The purpose of this study is to evaluate the safety, tolerability, and effectiveness of an investigational medication in individuals with Alpha-1 Antitrypsin Deficiency. The word “investigational” means this medication is not yet approved for use by the Food and Drug Administration (FDA) in the United States or other regulatory agencies in Canada, Europe, or elsewhere.
Who May Qualify
Eligible participants must meet the following criteria:
- Male or female between the ages of 18 and 78 (inclusive)
- Female participants must be of non-childbearing potential (i.e., postmenopausal or have a history of hysterectomy or bilateral removal of the ovaries)
- Diagnosed with Alpha-1 Antitrypsin Deficiency
- Have the genetic pattern most commonly associated with Alpha-1 (the PiZZ genotype)
- A genetic blood test will be used to confirm genotype
- Have never been treated with gene therapy
- Do not smoke cigarettes and/or use electronic nicotine delivery systems (i.e., electronic cigarettes, vape pens) or chewing tobacco products
- Willing and able to comply with the study instructions
There are additional eligibility requirements, which the study doctor can explain to you.
For Families and Caregivers
If your loved one is living with Alpha-1, share this website with them today, or talk to his or her doctor for more information.
Interested individuals will be evaluated during a screening period to determine their eligibility to participate. Your study participation could last approximately 11–16 weeks depending on whether or not you have ever been on augmentation therapy.
Those who qualify will receive all study-related medications and procedures at no cost. You and your study doctor may choose to have some of the study visits at your home with a home health nurse, allowing for a reduced number of times that you must travel to the study site. You may also be compensated financially for your study-related time and travel.
Join a Strong Alpha-1 Community to Research a New Investigational Medication
As you know, Alpha-1 Antitrypsin Deficiency is a rare genetic disorder characterized by low circulating levels of Alpha-1 Antitrypsin. This deficiency puts people at an increased risk for lung disease and liver disease.
But you are not alone, and there is certainly strength in numbers, which is why we’re joining together to research this unique condition – and you may be able to take part.
See If You May Qualify