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Study on Long-Term Safety of INBRX-101 for Adults with Alpha-1 Antitrypsin Deficiency Emphysema

Verified siteInvestigationalNo placebo
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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 condition known as Alpha-1 Antitrypsin Deficiency (AATD) Emphysema. This is a genetic disorder that can lead to lung problems, including emphysema, which is a type of chronic lung disease. The trial will use a treatment called INBRX-101, which is a concentrate for solution for infusion. This treatment contains a special protein called human alpha-1-proteinase inhibitor immunoglobulin G fusion protein, recombinant, designed to help manage the symptoms of AATD emphysema.

The purpose of the study is to evaluate the long-term safety and tolerability of INBRX-101 in adults with AATD emphysema. Participants in the study will receive the treatment through an infusion, which is a method of delivering medication directly into the bloodstream. The study will monitor participants over a period to observe any side effects and to assess how well the treatment works in managing the condition.

Throughout the study, researchers will keep track of any adverse events, which are any unwanted effects that occur during the treatment. They will also look at changes in lung density using a method called computed tomography (CT), which is a type of imaging that helps visualize the lungs. Additionally, the study will examine how the body processes the treatment and whether any antibodies, which are proteins made by the immune system, develop against INBRX-101. The study aims to provide valuable information on the safety and effectiveness of this treatment for people with AATD emphysema.

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 criteria such as age, diagnosis of alpha-1 antitrypsin deficiency (AATD), and current non-smoking status.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to establish baseline health status. This includes lung function tests to measure forced expiratory volume (FEV1) and other relevant health checks.

  3. Step 3

    Treatment administration

    The treatment involves receiving INBRX-101, a concentrate for solution for infusion, administered at a dosage of 50mg/ml.

    The medication is given through an infusion, which is a method of delivering the drug directly into the bloodstream over a set period.

  4. Step 4

    Ongoing monitoring

    Regular monitoring is conducted to evaluate the long-term safety and tolerability of INBRX-101. This includes tracking any adverse events and changes in lung density using CT scans.

    Blood tests are performed to assess the serum pharmacokinetic (PK) profile of INBRX-101 and to check for the presence of anti-drug antibodies.

  5. Step 5

    End of treatment evaluation

    At the end of the treatment period, a comprehensive evaluation is conducted to assess the overall impact of the treatment on lung function and any potential side effects experienced during the study.

Who can join the trial?

6 criteria

  • Must be a male or female between 18 and 80 years old at the time of screening.
  • Must have a diagnosis of **Alpha-1 Antitrypsin Deficiency (AATD)**, which is a genetic condition that can affect the lungs.
  • Must show evidence of **emphysema** caused by AATD. Emphysema is a lung condition that makes it hard to breathe.
  • Must have a **FEV1** (Forced Expiratory Volume in one second) that is 30% or more of what is predicted for a person of the same age, gender, and size. This is a measure of lung function.
  • For new patients in Cohort 1, the **FEV1/FVC** (Forced Vital Capacity) ratio must be less than 0.7 after using a bronchodilator, which is a medication that helps open the airways in the lungs.
  • Must be a current non-smoker, meaning you do not smoke cigarettes or use other tobacco products.

Who cannot join the trial?

9 criteria

  • Patients who have a different lung condition other than Alpha-1 Antitrypsin Deficiency (AATD) Emphysema cannot participate. This is a specific lung disease caused by a lack of a protein that protects the lungs.
  • Patients who are not within the specified age range for the study cannot participate. The study is looking for participants within certain age groups.
  • Patients who are not able to follow the study procedures or attend the required visits cannot participate.
  • Patients who have a medical condition that might interfere with the study or make it unsafe for them to participate cannot join the study.
  • Patients who are pregnant or breastfeeding cannot participate in the study.
  • Patients who are currently participating in another clinical trial cannot join this study.
  • Patients who have had a recent major surgery or are planning to have surgery during the study period cannot participate.
  • Patients who have a history of drug or alcohol abuse that might interfere with the study cannot participate.
  • Patients who have a known allergy to the study medication or its ingredients cannot participate.
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Investigated drugs

INBRX-101 is a medication being studied for its long-term safety and effectiveness in treating adults with Alpha-1 Antitrypsin Deficiency (AATD) emphysema. This condition is a genetic disorder that can lead to lung disease. The trial aims to understand how well patients tolerate this medication over an extended period.

What is already known about the treatment

INBRX-101 – This medication is administered intravenously, meaning it is given directly into a vein. It is currently being studied in clinical trials, specifically in a Phase 2 study, to assess its long-term safety and effectiveness. The main therapeutic use of INBRX-101 is for treating Alpha-1 Antitrypsin Deficiency (AATD) Emphysema, a condition affecting the lungs. At the molecular level, INBRX-101 works by supplementing the body with a protein that is deficient in individuals with AATD, helping to protect lung tissue from damage. It is classified pharmacologically as a protein replacement therapy.

Investigated diseases

Alpha-1 Antitrypsin Deficiency (AATD) Emphysema – This is a genetic disorder that affects the lungs and liver, primarily leading to lung disease. It occurs due to a deficiency of the protein alpha-1 antitrypsin, which protects the lungs from damage. Without enough of this protein, the lungs can become damaged, leading to emphysema, a condition where the air sacs in the lungs are gradually destroyed. Individuals with this condition may experience shortness of breath, wheezing, and a chronic cough. Over time, the lung function continues to decline, making breathing increasingly difficult. The progression of the disease can vary, with some individuals experiencing more rapid deterioration than others.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-508137-14-00Protocol codeINBRX101-01-202Estimated enrolment195 patientsSponsorSanofi AATD Inc.

sourced from the EU Clinical Trials Register and site verification

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