The RepAIR1 study is researching a potential therapy for people with alpha-1 antitrypsin deficiency (AATD). The study will determine if the study therapy is safe and tolerable in people living with alpha-1 antitrypsin deficiency (AATD).
Study participants must meet the following criteria*:
18 to 75 years of age
Have alpha-1 antitrypsin deficiency (AATD) due to PiZZ genotype
(note: genotype is confirmed during the screening process)
*Other study requirements will apply.
Participation in the RepAIR1 Study lasts about 13 months. Study participants can expect the following:
Participation in a clinical study is voluntary. You can ask any questions you have and may leave the study at any time, for any reason.
The investigational study therapy, called AIR-001, is designed to correct the mutation responsible for AATD at the RNA level. This correction by the study drug could help restore levels of AAT protein in the blood, hence treating the deficiency. The correction could also help to produce normal and functional AAT protein which protects the lungs and liver. This is not a permanent process as the study therapy does not target the DNA.
Yes, every person that qualifies for the study and chooses to participate will receive the study therapy (AIR-001). The study team will provide more information on frequency of study visits.
Investigational means the study therapy is not approved by regulatory authorities like the European Medicines Agency (EMA) or U.S. Food and Drug Administration (FDA) and it can only be used in clinical research studies like RepAIR1.
DNA is a set of instructions, which are like the blueprint to how the body operates. RNA is the process that translates the DNA instructions and makes the proteins that the body needs to function. RNA editing can change how a specific RNA transcript is translated into a protein so that the normal form of the protein is made. Unlike DNA editing, RNA editing is a reversible process that does not change the blueprint of genetic code forever.
Alpha-1 antitrypsin deficiency (AATD) is an inherited condition caused by a genetic mutation in the SERPINA1 gene. AATD causes low levels and misfolding of a protein called alpha-1 antitrypsin (AAT). The misfolding results in a non-working version of AAT. This abnormal protein becomes trapped in the liver and does not reach the lungs, which can gradually lead to liver and lung damage.
Here are some common questions and answers about study participation.
Clinical research studies, or trials, help scientists and doctors explore whether a medical strategy, device, or medication is safe and effective for people. Before any medication can be approved and made available to the public, it must go through several phases of clinical research.
Before enrolling in a clinical trial, you must sign an Informed Consent Form (ICF). The ICF contains information about the study, including study goals, how long the study will last, benefits and risks, and the tests and procedures you will receive.
Study participation usually involves visiting a clinic regularly, taking or receiving an investigational medicine, and having assessments to monitor your health. You can still see your regular doctor, but you should let them know that you are participating in a study.Study participation usually involves visiting a clinic regularly, taking or receiving an investigational medicine, and having assessments to monitor your health. You can still see your regular doctor, but you should let them know that you are participating in a study.
Participation in clinical research studies is your choice, and you may stop at any time.
If you have additional questions about participating in a clinical trial, contact a study clinic near you.
Use the filters, map, and listing to find the study clinic closest to you.