shortened to AATD. AATD is a genetic disorder that can cause serious long-term lung or liver problems. The main goal of this particular study is to test a new treatment, also called AIR-001, for the very first time in people. This 'Phase 1' type of study focuses on understanding if a new treatment is safe and how it behaves inside the human body, rather than how well it treats the condition. Researchers will be carefully checking how participants react to the new treatment. It's open to all adults aged 18 and over, regardless of their gender. This initial step is vital to see if the treatment could eventually help people with AATD in the future.

At a glance

Status

Authorised

Phase

Human Pharmacology (Phase I)- First administration to humans

Sponsor

Airna Corp.

Enrolment target

Start

17 Apr 2026

Alpha-1 Antitrypsin Deficiency

What is this study about?

This study is about a health condition called Alpha-1 Antitrypsin Deficiency, or AATD for short. People with AATD don't have enough of a protein called alpha-1 antitrypsin in their blood. This protein usually protects the lungs from damage, and without enough of it, people can develop lung diseases like emphysema. It can also affect the liver and other parts of the body. Since AATD is a genetic condition, it's something people are born with.

The study is testing a new potential treatment called AIR-001. This is a very early-stage study, known as 'Phase 1'. When a new medicine is developed, it goes through different phases of testing. Phase 1 is the first time it's given to people, after lots of testing in the lab. The main aim of this first phase is to see if the treatment is safe for humans and to understand how it works in the body, such as how much of it the body absorbs and how it gets rid of it. It's not primarily designed to see if it cures the condition, but rather to make sure it's safe enough to continue testing.

By taking part, you would be helping doctors and scientists learn more about this new treatment. The information gathered from this study, even if it's just about safety, is very important. It lays the groundwork for future studies that might eventually show if AIR-001 can effectively treat or manage AATD. This step is crucial for potentially bringing new medicines to people who need them.

Key takeaways

This is an early-stage study (Phase 1) of a new potential treatment for AATD.
The main aim is to check the safety of the new treatment, AIR-001, in humans.
Participants must be adults (18 years or older), regardless of gender.
You would receive close medical monitoring and will have regular clinic visits.
It's a way to contribute to developing new medicines for AATD.
You can stop participating at any time without affecting your normal healthcare.

Who may be eligible?

To be able to join this study, you need to be an adult, meaning you must be 18 years old or older. There is no upper age limit mentioned, so older adults can also be considered.

The study is open to both men and women. Your gender does not affect whether you can take part.

Beyond age and gender, specific medical details will be checked by the study doctors. These checks are important to make sure the study will be safe for you and that the results will be clear. For example, they might look at your current health, any other medical conditions you have, and the medicines you are currently taking. This information helps the research team decide if the study is the right fit for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a diagnosis of Alpha-1 Antitrypsin Deficiency (AATD)?
Are you willing to attend regular clinic visits and health check-ups?
Are you comfortable with receiving a new, untested treatment?
Do you understand that the primary goal is safety, not necessarily a cure?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, it will involve several visits to the study clinic or hospital. These visits will help the doctors monitor your health and how you react to the study treatment. You will receive the new treatment, AIR-001, which will be given in a specific way determined by the study plan. You won't know if you're getting the active drug or a placebo unless it's an open-label study.

During your visits, various assessments will be carried out. This might include physical examinations, blood tests, urine tests, and possibly other scans or tests depending on what the study needs to measure. These tests help the researchers understand how the treatment is working and if there are any side effects. You will need to attend all scheduled follow-up appointments, which could continue for a period after you stop taking the treatment, to ensure your health is stable. The total duration of your participation in the study, from your first visit to your final follow-up, will be explained in detail by the study team.

Potential risks and benefits

Participating in this study might offer some potential benefits, such as contributing to scientific understanding of AATD and its treatments, and you would receive very close medical care and monitoring. However, as AIR-001 is a new treatment being given to people for the first time, there are potential risks. You might experience side effects, some of which could be unexpected or serious. The study team will explain all known and potential risks to you. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (6)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Sweden
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Unverified
Ireland
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Unverified
Spain
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Unverified
Netherlands
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Unverified
Portugal
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Unverified
Germany

Common questions

What is Alpha-1 Antitrypsin Deficiency (AATD)?

AATD is a genetic condition where your body doesn't make enough of a special protein called alpha-1 antitrypsin. This protein helps protect your lungs from damage, and when it's missing, it can cause breathing problems or affect your liver.

What is a 'Phase 1' study?

A Phase 1 study is the very first time a new medicine is given to people. Its main goal is to find out if the treatment is safe and how the body handles it, like how much to give and how long it stays in the body.

Will AIR-001 cure my AATD?

This early-stage study is focused on safety, not on whether it cures AATD. It's a first step to see if the treatment is safe enough to test further for its effectiveness in future studies.

Who can join this study?

Any adult aged 18 or older, male or female, may be eligible. The study team will also check your other medical information to make sure it's safe for you to participate.

What if I get side effects from the treatment?

The study team will monitor you very closely for any side effects. They will explain all known and potential risks before you join, and you can always ask questions and share any concerns.