RecruitingPHASE3INTERVENTIONAL

Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation"

This study is investigating a new way to deliver medicine for a lung condition called Alpha-1-Antitrypsin Deficiency (AATD). People with AATD don't have enough of an important protein that protects their lungs, which can lead to breathing problems. The study uses an inhaled medicine called Kamada-AAT to see if it can slow down or stop the worsening of lung disease. Participants will use a device to breathe in the medicine daily. Researchers want to find out if this treatment can improve lung function, as measured by breathing tests, and protect lung tissue, as seen on CT scans. They will also track any side effects. This could be a new, easier way to treat this lung condition.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Kamada, Ltd.

Enrolment target

220

Start

25 Nov 2019

Estimated completion

01 Jun 2031

Alpha 1-Antitrypsin Deficiency

What is this study about?

This research is looking at a new way to help people with a specific lung condition called Alpha-1-Antitrypsin Deficiency (AATD). If you have AATD, your body doesn't make enough of a special protein called alpha-1 antitrypsin, which normally protects your lungs from damage. Without enough of this protein, your lungs can get sick, leading to conditions like emphysema or COPD.

Currently, some treatments involve getting the protein through a drip (infusion). This study is testing an inhaled version of the protein, called Kamada-AAT. The idea is that breathing the medicine directly into your lungs might be more effective and convenient because the medicine goes right to where it's needed most. Researchers want to see if taking this inhaled medicine every day can stop or slow down the damage to your lungs over several years.

The study will compare the Kamada-AAT medicine to a 'placebo' – a substance that looks exactly like the medicine but doesn't contain any active drug. This helps the researchers understand if the medicine itself is causing any changes. For the first two years, some participants will get the actual medicine, and others will get the placebo. For the next two years, everyone in the study will receive the Kamada-AAT medicine. This research is important because it could lead to a new treatment option that is easier to use and potentially more effective for people with AATD-related lung problems.

Key takeaways

This study explores an inhaled treatment for Alpha-1-Antitrypsin Deficiency (AATD).
It aims to see if daily inhalation can slow down lung damage and improve breathing.
Participants will use a daily inhaler and record their symptoms in an e-diary.
Regular clinic visits and tests will track health and lung function.
Some participants will receive a dummy treatment (placebo) initially.
The study lasts four years, plus a six-month follow-up period.

Who may be eligible?

To join this study, you need to be an adult between 18 and 65 years old and have been diagnosed with a severe form of Alpha-1-Antitrypsin Deficiency (AATD). Your blood levels of the protective protein must be low, and your lung function, as measured by breathing tests, should be within a specific range.

There are also some requirements about your previous treatments. You must not have been on AAT treatment for at least eight weeks before the study starts. You also need to be able to use the special device for inhaling the medicine and record your symptoms daily.

You won't be able to join if you have certain other serious health issues, like a very low level of a certain immune protein (IgA), a history of severe reactions to blood products, or if you've had too many severe chest infections recently. Women who are pregnant or breastfeeding cannot participate, and women of childbearing age will need to use contraception during the study.

Quick self-check

Are you between 18 and 65 years old?
Do you have a diagnosis of severe Alpha-1-Antitrypsin Deficiency (AATD)?
Have you stopped any previous AAT treatment for at least 8 weeks?
Are you able to use an inhaler device and record daily information?
Do you not have certain other serious health problems or allergies to blood products?
Are you not pregnant or breastfeeding, and willing to use contraception if female and of childbearing age?

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

What does participation involve?

If you join the study, you'll take the study medicine by inhaling it every day using a special device. You'll also need to clean and disinfect this device daily. To keep track of your health and how you're using the medicine, you'll fill out an electronic diary every day.

You'll also need to visit the clinic regularly for tests and check-ups. During the first two years of the study, you'll have about 11 clinic visits. In the third and fourth years, there will be 5 or 6 visits. After the main treatment period ends, you'll have 3 more follow-up visits over six months. The entire study lasts for four years, plus half a year of follow-up.

Potential risks and benefits

Participating in this study might offer potential benefits, such as receiving a new, potentially effective treatment for your lung condition that could slow down lung damage. However, there are also potential risks, like experiencing side effects from the medicine or the daily routine of using the inhaler and attending clinic visits. You might also receive a placebo for the first two years, meaning you wouldn’t get the active medicine. Your health will be closely monitored by medical professionals throughout the study. Remember, joining a clinical trial is always voluntary, and you have the right to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (9)

University Hospital (UZ) Leuven
Leuven, Belgium· Recruiting
Tays Central Hospital
Tampere, Finland· Recruiting
Beaumont Hospital
Dublin, Ireland· Recruiting
Leiden University Medical Center (LUMC)
Leiden, Netherlands· Recruiting
Canisius Wilhelmina Hospital (CWZ)
Nijmegen, Netherlands· Recruiting
Skåne University Hospital
Malmö, Sweden· Recruiting
University Hospitals Birmingham NHS Foundation Trust
Birmingham, United Kingdom· Recruiting
Royal Infirmary of Edinburgh
Edinburgh, United Kingdom· Recruiting
University Hospital Southampton NHS Foundation Trust
Southampton, United Kingdom· Recruiting

Common questions

What is Alpha-1-Antitrypsin Deficiency (AATD)?

It's a genetic condition where your body doesn't produce enough of a protein that protects your lungs, making you more likely to develop lung problems.

How is this new medicine given?

It's given through an inhaler, so you breathe the medicine directly into your lungs every day.

Will I definitely get the active medicine?

For the first two years, you might get the active medicine or a dummy treatment (placebo). For the next two years, everyone will receive the active medicine.

How long does the study last?

The main part of the study lasts for four years, plus an additional six months for follow-up appointments.

What are the main goals of the study?

Researchers want to see if the inhaled medicine can slow down lung damage, improve breathing, and protect lung tissue in people with AATD.

How to find out more

Sharon Gai

eligibility@innovaate-study.com 972 8 9406472 Official trial record

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