The Longitudinal Evaluation of Autoimmune Pulmonary Alveolar Proteinosis (NCT03531996) | Clinical Trial Compass
CompletedNot Applicable
The Longitudinal Evaluation of Autoimmune Pulmonary Alveolar Proteinosis
United States29 participantsStarted 2018-04-19
Plain-language summary
The major goal of this study is to conduct a prospective, longitudinal study of autoimmune PAP to examine outcome measures for disease severity of potential use in clinical practice and/or clinical research studies. These results will impact the field by: 1) improving an understanding of the clinical course of autoimmune PAP, 2) providing information on various clinical outcome and quality of life outcome measures to guide patients and physicians in making treatment choices, and 3) facilitate the development of pharmaco-therapeutics for autoimmune PAP and 4) better informing PAP researchers.
Who can participate
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:
* Written informed consent must be provided by:
* Participant if at least 18 years old -OR-
* Parent/legal guardian if participant is less than 18 years old -AND-
* Participant provides assent when appropriate
* History of diagnosis of autoimmune as indicated by a:
* History of chest CT or x-ray findings compatible with PAP -AND-
* History of a Positive (Abnormal) serum GMAb test
Exclusion Criteria:
* Individuals who have a serious medical illness that, in the opinion of the investigator, is likely to interfere with completion of the study will be excluded.
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
Longitudinal evaluation of GM-CSF autoantibody levels
Timeframe: Baseline, 1 year, and 2 years
2
Longitudinal evaluation of the maximal phospho-STAT5 level after GM-CSF stimulation
Timeframe: Baseline, 1 year, and 2 years
3
Longitudinal evaluation of the STAT5 Phosphorylation Index
Timeframe: Baseline, 1 year, and 2 years
4
Longitudinal evaluation of the GM-CSF Signaling Index
Timeframe: Baseline, 1 year, and 2 years
5
Longitudinal evaluation of the dose GM-CSF to stimulation 1/2 maximal STAT5 phosphorylation (EC50)
Timeframe: Baseline, 1 year, and 2 years
Trial details
NCT IDNCT03531996
SponsorChildren's Hospital Medical Center, Cincinnati