Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Prospective Phase III Multi-center, 2-Year Placebo Controlled, Double Blind Study to Evaluate the Efficacy and Safety of “Kamada-AAT for Inhalation” 80 mg per Day in Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% ≤ FEV1 ≤ 80% of predicted; FEV1/SVC ≤ 70%), Followed by a 2-Year Open- Label Extension

This research study is investigating a new inhaled medicine called "Kamada-AAT for Inhalation." It's for adults who have a genetic lung condition called Alpha-1 antitrypsin deficiency, where their breathing is moderately to severely affected. For the first two years, some participants will receive the new medicine and others will receive a dummy treatment (placebo), without knowing which they are getting. After these two years, everyone will receive the actual medicine for an additional two years. The main goal is to see if the new medicine helps improve breathing over time. Researchers will also look at lung scans, how far people can walk, and serious breathing problems to understand how well the treatment works and if it's safe.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Kamada Ltd.

Enrolment target

108

Start

23 Sep 2024

Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% ≤ FEV1 ≤ 80% of predicted FEV1/SVC ≤ 70%)and with no history of two or more moderate or one or more severe exacerbations of COPD during the past year.

What is this study about?

This study is a Phase III clinical trial, which means it's one of the final stages of testing a new medicine before it might be made widely available. It focuses on adults with Alpha-1 antitrypsin deficiency, a condition where the body doesn't make enough of a protective protein. This can lead to lung damage and breathing difficulties.

The researchers want to find out if an inhaled version of the missing protein, called Kamada-AAT, can help improve lung function and overall health. They are particularly interested in seeing if it can slow down or reduce the damage to the lungs and help people breathe more easily. The study compares the new medicine to a placebo, which looks identical but contains no active medicine, to ensure any improvements are genuinely due to Kamada-AAT.

Understanding how this medicine works, how safe it is, and if it truly benefits patients is very important. This research aims to provide a clear picture for doctors and patients about whether Kamada-AAT could be a valuable treatment option for Alpha-1 antitrypsin deficiency.

Key takeaways

It's a study for a new inhaled medicine (Kamada-AAT) for Alpha-1 antitrypsin deficiency.
The study lasts for four years, with the first two years possibly involving a dummy treatment (placebo).
It looks at improving breathing and overall lung health.
Only adults with moderate to severe breathing problems are eligible.
Close medical monitoring and regular assessments are part of taking part.
You can stop participating at any time.

Who may be eligible?

This study is looking for adult patients who have a specific genetic lung condition called Alpha-1 antitrypsin deficiency. To join, your breathing must be affected moderately or severely, meaning your lung function test results (FEV1) fall within a certain range.

Another important requirement is that you shouldn't have had more than two moderate or one severe flare-up of your breathing difficulties (often called COPD exacerbations) in the past year. This helps ensure that the study focuses on patients with a more stable form of the condition.

Basically, the study needs adults who have Alpha-1 antitrypsin deficiency with some breathing problems, but haven't had too many serious breathing incidents recently. Both men and women aged 18 and over can be considered.

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?
Do you have moderate to severe breathing difficulties (your doctor can check your FEV1 levels)?
Have you had fewer than two moderate or one severe breathing flare-up in the last year?
Are you willing to commit to four years of study participation?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, it would involve several visits to a study centre over a total of four years. For the first two years, you would be given either the new inhaled medicine, Kamada-AAT, or a dummy treatment (placebo). Neither you nor your study doctor would know which one you are receiving – this is to make sure the results are as fair as possible. After these two years, everyone in the study would then receive the actual Kamada-AAT medicine for another two years.

Throughout the study, you would have regular medical assessments to check your lung function, such as breathing tests (spirometry), and potentially lung scans (CT scans). You'd also do a walking test to see how your exercise ability changes. The research team would closely monitor your health, including any changes in your breathing or any side effects from the medication. You would receive the study medication to inhale daily as instructed.

Potential risks and benefits

Taking part in a study like this can have potential benefits, such as receiving a new medicine for your condition that is not yet widely available, and having very close medical supervision. However, there are also potential risks, including possible side effects from the study medication or the investigational nature of the treatment itself. You might also receive the placebo during the first two years, meaning you wouldn’t get the active treatment. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (5)

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

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Unverified
Finland
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Unverified
Ireland
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Unverified
Sweden
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Unverified
Belgium
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Unverified
Netherlands

Common questions

What is Alpha-1 antitrypsin deficiency?

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

What does a 'placebo' mean?

A placebo is a dummy treatment that looks just like the real medicine but contains no active ingredients. It helps researchers fairly compare the new medicine's effects.

How long will this study last?

The study will last for a total of four years: two years where you might get the medicine or a placebo, followed by two years where everyone gets the medicine.

Will I know if I'm getting the active medicine?

For the first two years, neither you nor your doctor will know if you're receiving the active medicine or the placebo. For the last two years, everyone will receive the active medicine.

What is the main thing the study hopes to achieve?

The main goal is to see if the new inhaled medicine, Kamada-AAT, can improve breathing and lung health in people with Alpha-1 antitrypsin deficiency.