NCT03007134

Brief Summary

The purpose of this study is to (1) compare a technically improved assay with an existing assay used to measure serum anti-GM-CSF antibodies in stored serum samples previously obtained from patients diagnosed with either primary, secondary, congenital or idiopathic pulmonary alveolar proteinosis (PAP), other chronic diseases or disease-free, healthy individuals; (2) determine the prevalence and levels of anti-GM-CSF autoantibodies and (3) define the breadth of the autoimmune antibody responses in primary PAP patients from the United States, Japan, Australia, and Europe using previously collected serum samples; and (4) using a chart review approach, compare the clinical, radiologic and laboratory features of primary PAP patients to determine if differences exist among patients in these globally geographically distributed regions.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
73

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Jul 2007

Longer than P75 for all trials

Geographic Reach
4 countries

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2007

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2010

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2011

Completed
5.4 years until next milestone

First Submitted

Initial submission to the registry

November 28, 2016

Completed
1 month until next milestone

First Posted

Study publicly available on registry

January 2, 2017

Completed
Last Updated

January 2, 2017

Status Verified

December 1, 2016

Enrollment Period

3 years

First QC Date

November 28, 2016

Last Update Submit

December 29, 2016

Conditions

Pulmonary Alveolar Proteinosis

Outcome Measures

Primary Outcomes (1)

  • Standardize the anti-GM-CSF autoantibody ELISA

    To compare a novel assay to an existing assay used to measure anti-GM-CSF autoantibodies in banked serum samples previously collected from individuals diagnosed with primary, secondary, congenital or idiopathic PAP, other chronic disorders, or disease-free, healthy individuals.

    1 year

Secondary Outcomes (11)

  • Geographic distribution of anti-GM-CSF autoantibody levels

    1 years

  • Breadth of the autoimmune antibody phenotype of individuals with autoimmune PAP

    3 years

  • Age of onset PAP symptoms

    2 years

  • Age at the time of definitive PAP diagnosis

    2 years

  • Length of time from onset of symptoms and definitive diagnosis of autoimmune PAP

    2 years

  • +6 more secondary outcomes

Interventions

Retrospective Chart ReviewOTHER

No intervention.

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

Autoimmune PAP

You may qualify if:

  • Data and Serum must be from individuals of any age with confirmed diagnosis of PAP based on the following criteria:
  • Radiographic features consistent with PAP
  • Pathological or cytological findings consistent with PAP determined by either open lung biopsy, transbronchial biopsy or bronchoalveolar lavage cell/fluid cytology.
  • Elevated anti-GM-CSF autoantibody concentration, when available
  • Ability of the international PI's and their research coordinators to ship samples in compliance with this study.

You may not qualify if:

  • No established diagnosis of PAP
  • Incomplete chart records in which \>25% of the requested data is unable to be confirmed.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Brody School of Medicine

Greenville, North Carolina, 27834, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Ruhrlandklinik Essen

Essen, Germany

Location

Clinica Malattie Apparato Respiratorio Università di Pavia

Pavia, Italy

Location

University of Niigata Medical and Dental School

Niigata, Japan

Location

NHO Kinki-Chuo Chest Center

Osaka, Japan

Location

MeSH Terms

Conditions

Pulmonary Alveolar Proteinosis

Condition Hierarchy (Ancestors)

Lung DiseasesRespiratory Tract Diseases

Study Officials

  • Bruce Trapnell

    Children's Hospital Medical Center, Cincinnati

    STUDY CHAIR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 28, 2016

First Posted

January 2, 2017

Study Start

July 1, 2007

Primary Completion

July 1, 2010

Study Completion

July 1, 2011

Last Updated

January 2, 2017

Record last verified: 2016-12

Data Sharing

IPD Sharing
Will not share

Locations

JP(2)US(2)IT(1)DE(1)