Development of a Prediction Score for the Occurrence of Death or Lung Transplantation in Patients… (NCT07715617) | Clinical Trial Compass
RecruitingNot Applicable
Development of a Prediction Score for the Occurrence of Death or Lung Transplantation in Patients With Emphysema Secondary to Alpha-1-anti-tripsin Deficiency
France230 participantsStarted 2026-02-16
Plain-language summary
Emphysema linked to alpha-1-antitrypsin deficiency (DAAT): towards a better prediction of risks Emphysema caused by alpha-1-antitrypsin deficiency (DAAT) is a rare genetic disorder that can lead to serious complications, such as the need for a lung transplant or death, affecting up to 15% of patients. The only specific treatment available is a weekly infusion of alpha-1-antitrypsin (IV-AAT), an expensive and burdensome therapy.
Currently, there is no reliable model to predict the course of the disease in these patients. Our study, conducted in several French hospitals, aims to develop a prediction tool combining clinical, biological, functional data and advanced medical image analysis (lung CT). This model will make it possible to identify the most at-risk patients, in order to better adapt their care, anticipate transplant needs and avoid unnecessary treatments for low-risk patients.
Ultimately, this approach could also improve access to care for patients who need it most, while optimizing health system resources.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
1. Inclusion criteria:
* diagnosed between 2010 and 2025
* in the pulmonology department
* diagnosis of emphysema and COPD secondary to alpha-1 antitrypsin deficiency ZZ, Znull, ZMalton, Z and rare mutations,
* emphysema according to the initial thoracic CT scan (+/- 12 months after diagnosis).
2. Exclusion criteria:
* Age \<18 years
* Patient opposed to the use of their data for research purposes
* Patient deprived of liberty by judicial decision
* Patient not affiliated with a social security scheme
* no CT scan available
* no lung function test available the year around CT scan
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial is focused on building a prediction score using AI and machine learning — does that mean my data and test results would be used to train a model, rather than me receiving any new treatment, and is that something worth participating in given my current situation with alpha-1-antitrypsin deficiency?
2Since the trial is tracking death and lung transplantation as its main outcomes, what would the process look like for me practically — would I just be providing data, answering follow-up questions, or undergoing additional tests over time?
3Given that this is a predictive modeling study rather than a treatment trial, could the AI-generated risk score eventually help guide decisions about when I might need to be evaluated for a lung transplant, and would my doctor have access to that information?
4Are there other treatment-focused trials or standard therapies for emphysema related to alpha-1-antitrypsin deficiency that I should consider alongside or before joining an observational study like this one?
5Because this study is specifically designed for patients with emphysema caused by alpha-1-antitrypsin deficiency, how would my doctor determine whether my diagnosis and disease stage make my data a good fit for what the researchers are trying to measure?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.