NCT03531996

Brief Summary

The major goal of this study is to conduct a prospective, longitudinal study of autoimmune PAP to examine outcome measures for disease severity of potential use in clinical practice and/or clinical research studies. These results will impact the field by: 1) improving an understanding of the clinical course of autoimmune PAP, 2) providing information on various clinical outcome and quality of life outcome measures to guide patients and physicians in making treatment choices, and 3) facilitate the development of pharmaco-therapeutics for autoimmune PAP and 4) better informing PAP researchers.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
29

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Apr 2018

Typical duration for all trials

Geographic Reach
1 country

1 active site

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

April 19, 2018

Completed
18 days until next milestone

First Submitted

Initial submission to the registry

May 7, 2018

Completed
15 days until next milestone

First Posted

Study publicly available on registry

May 22, 2018

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 16, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 16, 2020

Completed
Last Updated

June 24, 2021

Status Verified

June 1, 2021

Enrollment Period

2.4 years

First QC Date

May 7, 2018

Last Update Submit

June 21, 2021

Conditions

Autoimmune Pulmonary Alveolar Proteinosis

Keywords

Pulmonary SurfactantRare Lung DiseaseRegistry

Outcome Measures

Primary Outcomes (5)

  • Longitudinal evaluation of GM-CSF autoantibody levels

    Change in GM-CSF autoantibody levels in autoimmune PAP patients over time

    Baseline, 1 year, and 2 years

  • Longitudinal evaluation of the maximal phospho-STAT5 level after GM-CSF stimulation

    Change in phospho-STAT5 levels in autoimmune PAP patients over time

    Baseline, 1 year, and 2 years

  • Longitudinal evaluation of the STAT5 Phosphorylation Index

    Change in the STAT5 phosphorylation index in autoimmune PAP patients over time

    Baseline, 1 year, and 2 years

  • Longitudinal evaluation of the GM-CSF Signaling Index

    Change in GM-CSF signaling index in autoimmune PAP patients over time

    Baseline, 1 year, and 2 years

  • Longitudinal evaluation of the dose GM-CSF to stimulation 1/2 maximal STAT5 phosphorylation (EC50)

    Change in GM-CSF EC50 level in autoimmune PAP patients over time

    Baseline, 1 year, and 2 years

Secondary Outcomes (9)

  • Frequency of therapeutic intervention

    Baseline, 1 year, and 2 years

  • Concurrent infections

    Baseline, 1 year, and 2 years

  • Blood SP-D

    Baseline, 1 year, and 2 years

  • Blood cholestenoic acid

    Baseline, 1 year, and 2 years

  • Blood lipid levels

    Baseline, 1 year, and 2 years

  • +4 more secondary outcomes

Eligibility Criteria

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

Patients with Pulmonary Alveolar Proteinosis

You may qualify if:

  • Written informed consent must be provided by:
  • Participant if at least 18 years old -OR-
  • Parent/legal guardian if participant is less than 18 years old -AND-
  • Participant provides assent when appropriate
  • History of diagnosis of autoimmune as indicated by a:
  • History of chest CT or x-ray findings compatible with PAP -AND-
  • History of a Positive (Abnormal) serum GMAb test

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Related Publications (3)

  • Trapnell BC, Whitsett JA, Nakata K. Pulmonary alveolar proteinosis. N Engl J Med. 2003 Dec 25;349(26):2527-39. doi: 10.1056/NEJMra023226. No abstract available.

    PMID: 14695413BACKGROUND

  • Carey B, Trapnell BC. The molecular basis of pulmonary alveolar proteinosis. Clin Immunol. 2010 May;135(2):223-35. doi: 10.1016/j.clim.2010.02.017. Epub 2010 Mar 25.

    PMID: 20338813BACKGROUND

  • Uchida K, Nakata K, Carey B, Chalk C, Suzuki T, Sakagami T, Koch DE, Stevens C, Inoue Y, Yamada Y, Trapnell BC. Standardized serum GM-CSF autoantibody testing for the routine clinical diagnosis of autoimmune pulmonary alveolar proteinosis. J Immunol Methods. 2014 Jan 15;402(1-2):57-70. doi: 10.1016/j.jim.2013.11.011. Epub 2013 Nov 23.

    PMID: 24275678BACKGROUND

Biospecimen

Retention: NONE RETAINED

Biospecimens retained will be serum, plasma, and DNA.

MeSH Terms

Conditions

Pulmonary Alveolar Proteinosis, Acquired

Study Officials

  • Bruce Trapnell, MD

    Children's Hospital Medical Center, Cincinnati

    STUDY CHAIR

Study Design

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

Study Record Dates

First Submitted

May 7, 2018

First Posted

May 22, 2018

Study Start

April 19, 2018

Primary Completion

September 16, 2020

Study Completion

September 16, 2020

Last Updated

June 24, 2021

Record last verified: 2021-06

Data Sharing

IPD Sharing
Will not share

Locations

US(1)