Active not recruitingPHASE2INTERVENTIONAL

Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema

This study is looking at a new treatment called SAR447537 (also known as INBRX-101) for adults who have lung damage, known as emphysema, because of a genetic condition called Alpha-1 Antitrypsin Deficiency (AATD). It's an extension of an earlier study, meaning it will continue to follow participants to see how the treatment works over a longer period. The main goal is to understand if the treatment is safe for long-term use and if it can help improve or manage the symptoms of AATD emphysema. This type of study is important for developing new ways to help people with this condition. Participants will be carefully monitored throughout the study.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Sanofi

Enrolment target

185

Start

20 Jun 2024

Estimated completion

30 Sep 2028

Alpha 1-Antitrypsin Deficiency Emphysema

What is this study about?

This research is a study looking into a new medicine called SAR447537 (you might also hear it called INBRX-101). It’s designed for adults who have emphysema, a type of lung damage, because of a problem with a protein in their body called Alpha-1 Antitrypsin. This condition is also known as Alpha-1 Antitrypsin Deficiency, or AATD for short.

The main aim of this study is to see how safe this new medicine is when taken over a longer period. Researchers also want to find out if it can help improve or at least manage the symptoms of emphysema in people with AATD. This is an 'open-label' study, which means both the participants and the study team will know that the active medicine is being given.

Studies like this are really important because they help doctors and scientists understand if new treatments can make a real difference to people's health and quality of life. By carefully studying this medicine, researchers hope to find better ways to care for people living with AATD emphysema.

Key takeaways

Tests a new medicine (SAR447537) for AATD-related emphysema.
Aims to check long-term safety and effectiveness.
Designed for adults aged 18-80 with diagnosed AATD.
Requires participants to be non-smokers with specific lung function.
Participation involves regular clinic visits and health monitoring.

Who may be eligible?

This study is looking for both men and women who are between 18 and 80 years old. To be considered, you must have been officially diagnosed with Alpha-1 Antitrypsin Deficiency (AATD) and already show signs of emphysema, which is lung damage caused by AATD. Your lung function also needs to meet certain levels, and it's essential that you are currently a non-smoker.

There are also some reasons why you might not be able to join. For example, if you've recently received certain other treatments for AATD or other conditions, or if you have severe allergies to components of the study drug. You also couldn't join if you have uncontrolled diabetes, certain active cancers, specific lung infections, or are waiting for a lung or liver transplant.

This list isn't everything, and a study doctor would review your full health history to make sure the study is a safe and suitable option for you.

Quick self-check

Are you between 18 and 80 years old?
Do you have an official diagnosis of Alpha-1 Antitrypsin Deficiency (AATD)?
Have you been told you have emphysema caused by AATD?
Are you currently a non-smoker?
Are you generally in good health, apart from your AATD?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you'll be taking the study medicine, SAR447537, for an extended period. Because this is an 'open-label' study, both you and the study team will know you are receiving the active treatment. You will have regular visits to the clinic where the study team will do various check-ups, including physical exams, blood tests, and lung function tests, to monitor your health and how you're responding to the treatment. They will also ask you questions about how you are feeling and any side effects you might be experiencing. The exact duration of your participation and the number of visits would be explained in full detail by the study team.

Potential risks and benefits

Taking part in a study like this might offer potential benefits, such as gaining access to a new treatment for your condition before it's widely available, and receiving regular, close medical care and monitoring. However, there are also potential risks; the medicine might cause side effects, or it might not work as expected for you. There's also the time commitment involved with clinic visits. It’s very important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (40)

UAB Lung Health Center- Site Number : 105
Birmingham, United States
St Joseph's Hospital and Medical Center- Site Number : 126
Phoenix, United States
David Geffen School of Medicine at UCLA- Site Number : 124
Los Angeles, United States
University of California Davis Medical Center- Site Number : 110
Sacramento, United States
National Jewish Medical and Research Center- Site Number : 123
Denver, United States
Nuvance Health Medical Practices, Pulmonary & Sleep Specialists- Site Number : 119
Danbury, United States
University of Florida - Gainesville - 1600 SW Archer Rd- Site Number : 101
Gainesville, United States
Indiana University- Site Number : 127
Indianapolis, United States
Brigham and Women's Hospital -75 Francis Street- Site Number : 131
Boston, United States
University of Minnesota-420 Delaware Str SE- Site Number : 125
Minneapolis, United States
Hannibal Regional Healthcare System-HRMG-Hannibal- Site Number : 111
Hannibal, United States
Columbia University Irving Medical Center- Site Number : 104
New York, United States

+28 more sites — see the official record for the full list.

Common questions

What is SAR447537?

SAR447537 is an investigational new medicine being tested for people with lung damage (emphysema) due to Alpha-1 Antitrypsin Deficiency (AATD).

What is Alpha-1 Antitrypsin Deficiency (AATD)?

AATD is a genetic condition where your body doesn't produce enough of a protein that protects your lungs, leading to damage like emphysema.

What does 'open-label' mean?

It means that both you (if you join the study) and the research team will know that you are receiving the active study medicine, not a dummy treatment (placebo).

What 'phase' is this study?

This is a Phase 2 study. This means researchers are looking more closely at the medicine's safety and how well it works in a larger group of people, after earlier safety tests.

Can I join if I currently smoke?

No, to be eligible for this study, you must be a non-smoker.

How to find out more

Official trial record

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