RecruitingRare disease

Study on the Safety and Effectiveness of BEAM-302 with MR0005 and GR0015 for Adults with Alpha-1 Antitrypsin Deficiency-Related Lung or Liver Disease

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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), which can lead to lung and liver diseases. The trial will explore a new treatment called BEAM-302, which is a type of gene therapy. Gene therapy involves using genetic material to treat or prevent disease. In this study, BEAM-302 is administered as a solution for injection through an intravenous infusion, meaning it is delivered directly into the bloodstream.

The purpose of the study is to evaluate the safety and effectiveness of BEAM-302 in adults with AATD-associated lung and/or liver disease. The study is divided into two phases. The first phase will explore different doses to determine the safest and most effective amount of the treatment. The second phase will expand on this by confirming the optimal dose and further assessing how well the treatment works. Participants will receive the treatment and be monitored for any side effects or changes in their condition.

Throughout the study, researchers will collect information on how the treatment affects the levels of certain proteins in the blood, specifically those related to Alpha-1 Antitrypsin. This will help determine the treatment's impact on the disease. The study aims to provide valuable insights into the potential of BEAM-302 as a treatment option for individuals with AATD-related health issues.

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
Joining the study
Upon joining the study, you will be informed about the purpose and procedures of the clinical trial. You will be asked to provide informed consent, which means you agree to participate after understanding the study details.
Step 2
Initial assessment
An initial assessment will be conducted to confirm your eligibility. This includes a review of your medical history and specific tests to ensure you meet the study criteria for Alpha-1 Antitrypsin Deficiency (AATD)-associated lung and/or liver disease.
Step 3
Phase 1: dose exploration
In this phase, you will receive the study medication, BEAM-302, through an intravenous infusion. The goal is to evaluate the safety and tolerability of the medication and determine the optimal dose. You will be monitored for any side effects or adverse reactions.
Step 4
Phase 2: dose expansion
This phase aims to confirm the optimal dose and evaluate the effectiveness of BEAM-302. You will continue to receive the medication and undergo regular assessments to monitor your health and the medication's impact on your condition.
Step 5
Regular monitoring
Throughout the study, you will have regular visits to the clinic for health assessments. These may include blood tests, imaging studies, and other evaluations to track your response to the treatment and ensure your safety.
Step 6
Long-term follow-up
After completing the main phases of the study, you will enter a long-term follow-up period. This involves periodic check-ups to monitor your health and any long-term effects of the treatment. The total study participation is expected to last up to 15 years.

Who can join the trial?

13 criteria

Must be a male or female between 18 and 70 years old.
Must be able to understand the study and provide informed consent, which means agreeing to participate after being informed about the study details.
Must be willing to not use any other experimental treatments during the study.
Must have a diagnosis of Alpha-1 Antitrypsin Deficiency (AATD) and be homozygous for the PiZ mutation, confirmed by genetic testing. This means having two copies of a specific gene mutation related to AATD.
Must show evidence of liver fibrosis, which is a type of liver damage, at certain stages (F1, F2, or F3) based on a liver biopsy or a similar test done within 6 months before joining the study.
Must have a postbronchodilator FEV1 of 40% or more of the predicted value at screening. FEV1 is a measure of lung function that shows how much air you can forcefully exhale in one second.
Must be willing to avoid alcohol, herbal medications, and daily use of nonsteroidal anti-inflammatory drugs (NSAIDs), and not engage in strenuous exercise beyond their usual routine from the start of the screening period through 28 days after receiving the study medication.
Must be willing to participate in this study and a long-term extension study, understanding that the total study participation is 15 years.
Females of childbearing potential must be willing to use highly effective contraception from the signing of the informed consent form through at least 6 months after receiving the study medication. Males must be willing to use highly effective contraception from the time of dosing through at least 4 months after.
Must have a blood total AAT level of less than 11 micromolar (µM) or an equivalent protein level in mg/dL.
If receiving augmentation therapy in regions where it is not standard care, must be willing to stop this therapy for at least 6 weeks before signing the informed consent form and for the length of the study, unless clinically necessary.
Must have evidence of emphysema on a historic CT scan or a DLCO of 70% or less of the predicted value at screening. DLCO is a test that measures how well your lungs transfer gas from the air you breathe to your blood.
Must have a FibroScan result of 12 kPa or less. FibroScan is a test that measures liver stiffness, which can indicate liver damage.

Who cannot join the trial?

10 criteria

Patients with other serious health conditions that could interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who have participated in another clinical trial within the last 30 days.
Patients with a history of allergic reactions to similar treatments.
Patients who are unable to follow the study procedures or attend the required visits.
Patients with a history of drug or alcohol abuse within the last year.
Patients with unstable mental health conditions.
Patients who have received an organ transplant.
Patients with active infections that require treatment.
Patients with a history of cancer within the last 5 years, except for certain skin cancers.

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Verified sites

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Leids Universitair Medisch Centrum (LUMC)
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Leiden, The Netherlands

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Investigated drugs

BEAM-302 is a medication being studied for its potential to help people with Alpha-1 Antitrypsin Deficiency (AATD), which can cause lung and liver problems. This medication is being tested to see if it is safe and how well it works in treating these conditions. The study aims to find the best dose that provides the most benefit with the least side effects. BEAM-302 is designed to improve the health of patients by addressing the underlying issues caused by AATD in the lungs and liver.

What is already known about the treatment

BEAM-302 – BEAM-302 is a medication currently being studied in clinical trials for its potential to treat lung and liver diseases associated with Alpha-1 Antitrypsin Deficiency (AATD). It is administered to adult patients, although the specific form of administration is not detailed in the available data. The main goal of the ongoing study is to assess the safety and effectiveness of BEAM-302, as well as to determine the optimal dose for patients. This medication works at the molecular level by targeting the underlying causes of AATD-related diseases, although the precise mechanism of action is not specified. BEAM-302 is still in the experimental phase, and its classification in pharmacology is not yet fully established.

Investigated diseases

Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease
This condition is caused by a deficiency of the protein alpha-1 antitrypsin, which protects the lungs from damage. Without enough of this protein, the lungs can become damaged over time, leading to chronic obstructive pulmonary disease (COPD) or emphysema. The disease progresses as lung tissue is gradually destroyed, resulting in breathing difficulties and reduced lung function. Patients may experience symptoms such as shortness of breath, wheezing, and chronic cough. The progression can vary, with some individuals experiencing rapid decline while others have a slower progression. Environmental factors, such as smoking, can accelerate lung damage in affected individuals.
Alpha-1 Antitrypsin Deficiency (AATD)-Associated Liver Disease
This condition occurs when abnormal alpha-1 antitrypsin proteins accumulate in the liver, leading to liver damage. Over time, this accumulation can cause liver inflammation, scarring, and eventually cirrhosis. The disease progresses as liver function declines, potentially leading to symptoms such as jaundice, fatigue, and abdominal swelling. The rate of progression can vary, with some individuals developing significant liver damage early in life, while others may not experience symptoms until adulthood. Genetic factors play a significant role in the severity and progression of liver disease in individuals with AATD.