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Treatment Alternatives in iSGS (NoAAC PR-02 Study) (NoAAC PR-02)

This study is currently recruiting participants.
Verified September 2017 by Alexander Gelbard, MD, Vanderbilt University Medical Center
Sponsor:
ClinicalTrials.gov Identifier:
NCT02481817
First Posted: June 25, 2015
Last Update Posted: September 14, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
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
New York University School of Medicine
Mayo Clinic
University of Miami
University of Rochester
Information provided by (Responsible Party):
Alexander Gelbard, MD, Vanderbilt University Medical Center
  Purpose

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 Intervention
Idiopathic Subglottic Stenosis (iSGS) Procedure: Endoscopic dilation of subglottic stenosis Procedure: Endoscopic resection of the stenosis Procedure: Tracheal Resection

Study Type: Observational
Study Design: 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)

Resource links provided by NLM:


Further study details as provided by Alexander Gelbard, MD, Vanderbilt University Medical Center:

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

Secondary Outcome Measures:
  • 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
Biospecimens collected during routine standard of care from each patient (such as tracheal biopsies obtained during surgery, or surgical specimens generated thru open reconstructive surgeries), will be annotated in the EDC with such data including specimen collection, specimen type (e.g., frozen biopsy tissue; FFPE block), and storage location. We may examine excess tissue obtained at standard of care surgical biopsy. Additionally if blood is collected during standard of care (i.e., medically indicated labs, operative or office-based procedures), we will ask participants to provide a one-time, 30 mL blood specimen for research.

Estimated Enrollment: 1000
Actual Study Start Date: August 28, 2015
Estimated Study Completion Date: June 2019
Estimated Primary Completion Date: June 2018 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
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

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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
All clinically confirmed iSGS patients at the participating institutions are eligible for enrollment.
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.
  Contacts and Locations
Information from the National Library of Medicine

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


Contacts
Contact: Cheryl Kinnard, RN 615-322-0333 cheryl.kinnard@Vanderbilt.Edu
Contact: Kate Von Wahlde, MJ, CCRP 615-322-0333 kate.vonwahlde@Vanderbilt.Edu

  Show 42 Study Locations
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
New York University School of Medicine
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
  More Information

Responsible Party: Alexander Gelbard, MD, Assistant Professor, Department of Otolaryngology, Vanderbilt University Medical Center
ClinicalTrials.gov Identifier: NCT02481817     History of Changes
Other Study ID Numbers: 150917
First Submitted: June 22, 2015
First Posted: June 25, 2015
Last Update Posted: September 14, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
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
Laryngostenosis
Pathological Conditions, Anatomical
Laryngeal Diseases
Respiratory Tract Diseases
Otorhinolaryngologic Diseases
Respiratory System Abnormalities
Congenital Abnormalities