Mounier-Kuhn SyndromeNCT03101059
Airway Collapse in Patients With Mounier-Kuhn Syndrome: Titration With Positive Pressure to Reduce Collapse
A study testing Non invasive ventilation - Continuous Positive Airway Pressure in people with Mounier-Kuhn Syndrome, run by University of Sao Paulo General Hospital at 1 site in Brazil.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Mounier-Kuhn Syndrome
- What is being tested?
- Non invasive ventilation - Continuous Positive Airway Pressure
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteBrazil
- Study dates
- 8 Jun 2017to Apr 2019
- Phase
- Interventional
- 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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Has Mounier-Khun Syndrome
- Accept and signed informed consent form
Who cannot join
- Other morbidity avoiding study procedures
Trial locations and contacts
1 site in 1 country.
Brazil1 site, 1 recruiting
- Recruiting
Divisao de Pneumologia, Instituto do Coracao, Hospital das Clinicas HCFMUSP, Faculdade de Medicina, Universidade de Sao Paulo
São Paulo, 05403000
Rafael Stelmach, MD-PHD, 551126615191, rafael.stelmach@incor.usp.br
Evelise Lima, MD, 551126615191, eveliselima53@gmail.com
About this study
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
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.
Read the 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.
- Conditions
- Mounier-Kuhn Syndrome
- Being tested
- Non invasive ventilation - Continuous Positive Airway Pressure
- Keywords
- Tracheal, Bronchial, Tracheobronchiomegaly, Collapse, obstructive sleep apnea syndrome, Computer Tomography, Impedance Tomography
- Medical subject headings
- Tracheobronchomegaly, Unconsciousness, Sleep Apnea, Obstructive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Non invasive ventilation - Continuous Positive Airway PressureProcedure or surgery
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.
| Study group | Role | What they receive |
|---|---|---|
| MKS pax | Experimental | Munier-Kuhn Syndrome patients Receives: Procedure: Non invasive ventilation - Continuous Positive Airway Pressure |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of tracheal-bronchial collapse area before and after applied positive pressure through bronchoscopy
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
Measured: 18 months
Secondary outcomes (3)
Apnea+hypopnea (AHI) index in patients with SMK
Study the frequency of OSAHS in patients with SMK;
Measured: 24 months
Percentage of tracheal-bronchial collapse area before and after applied positive pressure measure using chest tomography
Record reversal of collapse with CPAP using chest tomography
Measured: 24 months
Inspiratory and expiratory lung volumes before and after applied positive pressure measure using electrical impedance tomography.
To identify the impact of CPAP on the distribution of pulmonary ventilation through the analysis of electrical impedance tomography.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Apr 2017
- Study start8 Jun 2017
- Primary completionSep 2018 (estimated)
- Study completionApr 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Mounier-Kuhn SyndromeStudies of the same condition, open and under way.
- More trials run by University of Sao Paulo General HospitalThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Sao Paulo General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Rafael Stelmach, University of Sao Paulo General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03101059
- Sponsor's protocol ID
- CAAE64001317400000068
- Status last verified
- Oct 2017
- First submitted
- 23 Mar 2017
- Last updated
- 26 Oct 2017
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Lima E, Nakamura MAM, Rodrigues AJ, Costa ELV, Rached SZ, Sawamura MVY, Genta PR, Athanazio RA. Effects of positive pressure on tracheobronchomalacia and collapse reversal. BMC Pulm Med. 2026 May 30. doi: 10.1186/s12890-026-04359-z. Online ahead of print.
PubMed 42216039Lima 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.
PubMed 32795996
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03101059
- Acronym / sponsor ID
- CAAE64001317400000068
- Conditions
- Mounier-Kuhn Syndrome
- Keywords
- Tracheal, Bronchial, Tracheobronchiomegaly, Collapse, obstructive sleep apnea syndrome, Computer Tomography, Impedance Tomography
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- University of Sao Paulo General HospitalUniversity, hospital or other
- Study start
- 8 Jun 2017 (actual)
- Primary completion
- Sep 2018 (estimated)
- Study completion
- Apr 2019 (estimated)
- First posted
- 4 Apr 2017 (actual)
- 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.
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.
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
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MKS pax | Experimental | Procedure: Non invasive ventilation - Continuous Positive Airway PressureMunier-Kuhn Syndrome patients |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Has Mounier-Khun Syndrome
- Accept and signed informed consent form
Key exclusion criteria (1 listed)
- Other morbidity avoiding study procedures
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 |
|---|---|---|
| Non invasive ventilation - Continuous Positive Airway PressureTo 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. | Procedure or surgery | MKS pax |
Primary outcome
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
3 secondary outcomes
Apnea+hypopnea (AHI) index in patients with SMK
Time frame: 24 months
Percentage of tracheal-bronchial collapse area before and after applied positive pressure measure using chest tomography
Time frame: 24 months
Inspiratory and expiratory lung volumes before and after applied positive pressure measure using electrical impedance tomography.
Time frame: 24 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2017.
- Derived from this study: PubMed 42216039Lima E, Nakamura MAM, Rodrigues AJ, Costa ELV, Rached SZ, Sawamura MVY, Genta PR, Athanazio RA. Effects of positive pressure on tracheobronchomalacia and collapse reversal. BMC Pulm Med. 2026 May 30. doi: 10.1186/s12890-026-04359-z. Online ahead of print.
- Derived from this study: PubMed 32795996Lima 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.
- Individual participant dataSharing planned: No
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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Non invasive ventilation - Continuous Positive Airway Pressure in people with Mounier-Kuhn Syndrome, run by University of Sao Paulo General Hospital at 1 site in Brazil.
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 1 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: status not confirmed, as of the sponsor's last update on 26 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Brazil. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Rafael Stelmach, MD-PHD (551126615191, rafael.stelmach@incor.usp.br), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of tracheal-bronchial collapse area before and after applied positive pressure through bronchoscopy (time frame: 18 months). 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 NCT03101059, which also lists 2 linked publications.
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 26 Oct 2017; AzTrials last refreshed its copy on 7 Nov 2026.