Treatment Alternatives in iSGS (NoAAC PR-02 Study)

NCT ID: NCT02481817

Last Updated: 2021-10-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

1239 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-08-28

Study Completion Date

2020-09-10

Brief Summary

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The study is aimed at answering the questions; (1) How well do the most commonly used treatments in iSGS work? and (2) What quality-of-life trade-offs are associated with each approach?

With the results of this trial, the investigators hope to provide information to iSGS patients: (1) Given my personal characteristics, conditions, and preferences, what should I expect will happen to me? and (2) What are my options, and what are the potential benefits and harms of these options?

Detailed Description

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Idiopathic subglottic stenosis (iSGS) is a rare disease in which the trachea narrows for no known reason. Although uncommon (with an estimated incidence of 1:400,000 persons per year), both the disease and its therapies profoundly affect patients' ability to breathe, communicate and swallow. Breathing difficulties (i.e. dyspnea) is the hallmark symptom and the primary cause of death and disability. However, patients can also experience debilitating voice changes and swallowing problems due to the condition or its treatment.

People with this disease often require several surgeries per year. A variety of treatments have been advanced to manage iSGS but are generally categorized into: 1) endoscopic dilation of the tracheal stenosis (accomplished with rigid instruments or inflatable balloons); 2) endoscopic resection of the stenosis (with prolonged medical therapy after surgery); or 3) open neck surgery with resection of the affected tracheal segment with end-to-end anastomosis. Each patient can require repeated surgeries to keep their trachea open, which increases odds of treatment side effects and complications. All approaches have unique and often disabling associated side effects, which can significantly affect a patient's quality of life.

Because the disease is rare, it is difficult for patients to find good information so that they can understand the spectrum of treatment options. This is particularly difficult because most patients present with severe breathing trouble and need treatment quickly, limiting their ability to explore options. Additionally, there is a general lack of high-quality, reliable, and accessible data to inform individual patient decision-making. Imperfect information and limited evidence on treatment outcomes complicate patient decision-making as they try to balance survival, symptoms, and quality of life considerations.

Beyond the gaps in understanding the relative effectiveness of the different treatments available, no studies have explored functional outcomes in iSGS (i.e. how well patients breathe, speak, and swallow after treatments). These endpoints are important to patients and are arguably a primary determinant in decision-making. Direct engagement with patients is critical to understand these quality of life considerations, since patient and physician perspectives aren't always the same. For example, results show that endoscopic dilation is associated with a higher rate of disease recurrence and thus need for repeated surgery. Meanwhile, open tracheal resection is a major surgery with significant immediate perioperative risks and has been associated with alterations in voice and swallowing. Open tracheal resection appears to reduce the risk of disease recurrence, but the degree of benefit, and the trade-offs associated with this approach are unanswered questions.

The proposed study from the North American Airway Collaborative (NoAAC PR- 02) is designed to fill this void. Our prospective study will directly compare the effectiveness of standard of care treatments and assess their associated quality of life tradeoffs in iSGS patients.

Conditions

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Idiopathic Subglottic Stenosis (iSGS)

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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iSGS patients

Participants will receive standard of care treatment at the respective center and will be followed longitudinally for symptom changes, need for further treatment, complications, and will have Patient-reported outcomes (PROs) administered at a priori determined intervals.

Endoscopic dilation of subglottic stenosis

Intervention Type PROCEDURE

This is accomplished with rigid instruments or inflatable balloons.

Endoscopic resection of the stenosis

Intervention Type PROCEDURE

This is accomplished with C02 lasers and paired with prolonged medical therapy after surgery.

Tracheal Resection

Intervention Type PROCEDURE

Resection of the affected tracheal segment with end-to-end anastomosis

Interventions

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Endoscopic dilation of subglottic stenosis

This is accomplished with rigid instruments or inflatable balloons.

Intervention Type PROCEDURE

Endoscopic resection of the stenosis

This is accomplished with C02 lasers and paired with prolonged medical therapy after surgery.

Intervention Type PROCEDURE

Tracheal Resection

Resection of the affected tracheal segment with end-to-end anastomosis

Intervention Type PROCEDURE

Other Intervention Names

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Cricotracheal Resection Open Airway Surgery

Eligibility Criteria

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Inclusion Criteria

* Greater than 18 years of age.
* The lesion must involve the subglottis.

Exclusion Criteria

* Less than 18 years of age
* Patients without capacity to consent for themselves
* History of significant laryngotracheal traumatic injury.
* History of endotracheal intubation or tracheotomy within 2 years of presentation.
* Major anterior neck surgery.
* History of neck irradiation.
* History of caustic or thermal injuries to the laryngotracheal complex.
* History of a clinically diagnosed vasculitis or collage vascular disease.
* Positive antinuclear cytoplasmic antibody titers.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Patient-Centered Outcomes Research Institute

OTHER

Sponsor Role collaborator

The Cleveland Clinic

OTHER

Sponsor Role collaborator

University of Utah

OTHER

Sponsor Role collaborator

Johns Hopkins University

OTHER

Sponsor Role collaborator

Vanderbilt University

OTHER

Sponsor Role collaborator

University of Virginia

OTHER

Sponsor Role collaborator

Massachusetts Eye and Ear Infirmary

OTHER

Sponsor Role collaborator

Oregon Health and Science University

OTHER

Sponsor Role collaborator

University of Washington

OTHER

Sponsor Role collaborator

University of California, San Francisco

OTHER

Sponsor Role collaborator

University of California, Los Angeles

OTHER

Sponsor Role collaborator

University of California, San Diego

OTHER

Sponsor Role collaborator

University of Colorado, Denver

OTHER

Sponsor Role collaborator

Baylor College of Medicine

OTHER

Sponsor Role collaborator

Louisiana State University Health Sciences Center in New Orleans

OTHER

Sponsor Role collaborator

Charing Cross Hospital

OTHER

Sponsor Role collaborator

University of Alabama at Birmingham

OTHER

Sponsor Role collaborator

University of Wisconsin, Madison

OTHER

Sponsor Role collaborator

University of Michigan

OTHER

Sponsor Role collaborator

University of Nebraska

OTHER

Sponsor Role collaborator

University of Iowa

OTHER

Sponsor Role collaborator

University of Texas

OTHER

Sponsor Role collaborator

Duke University

OTHER

Sponsor Role collaborator

University of North Carolina

OTHER

Sponsor Role collaborator

Augusta University

OTHER

Sponsor Role collaborator

University of Sydney

OTHER

Sponsor Role collaborator

North American Airway Collaborative

UNKNOWN

Sponsor Role collaborator

University of California, Irvine

OTHER

Sponsor Role collaborator

University of Pittsburgh

OTHER

Sponsor Role collaborator

Loma Linda University

OTHER

Sponsor Role collaborator

Stanford University

OTHER

Sponsor Role collaborator

University of Cincinnati

OTHER

Sponsor Role collaborator

Emory University

OTHER

Sponsor Role collaborator

Ohio State University

OTHER

Sponsor Role collaborator

University of Southern California

OTHER

Sponsor Role collaborator

Temple University

OTHER

Sponsor Role collaborator

Medical College of Wisconsin

OTHER

Sponsor Role collaborator

Landspitali University Hospital

OTHER

Sponsor Role collaborator

Bastian Voice Institute

UNKNOWN

Sponsor Role collaborator

NYU Langone Health

OTHER

Sponsor Role collaborator

Mayo Clinic

OTHER

Sponsor Role collaborator

University of Miami

OTHER

Sponsor Role collaborator

University of Rochester

OTHER

Sponsor Role collaborator

Vanderbilt University Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Alexander Gelbard, MD

Assistant Professor, Department of Otolaryngology

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Alexander Gelbard, MD

Role: PRINCIPAL_INVESTIGATOR

Vanderbilt University Medical Center

David O. Francis, MD, MS

Role: STUDY_CHAIR

Vanderbilt University Medical Center

Locations

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The University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

Mayo Clinic

Scottsdale, Arizona, United States

Site Status

University of California Irvine

Irvine, California, United States

Site Status

Loma Linda University Health Care

Loma Linda, California, United States

Site Status

University of Southern California

Los Angeles, California, United States

Site Status

University of California Los Angeles

Los Angeles, California, United States

Site Status

Stanford University

Palo Alto, California, United States

Site Status

University of California San Diego

San Diego, California, United States

Site Status

University of California San Francisco

San Francisco, California, United States

Site Status

University of Colorado

Denver, Colorado, United States

Site Status

University of Miami

Miami, Florida, United States

Site Status

Emory University School of Medicine

Atlanta, Georgia, United States

Site Status

Augusta University

Augusta, Georgia, United States

Site Status

Bastian Voice Institute

Downers Grove, Illinois, United States

Site Status

University of Iowa

Iowa City, Iowa, United States

Site Status

Louisiana State University

Baton Rouge, Louisiana, United States

Site Status

Johns Hopkins

Baltimore, Maryland, United States

Site Status

Harvard Medical School -Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

University of Nebraska

Omaha, Nebraska, United States

Site Status

New York University Medical Center

New York, New York, United States

Site Status

University of Rochester

Rochester, New York, United States

Site Status

University of North Carolina

Chapel Hill, North Carolina, United States

Site Status

Duke University

Durham, North Carolina, United States

Site Status

University of Cincinatti

Cincinnati, Ohio, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

The Ohio State University Wexner Medical Center

Columbus, Ohio, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Temple University

Philadelphia, Pennsylvania, United States

Site Status

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

University of Texas Southwestern

Dallas, Texas, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

University of Virginia

Charlottesville, Virginia, United States

Site Status

University of Washington

Seattle, Washington, United States

Site Status

University of Wisconsin

Madison, Wisconsin, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

University of Sydney

St Leonards, , Australia

Site Status

Landspitali University Hospital

Reykjavik, , Iceland

Site Status

Charing Cross Hospital, Imperial College London

London, , United Kingdom

Site Status

Countries

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United States Australia Iceland United Kingdom

References

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Gelbard A, Shyr Y, Berry L, Hillel AT, Ekbom DC, Edell ES, Kasperbauer JL, Lott DG, Donovan DT, Garrett CG, Sandhu G, Daniero JJ, Netterville JL, Schindler JS, Smith ME, Bryson PC, Lorenz RR, Francis DO. Treatment options in idiopathic subglottic stenosis: protocol for a prospective international multicentre pragmatic trial. BMJ Open. 2018 Apr 10;8(4):e022243. doi: 10.1136/bmjopen-2018-022243.

Reference Type DERIVED
PMID: 29643170 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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150917

Identifier Type: -

Identifier Source: org_study_id