A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alpha1-Antitrypsin Deficiency
Alpha-1 antitrypsin deficiency is an inherited condition that causes emphysema, a type of lung damage that makes breathing difficult. People with this condition lack a natural protein that protects the lungs from irritation and breakdown. This trial investigates whether replacing this missing protein through a weekly drip (infusion) into a vein can protect lung tissue. The study tests two different doses of the protein medicine against a dummy drip (placebo). Researchers will use special CT scans to measure lung density, checking whether the treatment helps keep the lungs healthier for longer. They will also count how many severe chest flare-ups occur that require hospital treatment. The study aims to find out if this treatment is safe, effective, and which dose works best.
At a glance
What is this study about?
Our lungs need natural defence systems to stay healthy. In a genetic condition called alpha-1 antitrypsin deficiency, the body does not make enough of a crucial protective protein. Without this protein, the lungs are vulnerable to ongoing irritation and damage. Over time, this leads to emphysema, a serious lung condition that damages air sacs and causes breathlessness.
This clinical trial is testing a treatment called alpha1-proteinase inhibitor. This medicine is made from natural human protein and is designed to replace what the body is missing. In this study, researchers are comparing two different dose levels of the medicine with a harmless salt-water dummy drip (a placebo). This comparison helps doctors understand whether the medicine works well and which dose gives the best balance of benefits and safety.
To see how well the medicine protects the lungs, doctors will use painless CT scans to measure lung thickness and tissue density over time. They will also track whether patients have fewer severe chest infections or flare-ups that require a stay in hospital. If successful, the results could help improve future treatment options for people living with this rare genetic condition.
Key takeaways
- Tests a weekly replacement protein drip for lung damage caused by alpha-1 antitrypsin deficiency.
- Compares two different active doses against an inactive dummy drip (placebo).
- Uses specialized CT scans to measure changes in lung density over time.
- Monitors whether the treatment reduces severe flare-ups that need hospital care.
- Open to adults aged 18 and older with confirmed genetic emphysema.
Who may be eligible?
This trial is open to adults aged 18 and older who have been diagnosed with pulmonary emphysema caused by alpha-1 antitrypsin deficiency. Both men and women can take part.
Because this study looks specifically at lung damage caused by this genetic condition, participants will need to have confirmed tests showing low levels of the protective protein and signs of emphysema on lung scans.
People with certain other severe medical conditions, women who are pregnant or breastfeeding, or those who cannot safely undergo regular CT scans or weekly drips may not be suitable. The trial medical team will review each person's full health history to make sure taking part is safe for them.
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.
- I am aged 18 or older.
- I have been diagnosed with emphysema (a form of COPD).
- I have confirmed alpha-1 antitrypsin deficiency.
- I am able and willing to attend weekly clinic visits for an intravenous drip.
- I am comfortable having regular CT scans and breathing tests.
What does participation involve?
Taking part in this study involves receiving a drip into a vein once a week at a hospital clinic. Because this is a 'double-blind' study, neither you nor your study doctor will choose or know whether you receive the standard dose, a higher dose, or the placebo drip.
During your visits, you will have routine health checks, including blood tests, physical examinations, and breathing tests. You will also have scheduled CT scans of your chest to measure the density of your lung tissue.
You will be asked to report any worsening of your breathing symptoms or chest infections. The study team will follow your progress closely over several months to a few years to monitor your safety and check how your lungs respond to the treatment.
Potential risks and benefits
Locations (5)
- —UnverifiedFinland
- —UnverifiedSweden
- —UnverifiedPoland
- —UnverifiedFrance
- —UnverifiedDenmark
Common questions
What is alpha-1 antitrypsin deficiency?
It is an inherited condition where the liver does not produce enough of a protein that protects the lungs, making them more likely to become damaged over time.
What is a placebo?
A placebo is a dummy treatment designed to look identical to the real medicine—in this case, a simple salt-water drip. It helps researchers prove whether changes are truly caused by the study drug.
Will I know which dose or treatment I am getting?
No. This is a double-blind trial, meaning neither you nor your study doctor will know whether you receive the active medicine or the placebo, ensuring the results are fair and unbiased.
How is the study medicine given?
The medicine is given as a slow drip directly into a vein in your arm once every week at the clinic.
Can I leave the study if I change my mind?
Yes. You can leave the trial at any point, for any reason, without giving an explanation and without it affecting your standard NHS care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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