National registry for pulmonary alveolar proteinosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to establish a National Registry for Pulmonary Alveolar Proteinosis (PAP) to enhance the diagnosis and treatment of this rare lung disease. It will collect data to improve awareness and knowledge of PAP, allowing patients to participate in research planning and clinical testing of new therapies. The study will also define the natural history of autoimmune PAP and develop tools to measure disease severity based on patient experiences. By utilizing new blood-based tests, the registry seeks to identify the underlying causes of PAP more effectively than current methods.

Why it matters

Who can join: the full rules

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

Where it's running

• Cincinnati Children's Hospital Medical Center
  Cincinnati, Ohio, United States Recruiting

Study contacts

• Study coordinator: Brenna C Carey, Ms, PhD
• Email: Brenna.Carey@cchmc.org
• Phone: 513-636-8916

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.