Status:

RECRUITING

A National Registry For Pulmonary Alveolar Proteinosis

Lead Sponsor:

Children's Hospital Medical Center, Cincinnati

Collaborating Sponsors:

Rare Diseases Clinical Research Network

National Heart, Lung, and Blood Institute (NHLBI)

Conditions:

Pulmonary Alveolar Proteinosis

Eligibility:

All Genders

Brief 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 kno...

Detailed Description

PAP is a rare syndrome of surfactant accumulation and resulting hypoxemic respiratory failure that occurs in a number of diseases classified pathogenically into three groups: primary PAP (caused by di...

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

Key Trial Info

Start Date :

April 1 2015

Trial Type :

OBSERVATIONAL

Allocation :

ESTIMATED

End Date :

December 1 2030

Estimated Enrollment :

500 Patients enrolled

Trial Details

Trial ID

NCT02461615

Start Date

April 1 2015

End Date

December 1 2030

Last Update

March 17 2026

Active Locations (1)

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1

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229