RecruitingPHASE1, PHASE2INTERVENTIONAL

A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)

This research study is testing a new medicine called TSRA-196 for adults who have a genetic condition called Alpha-1 Antitrypsin Deficiency (AATD), specifically the PiZZ type. This condition can affect the lungs and liver. The main goals of this study are to find out if TSRA-196 is safe, how well people can take it, and if it shows any signs of helping with the lung or liver issues caused by AATD. Researchers will also look at how the body handles the medicine. This is an early stage of testing, meaning it's one of the first times this medicine is being given to people to understand its effects and find the right dose. It's an open-label study, so both patients and doctors will know who is receiving the new medicine.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Tessera Therapeutics, Inc.

Enrolment target

Start

21 Apr 2026

Estimated completion

01 Mar 2029

Alpha-1 Antitrypsin Deficiency (AATD)

What is this study about?

Alpha-1 Antitrypsin Deficiency, or AATD, is a genetic condition that runs in families. It means your body doesn't make enough of a special protein called alpha-1 antitrypsin, which normally protects your lungs and liver from damage. When this protein is missing or doesn't work properly, it can lead to serious lung diseases like emphysema, and liver problems.

This study is testing a new medicine called TSRA-196. The aim is to see if this medicine can help adults who have a specific type of AATD, known as the PiZZ type. This particular type is often linked to more severe lung and liver problems. The researchers want to find out if TSRA-196 is safe to use and if it can help improve or manage the symptoms of AATD.

The study is divided into different parts. In the first part, a small group of people will be given increasing doses of the medicine to see what dose is safe and well-tolerated. Later parts will involve more people to further check safety and to see if the medicine has any helpful effects on the lungs and liver.

Key takeaways

This study is testing a new medicine called TSRA-196 for AATD.
It's for adults with the specific 'PiZZ' type of AATD, affecting lungs or liver.
The main goals are to check safety, how well people tolerate it, and early signs of its effectiveness.
Participation involves regular clinic visits and health checks.
It's an early-stage study, so the medicine isn't widely available yet.
You can stop participating at any time.

Who may be eligible?

To join this study, you need to be an adult between 18 and 70 years old. You must have a confirmed diagnosis of Alpha-1 Antitrypsin Deficiency (AATD) with the specific PiZZ gene type. Your body weight should also be within a certain range.

You cannot be a smoker, and if you used to smoke, you need to have stopped for at least six months before starting the study. If you've been on other treatments for AATD, you might need to stop those for a certain time before joining. The study is looking for people with lung disease, liver disease, or both, caused by AATD, but there are specific measures of how severe these conditions can be to qualify.

There are also some reasons why you might not be able to join. For example, if you have a different type of liver disease not related to AATD, or if your lung disease is caused by something else. Also, if you've been hospitalised often for lung problems in the past year, or if you have a specific genetic change that might stop the new medicine from working, you wouldn't be able to take part.

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.

Are you between 18 and 70 years old?
Do you have a confirmed diagnosis of PiZZ Alpha-1 Antitrypsin Deficiency?
Are you a non-smoker, and have you been for at least 6 months?
Do you have lung or liver problems caused by AATD (within specific limits)?
Have you not had many hospital visits for lung problems in the last year?
Do you have no other serious liver problems not linked to AATD?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive the new study medicine, TSRA-196. This is an 'open-label' study, which means both you and the study team will know you are receiving the active medicine. The study will involve regular visits to a clinic for health checks, blood tests, and other assessments to monitor your health and how your body reacts to the medicine.

These assessments might include tests to check your lung function or liver health, depending on which part of the study you join and what your main health issues are. The study team will explain exactly what tests are needed for you.

Your participation will involve multiple clinic visits over a period of time, including follow-up appointments after you've finished taking the medicine. The total duration of your involvement will depend on which part of the study you participate in and will be fully explained before you agree to join. You will be closely monitored by a team of doctors and nurses throughout the study.

Potential risks and benefits

Taking part in any clinical trial has potential benefits and risks. You might benefit from receiving a new medicine that is not yet widely available, and it could potentially help with your AATD symptoms. You'd also be helping researchers learn more about AATD and how to treat it, which could benefit others in the future. However, there's no guarantee the medicine will help you, and it might not work as expected. Like all medicines, TSRA-196 could cause side effects, which might be mild or more serious. The study team will closely monitor you for any side effects and will explain all known potential risks before you decide to join. Remember, taking part is completely voluntary, and you are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (7)

Clinical Study Center
Verified postcode
Boston, United States· Recruiting
Clinical Study Center
Verified postcode
Rochester, United States· Recruiting
Clinical Study Center
Verified postcode
Charleston, United States· Recruiting
Clinical Study Center
Verified postcode
Seattle, United States· Recruiting
Clinical Study Center
Verified postcode
Fitzroy, Australia· Recruiting
Clinical Study Center
Verified postcode
London, United Kingdom· Recruiting
Clinical Study Center
Verified postcode
Nottingham, United Kingdom· Recruiting

Common questions

What is Alpha-1 Antitrypsin Deficiency (AATD)?

It's a genetic condition where your body doesn't make enough of a protein that protects your lungs and liver, leading to damage.

What is the new medicine, TSRA-196?

TSRA-196 is a new medicine being tested to see if it can help people with AATD, specifically the PiZZ type, by addressing the underlying cause of the condition.

Is this medicine available to everyone?

No, TSRA-196 is an investigational medicine, meaning it's still being tested in clinical trials and is not yet available as a regular treatment.

What does 'Phase 1/2' mean?

This means it's an early-stage study primarily focused on checking the medicine's safety, how well people tolerate it, and finding the right dose, but also looking for early signs of how well it works.

Do I have to stop my current AATD treatment to join?

Possibly. If you're on other AATD treatments, you might need to stop them for a certain period before you can start the study medicine. The study team will advise you on this.

How to find out more

Tessera Clinical Trials Information

clinicalinfo@tesseratx.com 857-271-4800 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.