A Phase 2, Double-blind, Randomized, Active-control, Parallel Group Study to Assess the Pharmacokinetics, Pharmacodynamics, Immunogenicity, and safety of SAR447537 (INBRX-101) Compared to Plasma Derived Apha1-Proteinase Inhibitor (A1PI) Augmentation Therapy in Adults with Alpha-1 Antitrypsin Deficiency (AATD) Emphysema
This research study is for adults with a lung condition called Alpha-1 Antitrypsin Deficiency (AATD) emphysema. We are testing a new medicine called INBRX-101 against the usual treatment, which is a plasma-derived A1PI augmentation therapy (like Respreeza). The main goal is to understand how INBRX-101 works in the body compared to the standard treatment. We'll be looking at things like how much of the medicine stays in the blood, how it affects the body, and if it's safe. We also want to see if it causes any unwanted side effects or if the body reacts to it in unexpected ways. This is a Phase 2 study, meaning it's an earlier stage to see if the new medicine is promising before larger studies are done.
At a glance
What is this study about?
This study is designed for adults who have a lung condition called Alpha-1 Antitrypsin Deficiency, often shortened to AATD. People with AATD can develop emphysema, which is a type of serious lung damage, because their bodies don't make enough of a protective protein called alpha-1 antitrypsin (AAT). This protein usually helps protect the lungs from harm caused by certain enzymes.
The purpose of this research is to compare a new experimental medicine, currently called SAR447537 (also known as INBRX-101), with the current standard treatment for AATD emphysema. The standard treatment involves giving patients a similar protective protein (A1PI) that comes from donated plasma. Researchers want to understand how this new medicine behaves in the body, such as how long it stays in the blood and how it affects the levels of the protective protein. They will also be very carefully checking for any side effects and how safe the new treatment is overall.
By doing this study, doctors hope to find out if INBRX-101 could be a new and effective way to help people with AATD emphysema. This is a 'Phase 2' study, which means it's an important step in developing new medicines. If the results are promising, it could lead to further, larger studies, and potentially a new treatment option in the future. Participants will receive either the new medicine, the standard treatment, or a saline solution (a placebo) to allow fair comparison.
Key takeaways
- New medicine (INBRX-101) compared to standard treatment for AATD emphysema.
- Study examines effectiveness, safety, and how medicines act in the body.
- Participation involves infusions and regular health checks.
- It's a 'double-blind' study for fair comparison of treatments.
- Aimed at adults 18 or older with AATD emphysema.
Who may be eligible?
This study is looking for adult volunteers of any gender who are 18 years old or older. You will need to have a diagnosis of Alpha-1 Antitrypsin Deficiency (AATD) emphysema to be considered for participation.
There are other specific health checks and criteria that the study doctors will need to review to make sure it's safe and appropriate for you to join. For example, they will look at your overall health, any other medical conditions you might have, and any other medicines you are taking.
These checks are important to protect your safety and to make sure the study results are clear. The research team will discuss all the detailed requirements with you if you are interested in taking part.
- Are you 18 years old or older?
- Do you have a diagnosis of Alpha-1 Antitrypsin Deficiency (AATD)?
- Have you been diagnosed with emphysema related to AATD?
- Are you able to attend regular clinic appointments?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, you will receive one of three treatments: the new medicine (INBRX-101), the standard A1PI treatment, or a saline solution. These treatments will be given as an infusion, meaning through a drip into your arm.
Throughout the study, you will have regular visits to the clinic. At these visits, the study team will carry out various assessments. This includes taking blood samples to measure how the medicine is working in your body, checking your general health, and carefully recording any side effects you might experience. These checks are crucial for understanding the medicine's effects and safety.
The full length of your participation in the study, including all treatments, follow-up appointments, and medical checks, would be explained in detail by the study team. They will also tell you how often you would need to visit the clinic and for how long each visit might be.
Potential risks and benefits
Locations (5)
- —Denmark
- —Sweden
- —Ireland
- —Spain
- —Poland
Common questions
What is Alpha-1 Antitrypsin Deficiency (AATD) Emphysema?
It's a genetic condition where your body doesn't produce enough of a protein that protects your lungs, leading to damage and breathing problems.
What is 'Phase 2' in a clinical trial?
Phase 2 means the study is in an earlier stage, checking if a new medicine is safe and if it works for the condition, after initial safety tests.
Will I know if I'm getting the new medicine or the standard one?
No, this is a 'double-blind' study, meaning neither you nor the study team will know which treatment you are receiving until after the study is complete, to keep results fair.
What are the main things the researchers are looking for?
They're mainly looking at how the treatments affect the levels of a protective protein in your blood and checking for any side effects.
What is a placebo?
In this study, 'Sodium Chloride 0.9%' (saltwater) acts as a placebo. It contains no active medicine and is used to compare with the actual treatments.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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