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

Investigational

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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 is associated with lung disease, specifically pulmonary emphysema. The trial will explore the effects of a new treatment called NTLA-3001. This treatment involves a type of gene therapy that aims to introduce a healthy copy of a gene called SERPINA1 into the body. The treatment is delivered through an intravenous infusion, which means it is given directly into the bloodstream.

The purpose of the study is to evaluate the safety and tolerability of NTLA-3001 in adults with AATD-associated lung disease. Participants will receive a single treatment and will be monitored to see how their bodies respond. The study will look at various aspects, including how the treatment moves through the body and how it affects the levels of certain proteins related to the disease. The study will also check for any immune responses, such as the development of antibodies, which are proteins the body makes to fight off what it sees as foreign substances.

Throughout the study, participants will undergo regular check-ups and tests to ensure their safety and to gather information on how the treatment is working. The trial is designed to provide valuable insights into the potential of NTLA-3001 as a treatment for lung disease associated with Alpha-1 Antitrypsin Deficiency. This research could lead to new ways to manage and treat this condition in the future.

The research process

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

Step 1
Initial assessment
Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and specific laboratory tests to ensure all criteria are met.
Participants must be between 18 and 75 years old and have a diagnosis of alpha-1 antitrypsin deficiency-associated lung disease.
Step 2
Pre-treatment preparation
Participants are required to refrain from alcohol and nicotine use, confirmed by a negative cotinine test.
For those on alpha-1 antitrypsin augmentation therapy, it must be paused for at least 4 weeks before receiving the study treatment.
Step 3
Treatment administration
The treatment involves a single intravenous infusion of the study medication, which includes serpina1(human)-aav8-1 and messenger RNA encoding cas9.
The infusion is administered in a controlled clinical setting to monitor safety and tolerability.
Step 4
Post-treatment monitoring
Participants are monitored for any treatment-emergent adverse events (TEAEs) following the infusion.
Regular follow-up visits are scheduled to assess the levels of circulating alpha-1 antitrypsin protein and other study-related parameters.
Step 5
Long-term follow-up
Participants are required to refrain from initiating augmentation therapy for at least 12 weeks post-dosing.
Additional assessments include questionnaires to evaluate respiratory health and overall quality of life.

Who can join the trial?

12 criteria

Participants must be men or women aged between 18 and 75 years old.
Participants must provide written consent to join the study.
Participants must agree to follow rules about alcohol consumption.
Participants must have a negative test for cotinine, which shows they are not using tobacco or nicotine, and agree to follow rules about smoking and nicotine use.
Participants must have a diagnosis of lung disease related to **AATD** (Alpha-1 Antitrypsin Deficiency), meeting specific criteria.
Participants must have either used all available standard treatments or chosen not to use them. If they are on **AAT augmentation therapy** (a treatment to increase levels of a specific protein), they must be able to stop this therapy for at least 4 weeks before the study and not start it again for at least 12 weeks after the study treatment.
Participants must not have been diagnosed with conditions like protein-losing enteropathy or nephropathy, or have a history of low levels of a protein called albumin in their blood.
Participants must have a specific level of **AAV TAb**, which is a type of antibody, below a certain limit.
Participants must meet certain laboratory test criteria.
Participants must have normal levels of certain blood clotting factors, or any differences must be considered not significant by the study doctor.
Male participants must agree to follow rules about contraception and not donate sperm during the study.
Female participants must not be pregnant or breastfeeding and must agree to follow rules about contraception and not donate eggs.

Who cannot join the trial?

8 criteria

Individuals who are not adults cannot participate. This means only adults can join the study.
Participants must not have any other serious health conditions that could interfere with the study.
Pregnant or breastfeeding women are not allowed to participate.
Individuals who have participated in another clinical trial recently may be excluded.
Participants must not have a history of allergic reactions to similar treatments.
People with certain lung diseases other than pulmonary emphysema may not be eligible. Pulmonary emphysema is a condition where the air sacs in the lungs are damaged, making it hard to breathe.
Individuals who are unable to follow the study procedures or attend all required visits may be excluded.
Participants must not have any condition that the study doctors believe would make it unsafe for them to participate.

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Investigated drugs

NTLA-3001 is an investigational medication being studied for its potential to treat lung disease associated with Alpha-1 Antitrypsin Deficiency (AATD). This medication is designed to assess its safety and how well it is tolerated by patients after a single treatment. The study aims to understand how the body processes NTLA-3001 and its effects on the body, which could provide insights into its potential as a treatment option for individuals with AATD-related lung conditions.

What is already known about the treatment

NTLA-3001 – This medication is administered as a single treatment, likely through an intravenous infusion, as it is common for investigational therapies in clinical trials. NTLA-3001 is currently in the early stages of clinical research, specifically a Phase 1/2 trial, to assess its safety and tolerability in individuals with lung disease related to Alpha-1 Antitrypsin Deficiency (AATD). The main therapeutic indication for NTLA-3001 is pulmonary emphysema associated with AATD. At the molecular level, NTLA-3001 is designed to target and modify specific genes to address the underlying cause of the disease, potentially through a gene-editing mechanism. It falls under the pharmacological classification of gene therapy or genetic medicine.

Investigated diseases

Pulmonary emphysema – Pulmonary emphysema is a chronic lung condition characterized by damage to the air sacs (alveoli) in the lungs. This damage leads to the destruction of the walls of the alveoli, resulting in larger air spaces and reduced surface area for gas exchange. As the disease progresses, it becomes increasingly difficult for the lungs to transfer oxygen to the bloodstream, leading to shortness of breath. The condition is often associated with long-term exposure to irritants such as tobacco smoke, air pollution, or occupational dust. Over time, individuals with emphysema may experience a persistent cough, wheezing, and a feeling of tightness in the chest. The disease is a form of chronic obstructive pulmonary disease (COPD) and can significantly impact daily activities and quality of life.