RecruitingRare disease

Efficacy and safety of human alpha1‑proteinase inhibitor weekly infusions in adults with alpha1 antitrypsin deficiency–related emphysema

Verified siteRegistered drugNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

The study focuses on adults who have Alpha1 antitrypsin deficiency and have developed emphysema. The medication being tested is an intravenous solution that contains human alpha1-proteinase inhibitor and is sold under the names Respreeza and Zemaira. The purpose is to determine whether different doses of this medication can slow the loss of lung tissue compared with the standard dose.

Participants will receive a weekly infusion for three years. Some will get the regular dose, others will receive one of two alternative doses, and a small group will receive a placebo. Lung health will be checked regularly with scans using computed tomography and simple breathing tests, and any side effects will be recorded. The study concludes after the three‑year treatment period.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Baseline assessment
After you join the study, you receive a series of initial medical examinations to record your current health status and confirm continued eligibility.
These examinations include lung function tests, imaging of the chest, and blood sampling.
Step 2
Weekly infusion visits
You attend a clinic once each week for an intravenous infusion lasting several minutes.
The infusion contains either respreeza 4,000 mg or respreeza 1,000 mg powder reconstituted as a solution for infusion; the product provides human alpha1‑proteinase inhibitor.
The medication is delivered directly into a vein (intravenous route) at the dose indicated on the product label.
This weekly infusion schedule continues for a total period of 3 years.
Step 3
Regular safety monitoring
At each infusion visit, clinic staff check your vital signs and ask about any new symptoms or side effects.
Any adverse events are recorded and evaluated throughout the study.
Step 4
Periodic lung imaging
Approximately once per year, you undergo a computed tomography (ct) scan of the lungs to measure lung density changes.
The ct scan provides objective data on how the disease is progressing while you receive the study medication.
Step 5
Annual lung function tests
Each year you perform lung function tests, including forced expiratory volume in one second (fev1) and diffusion capacity of carbon monoxide (dlco), to assess respiratory performance.
Step 6
Study completion
After completing the 3‑year infusion period, you attend a final visit for comprehensive evaluation, which includes the last set of ct imaging, lung function testing, and safety assessments.
The data collected are used to determine the effect of the different doses on lung density decline and overall safety.

Who can join the trial?

7 criteria

You must be at least 18 years old and not older than 65 years when you sign the consent form.
You must agree to give written informed consent, which is a signed form showing you understand the study.
You must have a confirmed diagnosis of emphysema that is caused by Alpha‑1 antitrypsin deficiency (AATD). Emphysema is a lung disease that makes breathing difficult, and AATD is a rare genetic condition that lowers a protein that protects the lungs.
Your genetic test must show one of these genotypes: PiZZ, PiZ(null), or Pi(null/null). A genotype is the specific set of genes you have.
Your blood test must have shown a serum Alpha‑1 antitrypsin (AAT) level lower than 11 micromolar (which is the same as less than 50 mg/dL) at any time before the first dose of the study medication.
Both men and women are eligible to take part in the study.
The study may include participants who are considered vulnerable (for example, people who may need extra protection), as long as they meet all other criteria.

Who cannot join the trial?

3 criteria

Cannot take part if you have had an acute illness (a sudden, severe sickness) or a worsening of lung symptoms called a pulmonary exacerbation within the 6 weeks before the first dose.
Cannot take part if you have ever received gene therapy (a treatment that tries to change your genes) for alpha‑1 antitrypsin deficiency.
Cannot take part if you have liver disease secondary to AATD (liver problems caused by alpha‑1 antitrypsin deficiency).

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Aalborg University Hospital
Verified
Aalborg, Denmark
Sygehus Lillebaelt Vejle Sygehus
Verified
Vejle, Denmark

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Investigated drugs

Respreeza (higher dose)
is a medication made from a purified human protein called alpha‑1 proteinase inhibitor. It comes as a powder that is mixed with a liquid right before it is given. In the study, participants receive the medication through an IV (intravenous) infusion, which means it is delivered directly into a vein. The purpose of this higher‑dose version is to see if giving more of the protein can better protect the lungs of people with alpha‑1 antitrypsin deficiency, a condition that can cause emphysema.
Respreeza (lower dose)
contains the same human alpha‑1 proteinase inhibitor as the higher‑dose product, but it is prepared in a smaller amount of powder that is also mixed with a liquid for IV infusion. This lower‑dose version is tested to determine whether a smaller amount of the protein is still effective at slowing lung damage in participants with the same genetic deficiency. By comparing the two doses, researchers hope to find the optimal amount that balances benefit and safety.
Respreeza (standard marketed dose)
is the version of the medication that is already approved and commonly used for long‑term maintenance in adults with alpha‑1 antitrypsin deficiency–related emphysema. It is also given as an IV infusion of the human alpha‑1 proteinase inhibitor. In the trial, this standard dose serves as the reference point, allowing investigators to compare the new higher and lower doses against the established treatment to see if they provide any additional advantage.

Human Alpha1-Proteinase Inhibitor

What is already known about the treatment

Respreeza 4,000 mg powder and solvent for solution for infusion
This product is supplied as a powder that is mixed with a solvent to create a sterile solution for intravenous infusion, usually given once a week. It contains human alpha‑1‑proteinase inhibitor, a protein that is already approved and well‑studied for treating alpha‑1 antitrypsin deficiency‑related lung disease. The medication works by replacing the missing inhibitor, binding to the enzyme neutrophil elastase and preventing it from damaging lung tissue. It is classified as a plasma‑derived protein replacement (ATC code B02AB02) used to slow the progression of emphysema in people with this genetic deficiency.
Respreeza 1,000 mg powder and solvent for solution for infusion
This medication is also provided as a powder that is reconstituted into a sterile solution for intravenous infusion, typically administered once a week. It contains the same human alpha‑1‑proteinase inhibitor that is approved and extensively described in medical literature for managing alpha‑1 antitrypsin deficiency. The drug works by supplying the missing inhibitor, which blocks neutrophil elastase and helps protect the lungs from damage. It belongs to the same class of plasma‑derived protein therapies (ATC B02AB02) used to treat emphysema caused by this deficiency.

Investigated diseases

Alpha-1 proteinase inhibitor deficiency
It is a genetic condition where the body makes too little of a protein that protects the lungs from damage. As the deficiency continues, small airway inflammation can develop and the lung tissue may become less elastic. Over time, the reduced protection can lead to a gradual loss of lung capacity. The condition is often identified early in life but its effects become more noticeable as the person ages. The disease may coexist with other lung problems.
Alpha-1 antitrypsin deficiency
This inherited disorder results in low levels of antitrypsin, a protein that safeguards lung tissue from harmful enzymes. Without enough antitrypsin, the enzymes can damage the walls of the airways, causing them to become stiff and less functional. The damage progresses slowly, leading to a steady decline in breathing efficiency. Symptoms often appear in adulthood and can worsen with exposure to smoke or pollutants. The disease primarily affects the respiratory system.
Emphysema
Emphysema is a chronic lung disease characterized by the destruction of the tiny air sacs that help oxygen enter the blood. The loss of these sacs reduces the surface area for gas exchange, making breathing more difficult. Over time, the lungs become over‑inflated and the airways lose their shape. This results in a gradual decrease in lung capacity and endurance. The condition typically develops slowly and worsens with continued exposure to irritants.