🔎 Trials Near Me

Home › NCT02461615

A National Registry For Pulmonary Alveolar Proteinosis

RecruitingObservational study

Who can join

All ages · All sexes

Full eligibility criteria
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 diagnosis of autoimmune PAP

About the study

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

Sponsor: Children's Hospital Medical Center, Cincinnati · Participants: 500 · Started: 2015-04

Contact the study team

Official record on ClinicalTrials.gov — NCT02461615

Locations in the U.S.

OhioCincinnati Children's Hospital Medical Center, Cincinnati

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.