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

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What is this study about?

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.

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.

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.

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.

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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name City Country Status
Universitaetsklinikum Aachen AöR Aachen Germany
Turku University Hospital Turku Finland

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Finland Finland
Not recruiting
11.12.2024
Germany Germany
Not recruiting
11.12.2024

Trial locations

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.

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.

Trial ID:
2024-511981-36-00
Protocol code:
WVE-006-002
NCT ID:
NCT06405633
Trial Phase:
Human Pharmacology (Phase I) – Other

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