Treatment Alternatives in iSGS (NoAAC PR-02 Study)

• Sponsor: Vanderbilt University Medical Center (Other)
• Overall Status: Completed
• CT.gov ID: NCT02481817
• Collaborator: Patient-Centered Outcomes Research Institute (Other), The Cleveland Clinic (Other), University of Utah (Other), Johns Hopkins University (Other), Vanderbilt University (Other), University of Virginia (Other), Massachusetts Eye and Ear Infirmary (Other), Oregon Health and Science University (Other), University of Washington (Other), University of California, San Francisco (Other), University of California, Los Angeles (Other), University of California, San Diego (Other), University of Colorado, Denver (Other), Baylor College of Medicine (Other), Louisiana State University Health Sciences Center in New Orleans (Other), Charing Cross Hospital (Other), University of Alabama at Birmingham (Other), University of Wisconsin, Madison (Other), University of Michigan (Other), University of Nebraska (Other), University of Iowa (Other), University of Texas (Other), Duke University (Other), University of North Carolina (Other), Augusta University (Other), University of Sydney (Other), North American Airway Collaborative (Other), University of California, Irvine (Other), University of Pittsburgh (Other), Loma Linda University (Other), Stanford University (Other), University of Cincinnati (Other), Emory University (Other), Ohio State University (Other), University of Southern California (Other), Temple University (Other), Medical College of Wisconsin (Other), Landspitali University Hospital (Other), Bastian Voice Institute (Other), NYU Langone Health (Other), Mayo Clinic (Other), University of Miami (Other), University of Rochester (Other)
• Enrollment: 1,239
• Locations: 42
• Actual Duration (Months): 60.5
• Patients Per Site: 29.5
• Patients Per Site Per Month: 0.5

Study Details

Study Description

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?

Condition or Disease	Intervention/Treatment	Phase
Idiopathic Subglottic Stenosis (iSGS)	Procedure: Endoscopic dilation of subglottic stenosis; Procedure: Endoscopic resection of the stenosis; Procedure: Tracheal Resection

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.

Study Design

• Study Type: Observational
• Actual Enrollment: 1239 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• 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)
• Actual Study Start Date: Aug 28, 2015
• Actual Primary Completion Date: Sep 10, 2020
• Actual Study Completion Date: Sep 10, 2020

Arms and Interventions

Arm

Intervention/Treatment

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.

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

Outcome Measures

Primary Outcome Measures

1. Treatment Effectiveness: Time to recurrent procedure [3 years]

2. Treatment Effectiveness: Need for tracheostomy [3 years]

Secondary Outcome Measures

1. QOL [3 years]

   Patient quality of life assessment: voice (VHI-10)

2. QOL [3 years]

   Patient quality of life assessment: dysphagia (EAT-10)

3. QOL [3 years]

   Patient quality of life assessment: breathing (COPD dyspnea)

4. QOL [3 years]

   Patient quality of life assessment: general quality of life (SF-12)

5. Patient Reported Outcome [3 years]

   Non-traditional PRO focused on social support

6. Patient Reported Outcome [3 years]

   Non-traditional PRO focused on fear of disease recurrence

7. Patient Reported Outcome [3 years]

   Non-traditional PRO focused on disease anxiety and burden

8. Patient Reported Outcome [3 years]

   Non-traditional PRO focused on participatory decision-making style

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	The University of Alabama at Birmingham	Birmingham	Alabama	United States	35233
2	Mayo Clinic	Scottsdale	Arizona	United States	85054
3	University of California Irvine	Irvine	California	United States	92606
4	Loma Linda University Health Care	Loma Linda	California	United States	92354
5	University of Southern California	Los Angeles	California	United States	90033
6	University of California Los Angeles	Los Angeles	California	United States	90095
7	Stanford University	Palo Alto	California	United States	94304
8	University of California San Diego	San Diego	California	United States	92103
9	University of California San Francisco	San Francisco	California	United States	94115
10	University of Colorado	Denver	Colorado	United States	80045
11	University of Miami	Miami	Florida	United States	33136
12	Emory University School of Medicine	Atlanta	Georgia	United States	30322
13	Augusta University	Augusta	Georgia	United States	30912
14	Bastian Voice Institute	Downers Grove	Illinois	United States	60515
15	University of Iowa	Iowa City	Iowa	United States	52242
16	Louisiana State University	Baton Rouge	Louisiana	United States	70809
17	Johns Hopkins	Baltimore	Maryland	United States	21287
18	Harvard Medical School -Massachusetts Eye and Ear Infirmary	Boston	Massachusetts	United States	02114
19	University of Michigan	Ann Arbor	Michigan	United States	48109
20	Mayo Clinic	Rochester	Minnesota	United States	55902
21	University of Nebraska	Omaha	Nebraska	United States	68198
22	New York University Medical Center	New York	New York	United States	10016
23	University of Rochester	Rochester	New York	United States	14642
24	University of North Carolina	Chapel Hill	North Carolina	United States	27599
25	Duke University	Durham	North Carolina	United States	27705
26	University of Cincinatti	Cincinnati	Ohio	United States	45267
27	Cleveland Clinic	Cleveland	Ohio	United States	44195
28	The Ohio State University Wexner Medical Center	Columbus	Ohio	United States	43212
29	Oregon Health & Science University	Portland	Oregon	United States	97239
30	Temple University	Philadelphia	Pennsylvania	United States	19140
31	University of Pittsburgh	Pittsburgh	Pennsylvania	United States	15213
32	Vanderbilt University Medical Center	Nashville	Tennessee	United States	37232
33	University of Texas Southwestern	Dallas	Texas	United States	75390
34	Baylor College of Medicine	Houston	Texas	United States	77030
35	University of Utah	Salt Lake City	Utah	United States	84132
36	University of Virginia	Charlottesville	Virginia	United States	22908-0713
37	University of Washington	Seattle	Washington	United States	98195
38	University of Wisconsin	Madison	Wisconsin	United States	53792
39	Medical College of Wisconsin	Milwaukee	Wisconsin	United States	53226
40	University of Sydney	St Leonards		Australia
41	Landspitali University Hospital	Reykjavik		Iceland	101-155
42	Charing Cross Hospital, Imperial College London	London		United Kingdom	W6 8RF

Sponsors and Collaborators

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

Investigators

• Principal Investigator: Alexander Gelbard, MD, Vanderbilt University Medical Center
• Study Chair: David O. Francis, MD, MS, Vanderbilt University Medical Center

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Jul 12, 2018

More Information

Publications

None provided.

• Responsible Party: Alexander Gelbard, MD, Assistant Professor, Department of Otolaryngology, Vanderbilt University Medical Center
• ClinicalTrials.gov Identifier: NCT02481817
• Other Study ID Numbers: 150917
• First Posted: Jun 25, 2015
• Last Update Posted: Oct 28, 2021
• Last Verified: Oct 1, 2021

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Constriction, Pathologic

Study Results

No Results Posted as of Oct 28, 2021