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Study on the Effects of Alpha-1-Proteinase Inhibitor for Patients with Pulmonary Emphysema Due to Alpha-1 Antitrypsin Deficiency

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What is this trial about?

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

This clinical trial is focused on studying a lung condition called Pulmonary Emphysema, which is caused by a deficiency in a protein known as Alpha-1 Antitrypsin. This condition can lead to damage in the lungs, making it difficult to breathe. The study is testing a treatment called Alpha-1-Proteinase Inhibitor (Human), which is given as a weekly injection into a vein. The purpose of the study is to see if this treatment can slow down the loss of lung tissue in people with this condition.

Participants in the study will receive either the active treatment or a placebo, which is a substance with no active medication. The study will compare two different doses of the treatment, 60 mg/kg and 120 mg/kg, to see which is more effective. The study will last for a period of time, during which participants will have regular check-ups and tests to monitor their lung health and overall well-being.

The main goal is to determine if the treatment can help reduce the progression of lung damage, as measured by a special type of scan called CT densitometry. This scan helps doctors see how much lung tissue is being lost over time. The study will also look at other factors, such as the frequency of severe flare-ups of lung symptoms that require hospitalization. By participating in this study, researchers hope to find a better way to manage and treat Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed based on specific criteria such as age, documented alpha1-PI serum levels, and a diagnosis of congenital alpha-1 antitrypsin deficiency (AATD).

    Initial assessments include a blood draw for alpha1-PI serum levels and genotyping if not previously documented.

  2. Step 2

    Treatment assignment

    Participants are randomly assigned to receive either the active treatment or a placebo.

    The active treatment involves the administration of alpha-1-proteinase inhibitor (human) intravenously.

  3. Step 3

    Treatment administration

    The active treatment is administered weekly at a dosage of either 60 mg/kg or 120 mg/kg.

    The placebo consists of a 0.9% sodium chloride injection.

  4. Step 4

    Monitoring and assessments

    Participants undergo regular monitoring to assess the progression of lung tissue loss using CT densitometry.

    Additional assessments include monitoring for severe COPD exacerbations and evaluating lung function.

  5. Step 5

    Study duration

    The study is expected to continue until August 2026.

    Participants will be involved in the study for the duration necessary to meet the study's objectives.

Who can join the trial?

7 criteria

  • You must be between 18 and 70 years old.
  • You need to have a documented total **alpha1-PI serum level** less than 11 micromoles per liter. If this hasn't been documented yet, a blood test will be done during the screening visit.
  • You should have a diagnosis of **congenital AATD** (Alpha-1-Antitrypsin Deficiency) with specific genetic combinations. If this hasn't been documented yet, genetic testing will be done during the screening visit.
  • During the screening visit, your **post-bronchodilator FEV1** (a lung function test) should be between 30% and less than 80% of what is predicted for you, and your **FEV1/FVC** ratio should be less than 70%. This relates to certain stages of lung disease.
  • You should have a **DLCO** (a test measuring lung function) of 60% or less of what is predicted, corrected for hemoglobin, within the past 2 years, or show evidence of **pulmonary emphysema** on a CT scan within the past 2 years, as judged by the investigator.
  • Your **body mass index (BMI)** should be less than 40 kg/m².
  • You must be willing and able to provide informed consent, meaning you agree to participate in the study after understanding all the details.

Who cannot join the trial?

5 criteria

  • Patients who do not have **Pulmonary Emphysema** due to **Alpha-1-Antitrypsin Deficiency** cannot participate. **Pulmonary Emphysema** is a lung condition that causes shortness of breath, and **Alpha-1-Antitrypsin Deficiency** is a genetic disorder that can lead to lung and liver disease.
  • Patients who are not within the specified age range cannot participate. The age range is typically defined by the study and may include adults or specific age groups.
  • Patients who are not part of the specified clinical trial group cannot participate. This refers to the specific group of people the study is focusing on.
  • Patients who are not male or female cannot participate. The study includes both male and female participants.
  • Patients who are considered part of a vulnerable population cannot participate. A vulnerable population includes groups who may be at a higher risk of harm or exploitation.
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Investigated drugs

Alpha1-Proteinase Inhibitor (Human) is a medication used in this clinical trial to treat individuals with pulmonary emphysema caused by Alpha1-Antitrypsin Deficiency (AATD). This medication is administered weekly through an intravenous infusion. The purpose of using this medication in the trial is to slow down the progression of lung tissue loss in patients with AATD. The study is designed to compare the effects of this treatment to a placebo, aiming to demonstrate its efficacy and safety in managing the condition.

What is already known about the treatment

Alpha1-Proteinase Inhibitor (Human) – This medication is administered intravenously, typically on a weekly basis. It is currently being studied in clinical trials for its effectiveness and safety in treating pulmonary emphysema caused by Alpha-1-Antitrypsin Deficiency. The main therapeutic indication is to slow the progression of lung tissue loss in affected individuals. At the molecular level, it works by supplementing the deficient protein, Alpha-1-Antitrypsin, which helps protect lung tissue from damage. It is classified pharmacologically as a proteinase inhibitor.

Investigated diseases

Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency – This is a lung condition where the air sacs in the lungs are damaged, leading to breathing difficulties. It occurs due to a deficiency in a protein called alpha-1-antitrypsin, which normally protects the lungs from damage. Over time, the lack of this protein allows harmful substances to damage the lung tissue, causing the air sacs to lose their elasticity. As the disease progresses, individuals may experience shortness of breath, especially during physical activities. The condition can lead to a decrease in lung function, making it harder to breathe as time goes on. It is a rare disease, often requiring specific medical attention to manage symptoms.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-510030-83-00Protocol codeGTi1201Estimated enrolment345 patientsSponsorGrifols Therapeutics LLC

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