Health TrialsbyHealthPanel

A National Registry For Pulmonary Alveolar Proteinosis

ConditionPulmonary Alveolar Proteinosis
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorChildren's Hospital Medical Center, Cincinnati

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Written informed consent and assent, if applicable

History of chest computed tomogram or chest radiograph findings compatible with PAP

Positive (Abnormal) serum GMAb test -OR-

Lung biopsy clearly documenting the presence of PAP of any type or degree -OR-

Disqualifiers

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.

Individuals that do not have a diagnosis of PAP

Individuals that do not have a diagnosis of autoimmune PAP

Trial population

Patients with Pulmonary Alveolar Proteinosis (Part A) Patient with Autoimmune Pulmonary Alveolar Proteinosis (Part B)

Trial design

Design model

Other

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

500 Participants
are grouped into 1 trial group
Group A: Registry Participants

Trial outcomes

Primary outcomes

1

DBS card GM-CSF autoantibody levels to diagnose Autoimmune PAP

GM-CSF antibody measurement from a diagnostic blood spot (DBS) card to diagnose autoimmune PAP among participants with PAP of any type

Time frame
5 years

Secondary outcomes

1

Prevalence of Autoimmune PAP

Time frame
5 years
2

Genetic risks for PAP

Use diagnostic blood spot (DBS) card-based specimens to identify genetic factors that increase risk of developing PAP

Time frame
5 years
3

Sensitivity and Specificity of DBS card GM-CSF autoantibody test for Autoimmune PAP

Time frame
5 years
4

Retrospective, longitudinal, chart-based natural history study of aPAP

Time frame
2 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Children's Hospital Medical Center, Cincinnati

Lead sponsor

Rare Diseases Clinical Research Network

Collaborator

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

National Center for Advancing Translational Sciences (NCATS)

Collaborator

Source ClinicalTrials.gov
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