Treatment Alternatives in iSGS (NoAAC PR-02 Study) (NoAAC PR-02)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02481817|
Recruitment Status : Active, not recruiting
First Posted : June 25, 2015
Last Update Posted : January 22, 2020
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|
|Idiopathic Subglottic Stenosis (iSGS)||Procedure: Endoscopic dilation of subglottic stenosis Procedure: Endoscopic resection of the stenosis Procedure: Tracheal Resection|
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 Type :||Observational|
|Estimated Enrollment :||1056 participants|
|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 :||August 28, 2015|
|Estimated Primary Completion Date :||December 2020|
|Estimated Study Completion Date :||December 2020|
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
- Treatment Effectiveness: Time to recurrent procedure [ Time Frame: 3 years ]
- Treatment Effectiveness: Need for tracheostomy [ Time Frame: 3 years ]
- QOL [ Time Frame: 3 years ]Patient quality of life assessment: voice (VHI-10)
- QOL [ Time Frame: 3 years ]Patient quality of life assessment: dysphagia (EAT-10)
- QOL [ Time Frame: 3 years ]Patient quality of life assessment: breathing (COPD dyspnea)
- QOL [ Time Frame: 3 years ]Patient quality of life assessment: general quality of life (SF-12)
- Patient Reported Outcome [ Time Frame: 3 years ]Non-traditional PRO focused on social support
- Patient Reported Outcome [ Time Frame: 3 years ]Non-traditional PRO focused on fear of disease recurrence
- Patient Reported Outcome [ Time Frame: 3 years ]Non-traditional PRO focused on disease anxiety and burden
- Patient Reported Outcome [ Time Frame: 3 years ]Non-traditional PRO focused on participatory decision-making style
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02481817
|Principal Investigator:||Alexander Gelbard, MD||Vanderbilt University Medical Center|
|Study Chair:||David O. Francis, MD, MS||Vanderbilt University Medical Center|