Clinical trial NCT02481817: Treatment Alternatives in iSGS (NoAAC PR-02 Study)
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)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Idiopathic Subglottic Stenosis (iSGS)
Lead sponsor: Vanderbilt University Medical Center
Sponsor class: OTHER
Collaborators: name: Patient-Centered Outcomes Research Institute
name: The Cleveland Clinic
name: University of Utah
name: Johns Hopkins University
name: Vanderbilt University
name: University of Virginia
name: Massachusetts Eye and Ear Infirmary
name: Oregon Health and Science University
name: University of Washington
name: University of California, San Francisco
name: University of California, Los Angeles
name: University of California, San Diego
name: University of Colorado, Denver
name: Baylor College of Medicine
name: Louisiana State University Health Sciences Center in New Orleans
name: Charing Cross Hospital
name: University of Alabama at Birmingham
name: University of Wisconsin, Madison
name: University of Michigan
name: University of Nebraska
name: University of Iowa
name: University of Texas
name: Duke University
name: University of North Carolina
name: Augusta University
name: University of Sydney
name: North American Airway Collaborative
name: University of California, Irvine
name: University of Pittsburgh
name: Loma Linda University
name: Stanford University
name: University of Cincinnati
name: Emory University
name: Ohio State University
name: University of Southern California
name: Temple University
name: Medical College of Wisconsin
name: Landspitali University Hospital
name: Bastian Voice Institute
name: NYU Langone Health
name: Mayo Clinic
name: University of Miami
name: University of Rochester
First posted: 2015-06-25
Enrollment: 1239
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All clinically confirmed iSGS patients at the participating institutions are eligible for enrollment.

Brief 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?

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Treatment Effectiveness: Time to recurrent procedure
timeFrame: 3 years
measure: Treatment Effectiveness: Need for tracheostomy
timeFrame: 3 years
Secondary outcomes:
measure: QOL
timeFrame: 3 years
measure: QOL
timeFrame: 3 years
measure: QOL
timeFrame: 3 years
measure: QOL
timeFrame: 3 years
measure: Patient Reported Outcome
timeFrame: 3 years
measure: Patient Reported Outcome
timeFrame: 3 years
measure: Patient Reported Outcome
timeFrame: 3 years
measure: Patient Reported Outcome
timeFrame: 3 years
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: The University of Alabama at Birmingham
state: Alabama
city: Scottsdale
country: United States
facility: Mayo Clinic
state: Arizona
city: Irvine
country: United States
facility: University of California Irvine
state: California
city: Loma Linda
country: United States
facility: Loma Linda University Health Care
state: California
city: Los Angeles
country: United States
facility: University of Southern California
state: California
city: Los Angeles
country: United States
facility: University of California Los Angeles
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: San Diego
country: United States
facility: University of California San Diego
state: California
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Denver
country: United States
facility: University of Colorado
state: Colorado
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Downers Grove
country: United States
facility: Bastian Voice Institute
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Baton Rouge
country: United States
facility: Louisiana State University
state: Louisiana
city: Baltimore
country: United States
facility: Johns Hopkins
state: Maryland
city: Boston
country: United States
facility: Harvard Medical School -Massachusetts Eye and Ear Infirmary
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Omaha
country: United States
facility: University of Nebraska
state: Nebraska
city: New York
country: United States
facility: New York University Medical Center
state: New York
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Cincinnati
country: United States
facility: University of Cincinatti
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: Temple University
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: University of Texas Southwestern
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
city: Seattle
country: United States
facility: University of Washington
state: Washington
city: Madison
country: United States
facility: University of Wisconsin
state: Wisconsin
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: St Leonards
country: Australia
facility: University of Sydney
city: Reykjavik
country: Iceland
facility: Landspitali University Hospital
city: London
country: United Kingdom
facility: Charing Cross Hospital, Imperial College London
Study officials:
affiliation: Vanderbilt University Medical Center
name: Alexander Gelbard, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Vanderbilt University Medical Center
name: David O. Francis, MD, MS
role: STUDY_CHAIR
References:
citation: 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.