A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)

This study is looking for adults aged between 18 and 70 (inclusive) who have been diagnosed with Alpha-1 Antitrypsin Deficiency (AATD). To be eligible, your AATD must be caused by a specific genetic change, known as the PiZZ mutation, which will be confirmed by a genetic test. Also, your blood levels of the alpha-1 antitrypsin protein must be below a certain level.

There are also some specific health requirements related to your lungs and liver. For example, your lung function (measured by tests called FEV1 and FVC) needs to be within a certain range, and you should show signs of emphysema or have a specific result from another lung test (DLCO). If you are currently receiving augmentation therapy for AATD, you might need to stop this treatment for at least six weeks before joining the study and continue to stop it throughout the study, unless your doctor says otherwise.

However, some health conditions would prevent you from taking part. For instance, if you have severe liver scarring (cirrhosis) or are on a waiting list for a lung or liver transplant, or have previously had certain surgeries like lung volume reduction. People with very severe chest infections often, or a Body Mass Index (BMI) over 30, would also not be able to join. There are also specific rules about liver health, including certain blood test results that might make you ineligible.

If you decide to take part in this study, you would first go through a screening process to check if you are eligible based on the criteria mentioned. If eligible, you would receive the study medicine, BEAM-302. This study has two parts. The first part will test different doses of the medicine in small groups, and the second part will use a specific dose in more people.

Throughout the study, you would have regular visits to the clinic. These visits will involve various tests and checks, including blood tests, lung function tests, and scans, to monitor your health, look for any side effects, and see how the medicine is affecting your AATD. The doctors will also want to understand how your body handles the medicine.

It's important to remember that BEAM-302 is an investigational medicine, meaning it's still being tested and is not yet approved for general use. The study duration will vary depending on the phase you are in and your individual needs, but it will involve multiple visits and ongoing assessments over a period of time.