AuthorisedTherapeutic use (Phase IV)Interventional

A Phase 4, Multicenter, Double-blind, Study to Investigate the Efficacy, Safety, and Tolerability of 3 Active Doses of Respreeza® / Zemaira® Weekly Intravenous Infusions Administered over 3 Years as Longterm Maintenance Therapy in Adult Subjects with Emphysema Related to Alpha1 Antitrypsin Deficiency

This research study is for adults who have emphysema, a long-term lung condition, caused by a genetic problem called Alpha-1 Antitrypsin Deficiency. The study is called a 'Phase 4' trial, which means it’s looking at a medicine already approved for use, called Respreeza, in a new way or over a longer period. Participants will receive Respreeza as a weekly infusion for three years. Doctors want to understand how effective and safe this treatment is at helping to slow down lung damage and improve breathing over time, and if it reduces flare-ups of their lung condition. It’s a 'double-blind' study, meaning neither the participants nor their doctors will know which dose of the medicine they are receiving.

At a glance

Status

Authorised

Phase

Therapeutic use (Phase IV)

Sponsor

CSL Behring LLC

Enrolment target

Start

28 May 2026

Alpha1-proteinase inhibitor deficiency Alpha1 antitrypsin deficiency Emphysema

What is this study about?

This study is about a medicine called Respreeza (also known as Zemaira) for adults who have a specific type of lung condition called emphysema. Emphysema makes it hard to breathe because it damages the air sacs in your lungs. The type of emphysema this study focuses on is caused by something called Alpha-1 Antitrypsin Deficiency. This is a genetic condition where your body doesn't make enough of a special protein that protects your lungs from damage.

The main goal of this study is to see how well Respreeza works as a long-term treatment. Participants will receive weekly infusions of the medicine for three years. Doctors will be checking to see if Respreeza helps to slow down the damage to the lungs – for example, by looking at changes in lung density (how solid your lungs are). They also want to make sure the medicine is safe and that people can tolerate it well over a long period.

This is a 'Phase 4' study, which means the medicine has already been approved. This phase helps doctors learn more about how the treatment works over many years for a larger group of people. The study is also 'double-blind,' which means neither you nor your study doctor will know whether you are getting a specific dose of the medicine or if there's a comparison group. This helps make the study results as fair and unbiased as possible.

Key takeaways

This study investigates a medicine called Respreeza for emphysema caused by Alpha-1 Antitrypsin Deficiency.
It aims to see if weekly infusions for three years are effective and safe in adults.
The main goal is to understand if the treatment helps slow down lung damage.
Participants will have regular visits, lung function tests, and health checks.
The study is a 'Phase 4' trial, learning more about an already-approved medicine.

Who may be eligible?

To be able to join this study, you need to be an adult, aged 18 or older. There is no upper age limit, meaning older adults can also take part. Both men and women are welcome to participate.

Crucially, you must have emphysema that is specifically caused by Alpha-1 Antitrypsin Deficiency. This is a particular genetic condition, not just any type of emphysema. The study is designed to help people with this specific cause of their lung problems.

Beyond these basic requirements, the study will have other specific health checks to make sure it's safe for you to take part and that the study results will be clear. For example, doctors will need to review your overall health, other medications you take, and your lung function. These checks are important to ensure the study is right for you.

Quick self-check

Are you 18 years old or older?
Have you been diagnosed with emphysema?
Is your emphysema specifically caused by Alpha-1 Antitrypsin Deficiency?
Are you able to commit to weekly infusions for three years?

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

What does participation involve?

If you join this study, you would receive the study medicine, Respreeza, as an intravenous infusion once a week for a period of three years. An intravenous infusion means the medicine is given directly into your vein, similar to a drip. Throughout these three years, you would have regular visits to the clinic for assessments. These visits would involve taking measurements of your lung function, such as how much air you can breathe out in one second (FEV1) and how well your lungs transfer oxygen (diffusion capacity).

You would also have regular blood tests and other examinations to monitor your health, check for any side effects, and make sure the medicine is safe for you. The study team would also track any times your lung condition worsens rapidly (called severe pulmonary exacerbations) and how long these events last. After the three years of weekly infusions, there would likely be further follow-up appointments to continue monitoring your health, although the full duration of follow-up after the treatment period is not specified here.

Potential risks and benefits

Taking part in this study could potentially offer benefits, such as receiving a long-term treatment and close monitoring of your lung condition by medical experts. There is a chance that the Respreeza treatment could help slow down the progression of your emphysema or reduce the number of severe lung flare-ups. However, as with all medicines, there are potential risks, including side effects from the weekly infusions or other reactions. The study team will carefully monitor you for any adverse effects. It's important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

—
Denmark

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, leading to conditions like emphysema.

What is Respreeza?

Respreeza is a medicine given into a vein (infusion) that aims to replace the missing alpha-1 antitrypsin protein to protect the lungs.

How long will I take part in the study?

You would receive weekly infusions for three years, with regular check-ups during that time and potentially further follow-up.

What does 'double-blind' mean?

It means neither you nor the study doctors will know which specific dose of the medicine you are receiving to keep the study unbiased.

Will I receive any other treatments during the study?

The study focuses on Respreeza, but your study doctor will discuss how your other routine treatments would be managed alongside the study medicine.