Biomarkers Of Lung InVolvement In ASMD (NCT05914727) | Clinical Trial Compass
UnknownNot Applicable
Biomarkers Of Lung InVolvement In ASMD
Netherlands68 participantsStarted 2022-12-05
Plain-language summary
The goal of this study is to characterize ASMD patients' breath profile, exhaled breath and condensate as compared to healthy controls to identify specific volatile and non-volatile compounds that reflect inflammatory processes, fibrotic processes or sphingolipid accumulation in the lungs.
Participants will be provided exhaled breath samples in three ways (breath profile, volatile compounds and condensate). ASMD patients will be compared to age- and sex-matched healthy controls.
Who can participate
Age range
12 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.
Inclusion Criteria:
Patients:
* The patient has a biochemical and genetically confirmed diagnosis of the chronic visceral subtype of ASMD
* The patient is willing and able to provide written informed consent prior to the study-related procedure.
* The patient is ≥ 12 years of age
Healthy controls:
* The individual is willing and able to provide written informed consent prior to the study-related procedure
* The individual is ≥ 16 years of age
* General good health as determined by medical history
Exclusion Criteria:
Patients:
* Inability to adhere to the study protocol
* When a patient is not able to complete a spirometry test, the eNose sample will not be collected.
Healthy controls:
* Medical history of (systemic) disease for which medication was necessary
* Inability to adhere to the study protocol
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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.
What they're measuring
1
Markers of inflammation, fibrosis or sphingolipid accumulation in exhaled breath