Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humansInterventional

Phase 1/2 Multicenter, Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-3001 in Participants with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease

This research study is testing a new experimental treatment called NTLA-3001 for people who have a genetic lung condition called alpha-1 antitrypsin deficiency (AATD), which often leads to emphysema. It's a very early-stage study, meaning it's the first time this treatment is being given to people. The main aim is to see if the treatment is safe and to understand any side effects. Researchers will also look at how the treatment is processed by the body and if it helps increase a protective protein in the blood called alpha-1 antitrypsin (AAT). This new treatment uses gene therapy techniques, and the study will gather important information to help develop better treatments for AATD in the future.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Intellia Therapeutics Inc.

Enrolment target

Start

18 Nov 2024

Pulmonary emphysema

What is this study about?

This study is about a new and experimental treatment for a genetic lung condition called alpha-1 antitrypsin deficiency, often shortened to AATD. People with AATD don't produce enough of a special protein called alpha-1 antitrypsin (AAT), which protects the lungs from damage. Without enough of this protein, their lungs can become damaged over time, leading to a condition called emphysema, which makes it hard to breathe.

The experimental treatment, called NTLA-3001, is a type of gene therapy. This means it aims to help the body make more of the missing AAT protein itself. Because this is a very early study, called a Phase 1/2 trial, it's the first time this particular treatment is being given to people. This means researchers are taking extra care to make sure it's safe.

The main goal of this study is to carefully check for any side effects or problems that might happen while taking the treatment – this is known as looking at its safety. They also want to understand how the body processes the treatment and how long it stays in the system. Later on, they will also measure if the treatment helps to increase the levels of the protective AAT protein in the blood, and how this might affect lung health and quality of life for people with AATD.

Key takeaways

This is an early study of a new treatment for AATD-related lung disease.
The main focus is on the safety of the new treatment.
Researchers will also look at how the body handles the treatment and its effects.
All participants will receive the active study treatment.
You must be 18 or older to be considered for this study.

Who may be eligible?

To be considered for this study, you would need to be an adult, aged 18 or older. The study is open to both men and women.

More specific details about your health and medical history, such as the exact type of AATD you have, your current lung function, and any other medical conditions, would be reviewed by the study doctors to see if this study is right for you. This is to make sure the study treatment is as safe as possible for everyone involved.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Am I 18 years old or older?
Have I been diagnosed with alpha-1 antitrypsin deficiency (AATD)?
Do I have lung disease (emphysema) related to my AATD?
Am I able to attend regular clinic visits for monitoring?
Am I comfortable with receiving an experimental treatment?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive the experimental treatment. Because it's an early-stage study, you would have regular visits to the clinic so the study team can carefully monitor your health and how you respond to the treatment. These visits would likely involve blood tests, urine tests, and possibly other tests to check your lung function and general health. You would also be asked to complete questionnaires about your breathing and how you feel, to see if the treatment is affecting your quality of life. The study would involve a number of follow-up appointments over a period that would be explained to you in detail.

Potential risks and benefits

Taking part in this study may offer the potential benefit of access to a new, experimental treatment for your lung condition that is not yet available. However, because this is an early-stage study, there are potential risks, and side effects from the new treatment are possible and could be serious. The main purpose of this study is to identify these potential risks and side effects. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Ireland

Common questions

What is alpha-1 antitrypsin deficiency (AATD)?

It's a genetic condition where your body doesn't make enough of a special protein that protects your lungs, which can lead to breathing problems.

What is 'Phase 1/2' in a study?

This means it's an early study, primarily focused on making sure the new treatment is safe for people to use, and also looking at how it works in the body.

Will I know if I'm getting the treatment or a dummy medicine?

This study is 'open-label,' which means everyone involved, including you and the study team, will know you are receiving the active treatment.

What are the main things researchers want to find out?

They mainly want to check if the treatment is safe and what side effects it might have. They also want to see how your body reacts to it.

Will this study cure my condition?

This is an early study, so it's too soon to say if it will lead to a cure. The goal is to gather information to develop better treatments in the future.