TSRA196-AAT-201: A Phase 1/2, Open-Label, Multi-Center, Dose Escalation, Dose Expansion, and Single Repeat Dose Study of TSRA-196 in Adults With the PiZZ Genotype Who Have Lung and/or Liver Disease Associated with Severe Alpha-1 Antitrypsin Deficiency
This research study is looking at a new treatment called TSRA-196 for adults who have a specific genetic type of Alpha-1 Antitrypsin Deficiency (AATD), known as PiZZ. This condition can lead to problems with the lungs and/or liver. The study is in its early stages (Phase 1/2), which means it's one of the first times this medicine is being given to people. Researchers want to find out if TSRA-196 is safe, what dose works best, and how it affects the levels of a protective protein called alpha-1 antitrypsin in the blood. They will also look at whether one or two doses make a difference.
At a glance
What is this study about?
This study is about a new medicine called TSRA-196 for people with a condition called Alpha-1 Antitrypsin Deficiency, or AATD. This is a genetic condition, meaning it's passed down in families, and it can cause serious problems, especially in the lungs and liver. People with AATD don't produce enough of a special protective protein called alpha-1 antitrypsin. Without enough of this protein, these organs can get damaged over time.
The main goal of this study is to test TSRA-196 in adults who have AATD, specifically those with the 'PiZZ' genetic type. This is an earlier-stage study, which is split into two parts (Phase 1 and Phase 2). In these early phases, doctors are mainly focused on checking if the new medicine is safe for people to take. They'll also be trying to find the best dose and understand how the medicine behaves in the body.
Researchers will carefully monitor participants to see if the medicine causes any side effects. They will also measure the levels of the alpha-1 antitrypsin protein in their blood to see if TSRA-196 helps to increase these levels. They'll look at the effects over several months, including what happens after a single dose and after a second dose. All this information will help them decide if TSRA-196 could be a promising new treatment for AATD.
Key takeaways
- This study is for adults with a specific type of Alpha-1 Antitrypsin Deficiency (PiZZ).
- It's testing a new medicine, TSRA-196, for safety and how it affects protein levels.
- The study includes people with AATD-related lung and/or liver disease.
- Participation involves regular clinic visits for blood tests and health checks.
- This is an early-stage study, so potential benefits and risks are still being assessed.
- You can withdraw from the study at any time if you choose to.
Who may be eligible?
To be eligible for this study, you must be an adult, which means you need to be 18 years old or older. There is no upper age limit for taking part.
You must also have been diagnosed with Alpha-1 Antitrypsin Deficiency (AATD) and have the specific 'PiZZ' genetic type. This is very important as the study is designed for people with this particular type of AATD. You should also have either lung problems, liver problems, or both, that are linked to your AATD diagnosis.
Your study doctor will discuss all the specific criteria with you to make sure this study is the right fit. There will be other health checks and medical requirements to ensure your safety and that the study results are clear.
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 18 years old or older?
- Have you been diagnosed with Alpha-1 Antitrypsin Deficiency?
- Do you know if you have the 'PiZZ' genetic type of AATD?
- Do you have lung problems, liver problems, or both, related to your AATD?
What does participation involve?
If you decide to take part in this study, you would receive the study medicine, TSRA-196. Since this is an early-stage study, it involves regular visits to the study clinic for assessments. These will include blood tests to check your alpha-1 antitrypsin levels and to monitor for any side effects. You'll also have other medical checks like physical exams, vital signs (like blood pressure and heart rate), and possibly ECGs (heart rhythm tests).
The study will involve follow-up over several months, sometimes up to a year, to understand the long-term effects of the medicine. The exact number of visits, tests, and the total length of time you'd be involved will be explained in detail by the study team, as it can vary depending on which part of the study you are in.
Potential risks and benefits
Locations (1)
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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 and liver from damage.
What does 'Phase 1/2' mean?
It means this is one of the first times the new medicine is being tested in people. Researchers are checking its safety, side effects, and how it works in the body.
What is TSRA-196?
TSRA-196 is the new medicine being tested in this study for AATD. We don't know much about it yet, as it's still in early research stages.
Will I get better if I join this study?
This is a research study, so we can't guarantee you'll get better. Researchers are trying to find out if the medicine works and is safe. Your health will be closely monitored.
Can I leave the study if I change my mind?
Yes, you can leave the study at any time you wish, for any reason. Your decision will not affect your future medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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