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Clinical Trials for Emphysema

There are currently 5 ongoing clinical trials studying treatments for emphysema, particularly focusing on Alpha-1 Antitrypsin Deficiency (AATD)-related lung disease. These trials are evaluating both protein replacement therapies and innovative gene therapy approaches across multiple European countries including Denmark, Estonia, Finland, France, Ireland, Poland, Spain, and Sweden.

Clinical trial locations

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

This trial is evaluating the long-term safety and tolerability of INBRX-101 in adults with AATD-related emphysema. INBRX-101 is a specially designed protein called human alpha-1-proteinase inhibitor immunoglobulin G fusion protein, which is delivered through intravenous infusion.

Who can participate: Adults between 18 and 80 years old with a diagnosis of AATD showing evidence of lung emphysema are eligible. Participants must have a lung function measurement (FEV1) of at least 30% of predicted normal values and must be current non-smokers. They need to have an FEV1/FVC ratio less than 0.7 after using a bronchodilator medication.

Who cannot participate: People with lung conditions other than AATD-related emphysema, those outside the age range, pregnant or breastfeeding women, those participating in other clinical trials, individuals with recent major surgery, those with drug or alcohol abuse history, and anyone with known allergies to the study medication are excluded.

Study focus: The main goal is to monitor the long-term safety of INBRX-101 by tracking any side effects and adverse events. Researchers will use CT scans to measure changes in lung density over time and assess how the body processes the treatment. They will also check whether the immune system develops antibodies against the medication.

The trial is being conducted in Spain, Poland, Ireland, Sweden, and Denmark.

Study on Long-term Safety of Weekly Intravenous Alpha-1-Proteinase Inhibitor for Patients with Pulmonary Emphysema Due to Alpha-1-Antitrypsin Deficiency

This trial focuses on gathering long-term safety data for weekly intravenous Alpha-1-Proteinase Inhibitor treatment in people with AATD-related lung disease. The treatment is administered at a dosage of 60 mg per kilogram of body weight once weekly.

Who can participate: Patients who completed a previous study called GTi1201 are eligible, or those who experienced significant lung function decline (at least 134.4 mL per year in FEV1) after the Week 104 visit of that earlier study. All participants must be willing and able to provide informed consent.

Who cannot participate: People with severe allergic reactions to the study medication, other serious lung diseases not related to AATD, those currently in another clinical trial, pregnant or breastfeeding women, individuals with recent drug or alcohol abuse, those with recent major surgery, people with uncontrolled high blood pressure or heart disease, those with active infections requiring antibiotics, recent cancer diagnoses (except certain skin cancers), recent blood transfusions, non-AATD-related liver disease, and those unable to comply with study procedures are excluded.

Study focus: The trial aims to collect safety information over a two-year period, monitoring participants through regular check-ups and assessments including lung function tests and questionnaires about respiratory health and quality of life. The study is expected to continue until October 2028.

This trial is being conducted in Denmark, Estonia, Poland, Sweden, France, and Finland.

Study Comparing INBRX-101 and Human Alpha1-Proteinase Inhibitor for Adults with Alpha-1 Antitrypsin Deficiency Emphysema

This is a double-blind study comparing INBRX-101 to the existing plasma-derived Alpha1-Proteinase Inhibitor augmentation therapy. Neither participants nor researchers know which treatment is being given, which helps ensure unbiased results.

Who can participate: Adults between 18 and 80 years old with diagnosed AATD and evidence of emphysema can join. Participants must have an FEV1 between 30% and 80% of predicted values and must be current non-smokers at the time of enrollment.

Who cannot participate: Those with known allergies to the study medication, other lung diseases besides AATD, recent lung infections or breathing-related illnesses, people taking medications that could interfere with the study treatment, those with drug or alcohol abuse history, pregnant or breastfeeding women, and individuals unable to follow study procedures are excluded.

Study focus: The trial evaluates how well INBRX-101 maintains certain protein levels in the blood over up to 32 weeks compared to standard treatment. Researchers will measure blood levels of functional alpha-1 antitrypsin, monitor safety and adverse events, and assess the body’s immune response to the treatment. The study includes a placebo group receiving an inactive solution for comparison.

The trial is being conducted in Spain, Poland, Ireland, Sweden, and Denmark.

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

This trial tests whether Alpha-1-Proteinase Inhibitor treatment can slow down lung tissue loss in people with AATD. It compares two different doses (60 mg/kg and 120 mg/kg) given weekly through intravenous infusion against a placebo.

Who can participate: Adults between 18 and 70 years old with documented alpha1-PI serum levels below 11 micromoles per liter and confirmed congenital AATD diagnosis are eligible. Participants must have post-bronchodilator FEV1 between 30% and less than 80% of predicted values with an FEV1/FVC ratio below 70%. They must have DLCO of 60% or less of predicted (corrected for hemoglobin) or show emphysema evidence on CT scan within the past two years. Body mass index must be less than 40 kg/m².

Who cannot participate: The trial excludes people without AATD-related emphysema, those outside the specified age range, and members of vulnerable populations who may require extra protection.

Study focus: The main goal is to determine if treatment can reduce the progression of lung damage as measured by CT densitometry, which shows how much lung tissue is being lost over time. Secondary outcomes include monitoring the frequency of severe symptom flare-ups requiring hospitalization and overall lung function changes. The study is expected to continue until August 2026.

This trial is being conducted in Denmark, Poland, Sweden, France, and Finland.

Study on the Safety of NTLA-3001 for Adults with Alpha-1 Antitrypsin Deficiency-Related Lung Disease

This trial represents a different approach, using gene therapy rather than protein replacement. NTLA-3001 involves introducing a healthy copy of the SERPINA1 gene into the body through a single intravenous infusion.

Who can participate: Men and women aged 18 to 75 years with diagnosed AATD-related lung disease can participate. They must provide written consent, agree to avoid alcohol consumption, and test negative for cotinine (showing no tobacco or nicotine use). Participants must either have tried all available standard treatments or chosen not to use them. Those on AAT augmentation therapy must be able to stop it for at least 4 weeks before the study and not restart for at least 12 weeks after treatment. Participants must have specific AAV antibody levels below a certain threshold and meet various laboratory test requirements. Men must agree to contraception rules and not donate sperm, while women must not be pregnant or breastfeeding and must follow contraception guidelines.

Who cannot participate: Children cannot participate. Those with other serious health conditions that could interfere with the study, pregnant or breastfeeding women, recent participants in other clinical trials, people with allergies to similar treatments, those with certain lung diseases other than pulmonary emphysema, individuals unable to follow study procedures or attend all visits, and anyone the study doctors believe would be unsafe to participate are excluded.

Study focus: The trial aims to evaluate the safety and tolerability of NTLA-3001 after a single treatment. Researchers will monitor how the body processes the gene therapy, check for immune responses including antibody development, and assess its effects on alpha-1 antitrypsin protein levels. Participants will undergo regular check-ups with respiratory health questionnaires and quality of life assessments.

This trial is being conducted exclusively in Ireland.

Summary

All five clinical trials focus specifically on AATD-related emphysema, reflecting the genetic nature of this particular form of lung disease. Four of the five trials are testing protein replacement therapies using various forms of Alpha-1-Proteinase Inhibitor, while one trial takes an innovative gene therapy approach with NTLA-3001.

There is a notable concentration of trials in Nordic countries, with Denmark and Sweden hosting four trials each, while Poland participates in four trials as well. Ireland stands out as the only location for the gene therapy trial. The trials span multiple European countries, providing broad geographic access for patients seeking experimental treatments.

Most trials require participants to be non-smokers and have moderate lung function impairment (typically FEV1 between 30% and 80% of predicted values), suggesting these treatments target patients in the middle stages of disease progression. The age ranges generally span from 18 to 80 years, though the gene therapy trial has a narrower upper limit of 75 years.

The protein replacement trials focus primarily on long-term safety monitoring and comparing different dosing regimens, while the gene therapy trial represents a potentially transformative approach by addressing the underlying genetic cause of the disease. Several trials are follow-up or extension studies, indicating ongoing commitment to understanding the long-term effects of these treatments.

Ongoing Clinical Trials on Emphysema

  • Study Comparing INBRX-101 and Human Alpha1-Proteinase Inhibitor for Adults with Alpha-1 Antitrypsin Deficiency Emphysema

    Not recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    Denmark Ireland Poland Spain Sweden

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