NCT03182049

Brief Summary

The ultimate goal of this prospective natural history study is to define the natural history of the obstructive airway manifestations of Granulomatosis with polyangiitis (GPA). Additionally this proposal seeks to develop biomarkers of disease activity and define their correlation with clinical outcomes in an effort to transform clinical care and shape future drug development for this devastating rare disease.

Trial Health

30
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Timeline
Completed

Started Dec 2020

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
withdrawn

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

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 5, 2017

Completed
4 days until next milestone

First Posted

Study publicly available on registry

June 9, 2017

Completed
3.5 years until next milestone

Study Start

First participant enrolled

December 1, 2020

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2022

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2023

Completed
Last Updated

August 25, 2020

Status Verified

August 1, 2020

Enrollment Period

1.4 years

First QC Date

June 5, 2017

Last Update Submit

August 24, 2020

Conditions

Keywords

GPAGranulomatosis with polyangiitisWegener's granulomatosisSubglottic StenosisBronchial Stenosis

Outcome Measures

Primary Outcomes (1)

  • Time to recurrent intervention (TTR)

    The time between interventions aimed at preserving an open airway.

    5 years

Secondary Outcomes (5)

  • Patient Reported Outcome Measures

    5 years

  • Patient Reported Outcome Measures

    5 years

  • Patient Reported Outcome Measures

    5 years

  • Patient Reported Outcome Measures

    5 years

  • Disease Assessment clinical tool

    5 years

Study Arms (1)

GPA (Wegener's granulomatosis) patients

Procedure: Airway assessmentProcedure: Rheumatology assessment

Interventions

Airway Specialist (Otolaryngology, Pulmonology, Thoracic Surgery), assess clinical location and degree of airway compromise, glottic mobility, and sinonasal disease.

GPA (Wegener's granulomatosis) patients

Clinically assess systemic function (renal/pulmonary ect), biochemical profile (routine labs), neurocognitive function, and completing entry BVAS tool.

GPA (Wegener's granulomatosis) patients

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Adult Granulomtosis with Polyangiitis Population Affected by Obstructive Airway Disease patients are eligible for enrollment.

You may qualify if:

  • Greater than or equal to 18 years of age.
  • Stenotic Airway Disease (laryngeal, subglottic, distal tracheal or bronchial)

You may not qualify if:

  • \<18years of age
  • Patients without capacity to consent for themselves
  • History of significant laryngotracheal traumatic injury.
  • Endotracheal intubation 2 years prior to presentation.
  • Major anterior neck surgery.
  • History of neck irradiation.
  • History of caustic or thermal injury to the laryngotracheal complex.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

We plan to obtain a small amount of blood at a time when other medically indicated labs, operative or office-based procedures occur. Excess tissue obtained at standard of care surgical biopsy will be preserved for molecular study.

MeSH Terms

Conditions

Granulomatosis with Polyangiitis

Condition Hierarchy (Ancestors)

Lung Diseases, InterstitialLung DiseasesRespiratory Tract DiseasesAnti-Neutrophil Cytoplasmic Antibody-Associated VasculitisSystemic VasculitisVasculitisVascular DiseasesCardiovascular DiseasesSkin Diseases, VascularSkin DiseasesSkin and Connective Tissue DiseasesAutoimmune DiseasesImmune System Diseases

Study Officials

  • Alexander Gelbard, MD

    Vanderbilt University Medical Center

    PRINCIPAL INVESTIGATOR
0

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Assistant Professor, Department of Otolaryngology

Study Record Dates

First Submitted

June 5, 2017

First Posted

June 9, 2017

Study Start

December 1, 2020

Primary Completion

May 1, 2022

Study Completion

May 1, 2023

Last Updated

August 25, 2020

Record last verified: 2020-08

Locations