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

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What is this study about?

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.

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 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 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 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 Study?

  • 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 Study?

  • 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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Katholieke Universiteit te Leuven Leuven Belgium

Other Sites

Site Name City Country Status
Canisius Wilhelmina Ziekenhuis Nijmegen The Netherlands
University Of Skane Malmo Sweden
Pirkanmaan hyvinvointialue Tampere Finland
Beaumont Hospital Dublin Ireland
Lnrir Umwimzxvvtwj Mkihjkf Cvgxfyx (jnqej Leiden The Netherlands

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
28.10.2019
Finland Finland
Not recruiting
28.10.2019
Ireland Ireland
Not recruiting
28.10.2019
Sweden Sweden
Not recruiting
28.10.2019
The Netherlands The Netherlands
Not recruiting
28.10.2019

Trial locations

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.

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 ID:
2024-516054-21-00
Protocol code:
KamadaAATInhaled008
NCT ID:
NCT04204252
Trial Phase:
Therapeutic confirmatory (Phase III)

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