Treatment Alternatives in iSGS (NoAAC PR-02 Study)

Idiopathic Subglottic Stenosis (iSGS) Clinical Trial

— NoAAC PR-02  

Official title:

Treatment Alternatives in Adult Rare Disease; Assessment of Options in Idiopathic Subglottic Stenosis North American Airway Collaborative PR-02 Study (NoAAC PR-02 Study)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02481817
• Other study ID #: 150917
• Status: Completed
• Start date: August 28, 2015
• Est. completion date: September 10, 2020

Study information

• Verified date: October 2021
• Source: Vanderbilt University Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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?

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1239
• Est. completion date: September 10, 2020
• Est. primary completion date: September 10, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Constriction, Pathologic
• Idiopathic Subglottic Stenosis (iSGS)

Intervention

Procedure:
• Endoscopic dilation of subglottic stenosis
This is accomplished with rigid instruments or inflatable balloons.
• Endoscopic resection of the stenosis
This is accomplished with C02 lasers and paired with prolonged medical therapy after surgery.
• Tracheal Resection
Resection of the affected tracheal segment with end-to-end anastomosis

Locations

Country	Name	City	State
Australia	University of Sydney	St Leonards
Iceland	Landspitali University Hospital	Reykjavik
United Kingdom	Charing Cross Hospital, Imperial College London	London
United States	University of Michigan	Ann Arbor	Michigan
United States	Emory University School of Medicine	Atlanta	Georgia
United States	Augusta University	Augusta	Georgia
United States	Johns Hopkins	Baltimore	Maryland
United States	Louisiana State University	Baton Rouge	Louisiana
United States	The University of Alabama at Birmingham	Birmingham	Alabama
United States	Harvard Medical School -Massachusetts Eye and Ear Infirmary	Boston	Massachusetts
United States	University of North Carolina	Chapel Hill	North Carolina
United States	University of Virginia	Charlottesville	Virginia
United States	University of Cincinatti	Cincinnati	Ohio
United States	Cleveland Clinic	Cleveland	Ohio
United States	The Ohio State University Wexner Medical Center	Columbus	Ohio
United States	University of Texas Southwestern	Dallas	Texas
United States	University of Colorado	Denver	Colorado
United States	Bastian Voice Institute	Downers Grove	Illinois
United States	Duke University	Durham	North Carolina
United States	Baylor College of Medicine	Houston	Texas
United States	University of Iowa	Iowa City	Iowa
United States	University of California Irvine	Irvine	California
United States	Loma Linda University Health Care	Loma Linda	California
United States	University of California Los Angeles	Los Angeles	California
United States	University of Southern California	Los Angeles	California
United States	University of Wisconsin	Madison	Wisconsin
United States	University of Miami	Miami	Florida
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	New York University Medical Center	New York	New York
United States	University of Nebraska	Omaha	Nebraska
United States	Stanford University	Palo Alto	California
United States	Temple University	Philadelphia	Pennsylvania
United States	University of Pittsburgh	Pittsburgh	Pennsylvania
United States	Oregon Health & Science University	Portland	Oregon
United States	Mayo Clinic	Rochester	Minnesota
United States	University of Rochester	Rochester	New York
United States	University of Utah	Salt Lake City	Utah
United States	University of California San Diego	San Diego	California
United States	University of California San Francisco	San Francisco	California
United States	Mayo Clinic	Scottsdale	Arizona
United States	University of Washington	Seattle	Washington

Sponsors (44)

Lead Sponsor

Collaborator

Vanderbilt University Medical Center

Augusta University, Bastian Voice Institute, Baylor College of Medicine, Charing Cross Hospital, Duke University, Emory University, Johns Hopkins University, Landspitali University Hospital, Loma Linda University, Louisiana State University Health Sciences Center in New Orleans, Massachusetts Eye and Ear Infirmary, Mayo Clinic, Medical College of Wisconsin, North American Airway Collaborative, NYU Langone Health, Ohio State University, Oregon Health and Science University, Patient-Centered Outcomes Research Institute, Stanford University, Temple University, The Cleveland Clinic, University of Alabama at Birmingham, University of California, Irvine, University of California, Los Angeles, University of California, San Diego, University of California, San Francisco, University of Cincinnati, University of Colorado, Denver, University of Iowa, University of Miami, University of Michigan, University of Nebraska, University of North Carolina, University of Pittsburgh, University of Rochester, University of Southern California, University of Sydney, University of Texas, University of Utah, University of Virginia, University of Washington, University of Wisconsin, Madison, Vanderbilt University

Countries where clinical trial is conducted

United States,  Australia,  Iceland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Treatment Effectiveness: Time to recurrent procedure		3 years
Primary	Treatment Effectiveness: Need for tracheostomy		3 years
Secondary	QOL	Patient quality of life assessment: voice (VHI-10)	3 years
Secondary	QOL	Patient quality of life assessment: dysphagia (EAT-10)	3 years
Secondary	QOL	Patient quality of life assessment: breathing (COPD dyspnea)	3 years
Secondary	QOL	Patient quality of life assessment: general quality of life (SF-12)	3 years
Secondary	Patient Reported Outcome	Non-traditional PRO focused on social support	3 years
Secondary	Patient Reported Outcome	Non-traditional PRO focused on fear of disease recurrence	3 years
Secondary	Patient Reported Outcome	Non-traditional PRO focused on disease anxiety and burden	3 years
Secondary	Patient Reported Outcome	Non-traditional PRO focused on participatory decision-making style	3 years