Alpha-1 Study: A research study for individuals with Alpha-1 Antitrypsin Deficiency

Updated on 23 November 2020
alpha1-proteinase inhibitor (human)
alpha 1-antitrypsin


The Alpha-1 Study is a clinical research study currently enrolling individuals 18 to 78 (inclusive) with Alpha-1 Antitrypsin Deficiency.  

The purpose of this study is to evaluate the safety, tolerability, and effectiveness of an investigational medication in individuals with Alpha-1 Antitrypsin Deficiency.

Interested individuals will be evaluated during a screening period to determine their eligibility to participate. Your 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.


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Condition Alpha 1 Antitrypsin Deficiency
Clinical Study IdentifierTX232161
Last Modified on23 November 2020


Yes No Not Sure

Inclusion 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
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How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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