Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

Study of inhaled alpha1-proteinase inhibitor (Kamada-AAT) in adults with alpha-1 antitrypsin deficiency and moderate to severe airflow limitation

Verified siteInvestigational
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on Alpha-1 Antitrypsin Deficiency, a genetic condition that can cause lung problems. The study will test a medication called Kamada-AAT for Inhalation, which is an inhaled solution containing human alpha1-proteinase inhibitor. The treatment will be delivered using a special device called an eFlow Nebuliser System.

The purpose of this research is to determine if inhaling 80 mg of Kamada-AAT daily can help people with moderate to severe breathing difficulties caused by Alpha-1 Antitrypsin Deficiency. The study will compare the medication to placebo to see how well it works in improving lung function. Participants will use either the study medication or placebo through a nebulizer device for two years.

During the study, participants will have their lung function tested regularly using various breathing tests. The main focus will be on measuring how well the lungs work after using an inhaler. After the initial two-year period, participants will have the opportunity to continue treatment for an additional two years where all participants will receive the active medication.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial treatment period

    You will begin a 2-year double-blind treatment period where you will receive either Kamada-AAT for Inhalation (80 mg daily) or a placebo through inhalation

    The medication or placebo needs to be inhaled daily using a special nebulizer device called PARI Track

    You will need to use the medication for at least 20 out of every 28 days

  2. Step 2

    Regular assessments

    Throughout the 2-year period, your lung function will be regularly measured through several tests:

    - Breathing tests (FEV1 - amount of air you can exhale in one second)

    - CT scans to check lung density

    - 6-minute walk test to assess exercise capacity

    Any breathing problems or worsening of symptoms will be recorded and evaluated

  3. Step 3

    End of initial period

    After completing the first 2 years of treatment, you will attend an end-of-treatment visit

    Your lung function and overall health will be evaluated to determine the effectiveness of the treatment

  4. Step 4

    Extension period

    If you complete the first 2 years, you may continue into a 2-year extension period

    During this extension, all participants will receive the active medication (Kamada-AAT for Inhalation)

    You will need to continue using approved birth control methods during this period, unless post-menopausal or surgically sterilized

Who can join the trial?

10 criteria

  • Age between 18 to 65 years at screening
  • Diagnosed with severe Alpha-1 Antitrypsin deficiency (specific genetic types: Pi(ZZ), Pi(Z/Null), or Pi(Null/Null))
  • Blood levels of Alpha-1 Antitrypsin must be 11 μM or lower at screening
  • Have moderate to severe breathing problems with lung function tests showing: - FEV1 (amount of air you can forcefully exhale) between 40% and 80% of normal - FEV1/SVC ratio (measure of airway obstruction) of 70% or less
  • Either never received Alpha-1 Antitrypsin treatment or have not used it for at least 8 weeks before study start
  • Able to use an electronic diary for at least 20 out of 28 days during initial study period
  • Can use study medication through nebulizer for at least 20 out of 28 days during initial period
  • Must be able to read and sign informed consent form
  • For women: must not be pregnant or breastfeeding, and must use reliable birth control unless post-menopausal or surgically sterilized
  • For men: must agree to use reliable birth control methods during the study

Who cannot join the trial?

12 criteria

  • History of severe lung infections (exacerbations) in the past year that required hospitalization
  • History of two or more moderate lung infections in the past year that required antibiotics or steroids
  • Lung function tests showing values outside the specified range (FEV1 less than 40% or more than 80% of predicted value)
  • Current smokers or those who quit smoking less than 6 months ago
  • History of lung transplant or being on a waiting list for lung transplant
  • Presence of other serious lung diseases besides Alpha-1 Antitrypsin Deficiency
  • Current participation in other clinical trials or use of experimental medications
  • Allergic reactions to similar medications in the past
  • Significant liver disease or other serious medical conditions that could interfere with the study
  • Pregnancy or planning to become pregnant during the study period
  • Unable to use the inhaler device properly or follow study procedures
  • Mental conditions that could affect ability to provide informed consent or follow study instructions
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

  • Kamada-AAT for Inhalation

    is an inhaled form of alpha-1 antitrypsin, a protein that helps protect lung tissue from damage. This medication is designed to treat people who have a genetic condition called Alpha-1 Antitrypsin Deficiency, which can cause lung problems. The medication is inhaled directly into the lungs, where it works to slow down lung damage and help maintain lung function.

  • Bronchodilator

    is a type of medication that helps open up the airways in the lungs. In this study, it's used to help measure how well the lungs are functioning. This medication works by relaxing the muscles around the airways, making it easier to breathe.

What is already known about the treatment

Kamada-AAT for Inhalation - An inhalation therapy containing alpha-1 antitrypsin (AAT) administered at a daily dose of 80 mg through an inhaler device. This investigational medication is being studied in Phase III clinical trials for treating patients with congenital Alpha-1 Antitrypsin Deficiency who have moderate to severe airflow limitations. The medication works by delivering AAT protein directly to the lungs, where it helps protect lung tissue from damage caused by neutrophil elastase, an enzyme that can break down healthy tissue when not properly regulated by AAT. This respiratory medication belongs to the class of protease inhibitors and represents a targeted approach to treating this rare genetic condition by addressing the underlying protein deficiency through local delivery to the affected organ.

Investigated diseases

  • Alpha-1 Antitrypsin Deficiency

    A genetic condition where the body doesn't make enough of a protein called alpha-1 antitrypsin, which protects the lungs and liver from damage. The lack of this protein allows enzymes to harm lung tissue over time, leading to breathing problems and lung disease. The condition primarily affects the lungs, causing symptoms such as shortness of breath, wheezing, and decreased lung function. The disease typically develops slowly over years, with early symptoms often appearing in adulthood. People with this condition may experience gradually worsening breathing difficulties, particularly during physical activity.

  • Chronic Obstructive Pulmonary Disease (COPD)

    A progressive lung condition that causes airflow blockage and breathing-related problems. It involves the narrowing of airways, which makes breathing increasingly difficult over time. The condition causes symptoms such as shortness of breath, chronic cough, and increased mucus production. The airways become inflamed and thickened, making it harder to move air in and out of the lungs. People with COPD often experience episodes where symptoms temporarily worsen, known as exacerbations.

Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-516054-21-00Protocol codeKamadaAATInhaled008Estimated enrolment220 patientsSponsorKamada Ltd.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.