Airway Collapse in Patients With Mounier-Kuhn Syndrome: Titration With Positive Pressure to Reduce Collapse

• ClinicalTrials.gov Identifier: NCT03101059
• Recruitment Status: Unknown
• First Posted: April 4, 2017
• Last Update Posted: October 26, 2017
• Sponsor: University of Sao Paulo General Hospital
• Information provided by (Responsible Party): Rafael Stelmach, University of Sao Paulo General Hospital

Study Description

Brief Summary:

Mounier-Kuhn syndrome (MKS), or congenital tracheobronchiomegaly, is an entity characterized by dilation of the trachea and bronchi, associated with respiratory infections.The main signs and symptoms are cough, bulging and purulent expectoration, digital clubbing, dyspnoea, and wheezing.Some of these symptoms are believed to be due to excessive collapse of the intra-thoracic trachea and bronchi, resulting in airways obstruction of more than 50% . The purpose of this study is to identify and reduce tracheal collapse.

Condition or disease	Intervention/treatment	Phase
Mounier-Kuhn Syndrome	Procedure: Non invasive ventilation - Continuous Positive Airway Pressure	Not Applicable

Detailed Description:

Mounier-Kuhn syndrome (MKS), or congenital tracheobronchiomegaly, is an entity characterized by dilation of the trachea and bronchi, associated with respiratory infections. The prevalence among patients with respiratory symptoms is 0.4 to 1.6%. Its histological features include the atrophy or absence of longitudinal elastic fibers and smooth muscle cells of the airways, responsible for the structural alterations found, such as tracheobronchiomegaly, the presence of inter cartilaginous diverticula, bulging and dilation of the walls of the trachea and bronchi. The main signs and symptoms are cough, bulging and purulent expectoration, digital clubbing, dyspnoea, and wheezing and wheezing accompanied by recurrent respiratory infection. There may be association with other comorbidities such as gastroesophageal reflux disease, chronic obstructive pulmonary disease, bronchiectasis and obstructive sleep apnea / hypopnea syndrome (OSAS). Some of these symptoms are believed to be due to the tracheobronchial disease present in some patients, defined by excessive collapse of the intrathoracic trachea and bronchi resulting in airways obstruction of more than 50%. The main clinical impact is obstruction to expiratory airflow, with consequent air entrapment, reduction of cough and bronchial hygiene effectiveness, facilitating recurrent respiratory infections. Because it is a rare morbidity and little studied, the specific therapy is not consensual, and the main interventions are extrapolated from other pathologies. The use of non invasive mechanical ventilation (NIMV) with continuous positive airway pressure (CPAP) is reported as an option for treatment, however, there are no randomized studies proving its efficacy. The purpose of this study is to identify and reduce tracheal collapse and bronchi of SMK carriers with the use of positive pressure (CPAP-NIV) and to analyse their repercussion in the small airways.

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 15 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Supportive Care
• Official Title: Evaluation of the Presence of Airway Collapse in Patients With Mounier-Kuhn Syndrome and Titration With Continuous Positive Pressure Through Non-invasive Mechanical Ventilation to Reduce Collapse
• Actual Study Start Date: June 8, 2017
• Estimated Primary Completion Date: September 2018
• Estimated Study Completion Date: April 2019

Arms and Interventions

Arm

Intervention/treatment

Experimental: MKS pax; Munier-Kuhn Syndrome patients

Procedure: Non invasive ventilation - Continuous Positive Airway Pressure; To identify, through bronchoscopy, the prevalence of collapse and whether it is possible to counteract an optimum pressure generated by NIV with CPAP that reduces tracheal and bronchial collapse in patients with SMK; To study the frequency of OSAS in patients with MKS ; Record reversal of collapse with CPAP using chest tomography; To identify the impact of CPAP on the distribution of pulmonary ventilation through the analysis of electrical impedance tomography.

Outcome Measures

Primary Outcome Measures :

1. Percentage of tracheal-bronchial collapse area before and after applied positive pressure through bronchoscopy [ Time Frame: 18 months ]
   Identify the prevalence of collapse and whether it is possible to counteract an optimum pressure generated by NIV with CPAP that reduces tracheal and bronchial collapse in patients with SMK

Secondary Outcome Measures :

1. Apnea+hypopnea (AHI) index in patients with SMK [ Time Frame: 24 months ]
   Study the frequency of OSAHS in patients with SMK;
2. Percentage of tracheal-bronchial collapse area before and after applied positive pressure measure using chest tomography [ Time Frame: 24 months ]
   Record reversal of collapse with CPAP using chest tomography
3. Inspiratory and expiratory lung volumes before and after applied positive pressure measure using electrical impedance tomography. [ Time Frame: 24 months ]
   To identify the impact of CPAP on the distribution of pulmonary ventilation through the analysis of electrical impedance tomography.

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 70 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Has Mounier-Khun Syndrome
• Accept and signed informed consent form

Exclusion Criteria:

• Other morbidity avoiding study procedures

Contacts and Locations

Contacts

Contact: Rafael Stelmach, MD-PHD; 551126615191; rafael.stelmach@incor.usp.br

Contact: Evelise Lima, MD; 551126615191; eveliselima53@gmail.com

Locations

Brazil

Divisao de Pneumologia, Instituto do Coracao, Hospital das Clinicas HCFMUSP, Faculdade de Medicina, Universidade de Sao Paulo; Recruiting

Sao Paulo, Brazil, 05403000

Contact: Rafael Stelmach, MD-PHD 551126615191 rafael.stelmach@incor.usp.br

Contact: Evelise Lima, MD 551126615191 eveliselima53@gmail.com

Sponsors and Collaborators

University of Sao Paulo General Hospital

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Lima E, Nakamura MAM, Genta PR, Rodrigues AJ, Athanazio RA, Rached S, Costa ELV, Stelmach R. Improving Airways Patency and Ventilation Through Optimal Positive Pressure Identified by Noninvasive Mechanical Ventilation Titration in Mounier-Kuhn Syndrome: Protocol for an Interventional, Open-Label, Single-Arm Clinical Trial. JMIR Res Protoc. 2020 Aug 14;9(8):e14786. doi: 10.2196/14786.

• Responsible Party: Rafael Stelmach, Clinical Professor, University of Sao Paulo General Hospital
• ClinicalTrials.gov Identifier: NCT03101059
• Other Study ID Numbers: CAAE64001317400000068
• First Posted: April 4, 2017
• Last Update Posted: October 26, 2017
• Last Verified: October 2017

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Rafael Stelmach, University of Sao Paulo General Hospital:

Tracheal; Bronchial; Tracheobronchiomegaly; Collapse; obstructive sleep apnea syndrome; Computer Tomography; Impedance Tomography

Additional relevant MeSH terms:

Tracheobronchomegaly; Syndrome; Shock; Disease; Pathologic Processes; Bronchial Diseases; Respiratory Tract Diseases; Respiratory System Abnormalities; Tracheal Diseases; Congenital Abnormalities