AATD Participant Engagement Study
Research type
Research Study
Full title
Engagement Study to Familiarise Participants with known or suspected Alpha-1 Antitrypsin Deficiency (AATD) with the Clinical Trials Unit and Establish a Baseline for Future Trial Participation
IRAS ID
361938
Contact name
Ulrike Lorch
Contact email
Sponsor organisation
AIRNA Corporation
Duration of Study in the UK
0 years, 6 months, 5 days
Research summary
This participant engagement study will provide participants with known or suspected diagnosis of AATD the opportunity to become familiar with the clinical trials environment and clinical team before considering if they want to take part in a future AATD-related RNA editing clinical trial. The study will also allow participants to undergo a series of assessments to establish a clinical baseline. In addition, this study will use targeted PiZZ genotype testing to confirm AATD in participants from populations with high prevalence of developing AATD, providing them with the opportunity to receive a diagnosis.
This study will take approximately 3 weeks for the participants to complete. The participants will attend an in-person visit to the study site on Day 1, during which they will be asked to provide their informed consent. Following the informed consent, they will receive a tour of the site and undergo a series of basic clinical assessments (such as vital signs, ECGs, blood samples, liver FibroScan and pulmonary function tests) to establish a clinical baseline. Participants may be provided with a hard copy of a Participant Information Sheet/Informed Consent Form (PIS/ICF) (provided this document has received approval from a REC) for upcoming RNA-editing AATD-related clinical studies. This will allow potential participants to take the information home for a thorough review and discussion with friends and family before potentially attending a screening appointment for an RNA-editing clinical trial.
A follow-up telephone call with the Study Doctor will be conducted within 3 weeks after Day 1 to discuss the results of the clinical assessments and allow participants to ask any questions.
REC name
London - City & East Research Ethics Committee
REC reference
25/PR/1378
Date of REC Opinion
17 Oct 2025
REC opinion
Further Information Favourable Opinion