Idiopathic Subglottic Stenosis (iSGS)NoAAC PR-02NCT02481817
Treatment Alternatives in iSGS (NoAAC PR-02 Study)
An observational study of Idiopathic Subglottic Stenosis (iSGS), run by Vanderbilt University Medical Center at 42 sites in 4 countries.
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Idiopathic Subglottic Stenosis (iSGS)
- What is being tested?
- Endoscopic dilation of subglottic stenosis, Endoscopic resection of the stenosis and Tracheal Resection
- Type of treatment
- Surgery or procedure
- How many people
- 1,239 participants
- Where
- 42 sitesin 4 countries
- Study dates
- 28 Aug 2015to 10 Sep 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: All clinically confirmed iSGS patients at the participating institutions are eligible for enrollment.
Who may be able to join
- Greater than 18 years of age.
- The lesion must involve the subglottis.
Who cannot join
- 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.
Show all 9Show fewer
- 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.
Trial locations and contacts
42 sites in 4 countries.
42 sites in 4 countries
United States39 sites
University of Michigan
Ann Arbor, Michigan, 48109
Emory University School of Medicine
Atlanta, Georgia, 30322
Augusta University
Augusta, Georgia, 30912
Johns Hopkins
Baltimore, Maryland, 21287
Louisiana State University
Baton Rouge, Louisiana, 70809
The University of Alabama at Birmingham
Birmingham, Alabama, 35233
Harvard Medical School -Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114
University of North Carolina
Chapel Hill, North Carolina, 27599
University of Virginia
Charlottesville, Virginia, 22908-0713
University of Cincinatti
Cincinnati, Ohio, 45267
Cleveland Clinic
Cleveland, Ohio, 44195
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43212
University of Texas Southwestern
Dallas, Texas, 75390
University of Colorado
Denver, Colorado, 80045
Bastian Voice Institute
Downers Grove, Illinois, 60515
Duke University
Durham, North Carolina, 27705
Baylor College of Medicine
Houston, Texas, 77030
University of Iowa
Iowa City, Iowa, 52242
University of California Irvine
Irvine, California, 92606
Loma Linda University Health Care
Loma Linda, California, 92354
University of Southern California
Los Angeles, California, 90033
University of California Los Angeles
Los Angeles, California, 90095
University of Wisconsin
Madison, Wisconsin, 53792
University of Miami
Miami, Florida, 33136
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
New York University Medical Center
New York, New York, 10016
University of Nebraska
Omaha, Nebraska, 68198
Stanford University
Palo Alto, California, 94304
Temple University
Philadelphia, Pennsylvania, 19140
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Oregon Health & Science University
Portland, Oregon, 97239
Mayo Clinic
Rochester, Minnesota, 55902
University of Rochester
Rochester, New York, 14642
University of Utah
Salt Lake City, Utah, 84132
University of California San Diego
San Diego, California, 92103
University of California San Francisco
San Francisco, California, 94115
Mayo Clinic
Scottsdale, Arizona, 85054
University of Washington
Seattle, Washington, 98195
United Kingdom1 site
Charing Cross Hospital, Imperial College London
London, W6 8RF
About this 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)
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?
Read the 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.
- Conditions
- Idiopathic Subglottic Stenosis (iSGS)
- Being tested
- Endoscopic dilation of subglottic stenosis, Endoscopic resection of the stenosis, Tracheal Resection
- Medical subject headings
- Idiopathic subglottic tracheal stenosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Endoscopic dilation of subglottic stenosisProcedure or surgery
This is accomplished with rigid instruments or inflatable balloons.
Endoscopic resection of the stenosisProcedure or surgery
This is accomplished with C02 lasers and paired with prolonged medical therapy after surgery.
Tracheal ResectionProcedure or surgery
Also called Cricotracheal Resection, Open Airway Surgery
Resection of the affected tracheal segment with end-to-end anastomosis
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Procedure: Endoscopic dilation of subglottic stenosis; Procedure: Endoscopic resection of the stenosis; Procedure: Tracheal Resection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment Effectiveness: Time to recurrent procedure
Measured: 3 years
Treatment Effectiveness: Need for tracheostomy
Measured: 3 years
Secondary outcomes (8)
QOL
Patient quality of life assessment: voice (VHI-10)
Measured: 3 years
QOL
Patient quality of life assessment: dysphagia (EAT-10)
Measured: 3 years
QOL
Patient quality of life assessment: breathing (COPD dyspnea)
Measured: 3 years
QOL
Patient quality of life assessment: general quality of life (SF-12)
Measured: 3 years
Patient Reported Outcome
Non-traditional PRO focused on social support
Measured: 3 years
Patient Reported Outcome
Non-traditional PRO focused on fear of disease recurrence
Measured: 3 years
Patient Reported Outcome
Non-traditional PRO focused on disease anxiety and burden
Measured: 3 years
Patient Reported Outcome
Non-traditional PRO focused on participatory decision-making style
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2015 (estimated)
- Study start28 Aug 2015
- Primary completion10 Sep 2020
- Study completion10 Sep 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Idiopathic Subglottic Stenosis (iSGS)Studies of the same condition, open and under way.
- More trials run by Vanderbilt University Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,239 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vanderbilt University Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- 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
- Responsible party
- Principal investigator, Alexander Gelbard, MD, Vanderbilt University Medical Center
- Investigators
- Alexander Gelbard, MD, Principal investigator, Vanderbilt University Medical CenterDavid O. Francis, MD, MS, Study chair, Vanderbilt University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02481817
- Sponsor's protocol ID
- 150917
- Acronym
- NoAAC PR-02
- Status last verified
- Oct 2021
- First submitted
- 22 Jun 2015
- Last updated
- 28 Oct 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 29643170
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02481817
- Acronym / sponsor ID
- NoAAC PR-02 / 150917
- Conditions
- Idiopathic Subglottic Stenosis (iSGS)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,239 participants (actual)
- Lead sponsor
- Vanderbilt University Medical CenterUniversity, hospital or other
- Collaborators
- 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
- Study start
- 28 Aug 2015 (actual)
- Primary completion
- 10 Sep 2020 (actual)
- Study completion
- 10 Sep 2020 (actual)
- First posted
- 25 Jun 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| iSGS patients | – | Procedure: Endoscopic dilation of subglottic stenosis, Procedure: Endoscopic resection of the stenosis, Procedure: Tracheal ResectionParticipants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- All clinically confirmed iSGS patients at the participating institutions are eligible for enrollment.
Key inclusion criteria (2 listed)
- Greater than 18 years of age.
- The lesion must involve the subglottis.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Endoscopic dilation of subglottic stenosisThis is accomplished with rigid instruments or inflatable balloons. | Procedure or surgery | iSGS patients |
| Endoscopic resection of the stenosisThis is accomplished with C02 lasers and paired with prolonged medical therapy after surgery. | Procedure or surgery | iSGS patients |
| Tracheal ResectionAlso: Cricotracheal Resection, Open Airway SurgeryResection of the affected tracheal segment with end-to-end anastomosis | Procedure or surgery | iSGS patients |
Primary outcome
Treatment Effectiveness: Time to recurrent procedure
Time frame: 3 years
Treatment Effectiveness: Need for tracheostomy
Time frame: 3 years
8 secondary outcomes
QOL
Time frame: 3 years
QOL
Time frame: 3 years
QOL
Time frame: 3 years
QOL
Time frame: 3 years
Patient Reported Outcome
Time frame: 3 years
Patient Reported Outcome
Time frame: 3 years
Patient Reported Outcome
Time frame: 3 years
Patient Reported Outcome
Time frame: 3 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2021.
- Derived from this study: PubMed 29643170Gelbard 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.
- Individual participant dataSharing planned: Undecided
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alexander Gelbard, MD principal investigator
Vanderbilt University Medical Center
David O. Francis, MD, MS study chair
Vanderbilt University Medical Center
42 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Idiopathic Subglottic Stenosis (iSGS), run by Vanderbilt University Medical Center at 42 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 42 sites in United States, Australia, Iceland, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment Effectiveness: Time to recurrent procedure (time frame: 3 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02481817, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Oct 2021.