Clinical Trial: Treatment Alternatives in iSGS (NoAAC PR-02 Study)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

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)

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 Summary:

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
Sponsor: Vanderbilt University Medical Center

Current Primary Outcome:

• Treatment Effectiveness: Time to recurrent procedure [ Time Frame: 3 years ]
• Treatment Effectiveness: Need for tracheostomy [ Time Frame: 3 years ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

• 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

Original Secondary Outcome: Same as current

Information By: Vanderbilt University Medical Center

Dates:
Date Received: June 22, 2015
Date Started: August 28, 2015
Date Completion: June 2019
Last Updated: March 13, 2017
Last Verified: March 2017