Not recruitingRare disease

Study on the Safety and Tolerability of WVE-006 for Patients with Alpha-1 Antitrypsin Deficiency (AATD) Pi*ZZ

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). AATD is a genetic disorder that can lead to lung and liver problems. The study will use a treatment called WVE-006, which is a solution for injection. WVE-006 is a type of medication known as an oligonucleotide, which is a small piece of genetic material designed to target specific genes.

The purpose of the study is to evaluate the safety and tolerability of WVE-006 in participants with a specific genetic form of AATD, known as Pi*ZZ. Participants will receive either single or multiple doses of WVE-006. The study will monitor how the body processes the medication and any potential side effects. Participants will receive the medication through a subcutaneous injection, which means it is injected under the skin.

Throughout the study, researchers will observe changes in the levels of a protein called M-AAT in the blood, which is related to the condition. The study will also measure how much of the medication is present in the blood over time. This research aims to gather important information about how WVE-006 works in people with AATD and to ensure it is safe for use. The study is expected to continue until the end of 2025.

The research process

The trial runs in 4 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 will be conducted to confirm eligibility. This includes a medical evaluation to ensure you are healthy or have mild to moderate lung or liver disease caused by alpha-1 antitrypsin deficiency (AATD). Genetic testing will confirm the presence of the Pi*ZZ genotype.
You must have been a non-smoker for at least one year prior to this assessment.
Step 2
Single ascending dose phase
In this phase, you will receive a single dose of the study medication, WVE-006, through a subcutaneous injection, which means it is injected under the skin.
The purpose is to evaluate the safety and how your body processes the medication. Your blood will be tested to measure the levels of a protein called M-AAT and the concentration of WVE-006 in your plasma over time.
Step 3
Multiple ascending dose phase
If you continue to the next phase, you will receive multiple doses of WVE-006 over a specified period. The frequency and duration of these doses will be determined by the study team.
This phase aims to further assess the safety and how your body responds to repeated doses. Blood tests will continue to monitor the levels of M-AAT protein and the concentration of WVE-006 in your plasma.
Step 4
Monitoring and follow-up
Throughout the study, you will be closely monitored for any side effects or changes in your health. Regular follow-up visits will be scheduled to ensure your safety and to collect necessary data.
The study is expected to conclude by the end of 2025, and you will be informed of any significant findings related to your participation.

Who can join the trial?

5 criteria

The participant must be healthy or have mild to moderate lung disease caused by Alpha-1 antitrypsin deficiency (AATD) with a lung function test result of at least 50%.
The participant can also have stable mild liver disease caused by AATD, with a FibroScan result of 10 kPa or less. A FibroScan is a special test that checks the stiffness of the liver.
The participant must have a genetic test confirming they have the Pi*ZZ type of AATD.
The participant must have been a non-smoker for at least 1 year before the screening.
The study is open to both male and female participants.

Who cannot join the trial?

9 criteria

Participants with any other serious health condition that could affect their safety or the study results.
Participants who are pregnant or breastfeeding.
Participants who have had a recent infection or illness that could interfere with the study.
Participants who are currently taking certain medications that might interfere with the study treatment.
Participants who have a history of allergic reactions to similar treatments.
Participants who have participated in another clinical trial recently.
Participants who have a history of substance abuse or alcohol dependency.
Participants who have a mental health condition that might affect their ability to participate in the study.
Participants who have a history of non-compliance with medical treatments or study protocols.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Turku University Hospital
Turku, Finland
Verified
Universitaetsklinikum Aachen AöR
Aachen, Germany

Trial locations

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Investigated drugs

WVE-006 is a medication being studied to see if it is safe and well-tolerated in people with a specific genetic condition called AATD Pi*ZZ. This condition affects the lungs and liver, and the medication aims to help manage or improve the symptoms associated with it. The study is looking at how the body processes the medication and how it affects the body over time. Participants in the trial will receive this medication to help researchers understand its potential benefits and any side effects it might have.

Wve-006

What is already known about the treatment

WVE-006 – WVE-006 is administered in a clinical trial setting to evaluate its safety and tolerability for individuals with Alpha-1 antitrypsin deficiency (AATD), specifically those with the Pi*ZZ genotype. This medication is currently in the early stages of clinical research, and its effects are being studied to understand how it can help manage AATD. The main therapeutic indication for WVE-006 is to address the deficiency of alpha-1 antitrypsin, a protein that protects the lungs and liver from damage. At the molecular level, WVE-006 is designed to interact with specific genetic components to increase the production of functional alpha-1 antitrypsin protein. It falls under the pharmacological classification of gene therapy or genetic medicine, aiming to correct or modify genetic issues causing the deficiency.

Investigated diseases

Alpha-1 antitrypsin deficiency – Alpha-1 antitrypsin deficiency is a genetic disorder that affects the production of a protein called alpha-1 antitrypsin, which protects the lungs and liver from damage. In individuals with this condition, the liver produces an abnormal form of the protein that cannot be released into the bloodstream, leading to its accumulation in the liver. This can cause liver damage and increase the risk of liver disease. The lack of alpha-1 antitrypsin in the bloodstream also leaves the lungs vulnerable to damage from enzymes, which can lead to lung diseases such as emphysema. The progression of the disease varies among individuals, with some experiencing significant lung or liver problems, while others may have mild or no symptoms. The condition is inherited in an autosomal recessive pattern, meaning both copies of the gene in each cell have mutations.