NCT03679598

Who might be able to join this trial:

• You are between 18 and 80 years old
• You have a confirmed diagnosis of Alpha-1 Antitrypsin Deficiency (AATD) with a specific genetic type known to cause low or poorly functioning AAT protein (such as Pi*ZZ, Pi*SZ, Pi*null, or certain rare types including "F" or "I" mutations)
• Your lung function test (a breathing test called FEV1) shows your lungs are working at 25% or more of what is expected for someone your age and size
• You are either not currently receiving AAT protein infusion therapy (and have not received it in the last 12 weeks), OR you have been receiving weekly AAT infusions at the standard dose for at least 12 weeks and plan to continue throughout the study
• You are able and willing to give your signed agreement to take part, following all the study rules
• If you are male, you agree to use highly effective birth control during the treatment period and for at least 4 days after your last dose, and you agree not to donate sperm during this time
• If you are female, you are not pregnant, not breastfeeding, and either cannot become pregnant or agree to use reliable birth control during treatment and for at least 4 days after your last dose

Who may not be able to join:

• You have a type of AATD (such as Pi*MZ, Pi*MS, Pi*SS, or Pi*FM) that is not known on its own to cause the lung condition emphysema
• You have been diagnosed with a lung condition other than COPD, such as cystic fibrosis, widespread scarring of the lung tissue, or a significant airway condition called bronchiectasis
• You have had a flare-up of your lung condition requiring steroid tablets or antibiotics within the last 4 weeks
• You have hepatitis B, hepatitis C, or any other active or long-term liver infection
• You have HIV or another condition that weakens your immune system, or your white blood cell count is too low
• Your liver blood test results are significantly above the normal range (confirm with trial site)
• You have abnormal results for certain blood tests, including low platelet count, low protein (albumin) levels, abnormal blood clotting test (INR), or elevated muscle enzyme levels (CPK) (confirm with trial site)
• You have a history of, or currently have, serious liver problems such as cirrhosis, varicose veins in the food pipe (esophageal varices), fluid in the abdomen (ascites), or confusion caused by liver disease
• You have another type of long-term liver disease, such as autoimmune liver disease, Wilson's disease, or iron overload conditions
• You have been diagnosed with fatty liver disease (NAFLD) on any scan or imaging, or have taken medications linked to fatty liver for more than 2 weeks in the year before the study
• You have a history of heavy alcohol use (more than an average of 20g per day for women or 30g per day for men) for more than 3 months in a row within the past year
• Your liver scarring score (called FIB-4) is above 3.25 (confirm with trial site)
• You have had a heart attack, unstable chest pain, heart bypass surgery, stroke, or mini-stroke within the last 6 months, or you have uncontrolled high blood pressure
• You have significant heart failure with poor heart pumping function
• You have an abnormal heart rhythm reading (ECG) at the start of the study, or a history of a serious heart rhythm condition
• You have been diagnosed with cancer in the last 5 years (except for certain treated skin cancers)
• You take high-dose steroids daily (more than 10mg of prednisone or equivalent), other immune-suppressing medicines, or certain anti-inflammatory drugs daily
• You have received immune-modifying biological medicines (such as certain injected or infused treatments) within the last 6 months
• You have started taking certain medicines known to affect the liver within the last 28 days, including some cholesterol medications, anti-inflammatory drugs, or certain breathing or epilepsy medicines (those already on these medicines for more than 28 days before the study may not be excluded — confirm with trial site)
• You are taking blood