A National Registry For Pulmonary Alveolar Proteinosis (NCT02461615) | Clinical Trial Compass
RecruitingNot Applicable
A National Registry For Pulmonary Alveolar Proteinosis
United States500 participantsStarted 2015-04
Plain-language summary
The major goal of Part A of this study is to establish a National PAP Registry to help make reliable new research tests available to doctors to improve the diagnosis of PAP, increase awareness and knowledge of PAP, and give patients a 'seat at the table' in planning and conducting PAP research including the clinical testing of several new potential therapies. The major goal of Part B of this study is to define the natural history of autoimmune PAP (aPAP), develop a disease severity score that reflects how aPAP patients feel and function, and to develop and test novel tools to measure the severity of aPAP lung disease. Funding Source - FDA OOPD
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 for Part A and Part B:
* Written informed consent and assent, if applicable
Inclusion Criteria for Part A (Cross Sectional Study of PAP Syndrome)
* History of chest computed tomogram or chest radiograph findings compatible with PAP
* History of diagnosis of PAP made by at least one of the following methods:
* Positive (Abnormal) serum GMAb test -OR-
* Lung biopsy clearly documenting the presence of PAP of any type or degree -OR-
* Bronchoalveolar lavage cytology compatible with PAP -OR-
* Recessive or compound mutations in genes known to cause PAP, i.e. GM-CSF receptor α or β chain, GM-CSF, surfactant protein B or C or ABCA3, ABCG1, ABCA1, TTF1
Inclusion Criteria For Part B (Longitudinal \& PRO Survey Study of autoimmune PAP Patients)
* Diagnosis of autoimmune PAP as indicated by:
* Positive (Abnormal) Serum GMAb Test -AND-
* History of chest CT or x-rays findings compatible with PAP -OR-
* Lung biopsy clearly documenting the presence of PAP of any type or degree -OR-
* Bronchoalveolar lavage cytology compatible with PAP
Exclusion Criteria or Part A and Part B:
* 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.
For Part A (Cross-sectional Study of PAP Syndrome)
* Individuals that do not have a diagnosis of PAP
For Part B (Longitudinal \& PRO Survey Study of autoimmune PAP Patients)
* Individuals that do not have a diagno…
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
DBS card GM-CSF autoantibody levels to diagnose Autoimmune PAP
Timeframe: 5 years
Trial details
NCT IDNCT02461615
SponsorChildren's Hospital Medical Center, Cincinnati