Primary Ciliary DyskinesiaNCT04793724
Respiratory Physiotherapy Performed by Simeox In Patients With Primary Ciliary Dyskinesia
A study testing Simeox and PARI O PEP in people with Primary Ciliary Dyskinesia, run by University Hospital, Motol at 1 site in Czechia.
Trial summary
Primary ciliary dyskinesia (PCD) is characterized by impaired airway clearance and mucus stagnation. This results in recurrent upper and lower respiratory tract infections often leading to chronic inflammation and, if not treated early and properly, to irreversible functional and structural changes of the respiratory… Read more about this study
- Who is it for?
- Ages 4 to 18 years
- Conditions the study is for
- Primary Ciliary Dyskinesia
- What is being tested?
- Simeox and PARI O PEP
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteCzechia
- Study dates
- 15 Jul 2019to 28 Feb 2023
- 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 4 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- diagnosis of PCD confirmed by Transmission Electron Microscopy (TEM) analysis of ciliary ultrastructure showing clear structural axonemal defect and/or positive genetic testing for one (autosomal dominant) or two (autosomal recesive) PCD-causing mutations;
- age range 4 - 18 years;
- established chest physiotherapy with PARI O PEP
Who cannot join
- inability to undergo the assessment and intervention
- noncompliance and/or nonadherence
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Evaluation of Short-term Effect of Respiratory Physiotherapy Performed by Simeox on Lung Function in Patients With Primary Ciliary Dyskinesia - Randomised Cross-over Interventional Study
Primary ciliary dyskinesia (PCD) is characterized by impaired airway clearance and mucus stagnation. This results in recurrent upper and lower respiratory tract infections often leading to chronic inflammation and, if not treated early and properly, to irreversible functional and structural changes of the respiratory tract. As there is no causal treatment of PCD yet, airway clearance techniques (ACT) provide fundamental care for these patients.
Simeox is a new airway clearance device, recently developed by the French company PhysioAssist. This technology is based on pneumatic vibrations generated by the device itself. Vibrations are induced by rapidly alternating between atmospheric and negative pressure as the patient exhales, providing the most effective clearance of mucus from the lungs. Vibrations of different intensity and frequency are known to alter the rheological properties of mucus in the airways, whilst the negative pressure during exhalation helps to mobilise and drain the mucus to the central bronchi.
Although there have not yet been any evidence based papers published clarifying the effect of Simeox specifically in patients with PCD, using up-to-date information, experience, and positive feedback from our patients, we assume that there could be a significant benefit for the effectiveness of ACT.
Read the detailed description
In this study the effectiveness of ACT will be evaluated based on the short-term effect(s) of respiratory physiotherapy with Simeox on changes in lung function and thoracic expansibility paediatric patients with PCD.
An interventional randomised non-inferiority crossover trial will be conducted in Motol University Hospital; the project was developed by the multidisciplinary team (MDT) of the Division of Paediatric Pulmonology of the Department of Paediatrics, 2nd Faculty of Medicine, Charles University in Prague and Motol University Hospital; and the Department of Rehabilitation and Sports Medicine, 2nd Faculty of Medicine, Charles University in Prague and Motol University Hospital.
Eligible patients will be randomly assigned into 2 arms - "Simeox-first group" and "Pari-O-PEP first" group. Randomisation will be done using computer generated code for each patient. The protocol will include 3 study visits - K0, K1 and K2 planned 3 months apart. After 6 months (second visit - K1), each patient will be switched to the other interventional group (crossed over). The first session (K0) includes an initial lung function assessment using spirometry, nitrogen-Multiple Breath Washout test (N2-MBW), Electrical Impedance Tomography (EIT), 6 Minute Walk Test (6 MWT), respiratory amplitudes (RA), and a theoretical introduction to therapy with the Simeox device. The second (K1) and third (K2) sessions will be interventional, including a respiratory physiotherapy session in between each measurement, excluding 6 MWT, which will be measured just once per session.
According to randomisation criteria, participants will initially undergo either physiotherapist-administered 20-minute ACT session using the Simeox device or 30-minute therapy with PARI O-PEP. In accordance with the rules for crossover study design, each patient will undergo therapy with both ACT devices, ideally within six-month duration of the study. These interventions will take place in between each measurement (spirometry, MBW, EIT, RA) during the second (K1) and the third (K2) session.
- Conditions
- Primary Ciliary Dyskinesia
- Being tested
- Simeox, PARI O PEP
- Keywords
- primary ciliary dyskinesia, respiratory physiotherapy, airway clearance techniques, lung function testing, ventilation inhomogeneity, nitrogen multiple breath washout test, spirometry, electric impedance tomography
- Medical subject headings
- Ciliary Motility Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SimeoxDevice
A respiratory physiotherapy session with the Simeox device will be performed in accordance with the official PhysioAssist recommendations, which are;
- clearance of the upper airways before the physiotherapy session
- correct position of the mouthpiece in patient's mouth, with the lips placed on the thinnest part of the mouthpiece and the tongue positioned underneath the mouthpiece
- slow nasal inhalation without too much recruitment of the inspiratory residual volume (IRV) and an exhalation with real deflation of the thorax
- step-by-step shifting of the patient's tidal volume towards the expiratory residual volume (ERV), in order to target the most distal regions of the lungs
- comfortable, relaxed sitting position with straightened spine
- controlled cough, which will be encouraged only when it is productive
PARI O PEPDevice
ACT with PARI O-PEP will be performed in an upright sitting position, using four positions of the device for the most effective clearance of mucus from the lungs. These positions will be:
- horizontal;
- low;
- transition of the device from the low position to the horizontal position;
- upside down.
The mouthpiece will be placed between the patient's teeth as per standard procedure, and enclosed properly within the lips. Patients will perform slow and deep inhalation via the nose, then hold their breath for about 1 to 2 seconds at the end of inhalation. Exhalation will be performed slowly and completely into the PARI O-PEP through the patient's mouth. At the end of the session, the patient will perform "huffing" with the PARI O-PEP device in the upside down position in order to maintain open airways, and, if possible, expectoration sputum.
| Study group | Role | What they receive |
|---|---|---|
| Simeox first | Experimental | Patients who will undergo Simeox intervention first. After three months cross-over to PARI O PEP intervention. Receives: Device: Simeox |
| PARI O PEP first | Active comparator | Patients who will undergo PARI O PEP intervention first. After three months cross-over to Simeox intervention. Receives: Device: PARI O PEP |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Lung Clearance Index
Ventilation Inhomogenity Assesment
Measured: Through study completion, an average of 1 year
Secondary outcomes (6)
Change in Forced Residual Capacity (FRC)
Volume of air present in the lungs at the end of passive expiration, in Litres [L].
Measured: Through study completion, an average of 1 year
Change in Forced Vital Capacity (FVC)
Volume of air that can forcibly be blown out after full inspiration, in Litres [L]
Measured: Through study completion, an average of 1 year
Change in Forced Expiratory Volume in 1 second (FEV1)
Volume of air exhaled in the first second during forced exhalation after full inspiration, in Litres per second [L/s]
Measured: Through study completion, an average of 1 year
Change in Maximal Expiratory Flow (MEF25-75)
Volume of air where the certain amount (25-75%) of Forced Vital Capacity (FVC) remains to be exhaled, in Litres [L]
Measured: Through study completion, an average of 1 year
Change in Acinar airway inhomogeneity (Sacin)
MBW indices reflecting ventilation inhomogeneity in the acinar airway region, in Litres [L]
Measured: Through study completion, an average of 1 year
Change in Conductive airway inhomogeneity (Scond)
MBW indices reflecting ventilation inhomogeneity in the conductive airway region, in Litres [L]
Measured: Through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Mar 2021
- Study start15 Jul 2019
- Primary completion30 Sep 2022
- Study completion28 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Ciliary DyskinesiaStudies of the same condition, open and under way.
- 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
- Randomised
- Intervention model
- Crossover
- Model description
- First session - initial medical and physiotherapy assessment Second session - medical and physiotherapy assessment, respiratory physiotherapy with Simeox/PARI O PEP (according the randomization) Third session - medical and physiotherapy assessment, respiratory physiotherapy with Simeox/PARI O PEP (according the randomization)
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Motol
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Anna Chmelarova, Mgr, University Hospital, Motol
- Investigators
- Anna Chmelarova, MD, Principal investigator, Department of Rehabilitation and Sports Medicine, 2nd Faculty of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04793724
- Sponsor's protocol ID
- PCD-375.2/20
- Status last verified
- Feb 2023
- First submitted
- 22 Feb 2021
- Last updated
- 2 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Short-term Effect of Respiratory Physiotherapy Performed by Simeox on Lung Function in Patients With Primary Ciliary Dyskinesia - Randomised Cross-over Interventional Study
- ClinicalTrials.gov ID
- NCT04793724
- Acronym / sponsor ID
- PCD-375.2/20
- Conditions
- Primary Ciliary Dyskinesia
- Keywords
- primary ciliary dyskinesia, respiratory physiotherapy, airway clearance techniques, lung function testing, ventilation inhomogeneity, nitrogen multiple breath washout test, spirometry, electric impedance tomography
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- University Hospital, MotolUniversity, hospital or other
- Study start
- 15 Jul 2019 (actual)
- Primary completion
- 30 Sep 2022 (actual)
- Study completion
- 28 Feb 2023 (actual)
- First posted
- 11 Mar 2021 (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.
Primary ciliary dyskinesia (PCD) is characterized by impaired airway clearance and mucus stagnation. This results in recurrent upper and lower respiratory tract infections often leading to chronic inflammation and, if not treated early and properly, to irreversible functional and structural changes of the respiratory tract. As there is no causal treatment of PCD yet, airway clearance techniques (ACT) provide fundamental care for these patients.
Simeox is a new airway clearance device, recently developed by the French company PhysioAssist. This technology is based on pneumatic vibrations generated by the device itself. Vibrations are induced by rapidly alternating between atmospheric and negative pressure as the patient exhales, providing the most effective clearance of mucus from the lungs. Vibrations of different intensity and frequency are known to alter the rheological properties of mucus in the airways, whilst the negative pressure during exhalation helps to mobilise and drain the mucus to the central bronchi.
Although there have not yet been any evidence based papers published clarifying the effect of Simeox specifically in patients with PCD, using up-to-date information, experience, and positive feedback from our patients, we assume that there could be a significant benefit for the effectiveness of ACT.
Detailed description
In this study the effectiveness of ACT will be evaluated based on the short-term effect(s) of respiratory physiotherapy with Simeox on changes in lung function and thoracic expansibility paediatric patients with PCD.
An interventional randomised non-inferiority crossover trial will be conducted in Motol University Hospital; the project was developed by the multidisciplinary team (MDT) of the Division of Paediatric Pulmonology of the Department of Paediatrics, 2nd Faculty of Medicine, Charles University in Prague and Motol University Hospital; and the Department of Rehabilitation and Sports Medicine, 2nd Faculty of Medicine, Charles University in Prague and Motol University Hospital.
Eligible patients will be randomly assigned into 2 arms - "Simeox-first group" and "Pari-O-PEP first" group. Randomisation will be done using computer generated code for each patient. The protocol will include 3 study visits - K0, K1 and K2 planned 3 months apart. After 6 months (second visit - K1), each patient will be switched to the other interventional group (crossed over). The first session (K0) includes an initial lung function assessment using spirometry, nitrogen-Multiple Breath Washout test (N2-MBW), Electrical Impedance Tomography (EIT), 6 Minute Walk Test (6 MWT), respiratory amplitudes (RA), and a theoretical introduction to therapy with the Simeox device. The second (K1) and third (K2) sessions will be interventional, including a respiratory physiotherapy session in between each measurement, excluding 6 MWT, which will be measured just once per session.
According to randomisation criteria, participants will initially undergo either physiotherapist-administered 20-minute ACT session using the Simeox device or 30-minute therapy with PARI O-PEP. In accordance with the rules for crossover study design, each patient will undergo therapy with both ACT devices, ideally within six-month duration of the study. These interventions will take place in between each measurement (spirometry, MBW, EIT, RA) during the second (K1) and the third (K2) session.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- First session - initial medical and physiotherapy assessment Second session - medical and physiotherapy assessment, respiratory physiotherapy with Simeox/PARI O PEP (according the randomization) Third session - medical and physiotherapy assessment, respiratory physiotherapy with Simeox/PARI O PEP (according the randomization)
| Arm | Type | Receives |
|---|---|---|
| Simeox first | Experimental | Device: SimeoxPatients who will undergo Simeox intervention first. After three months cross-over to PARI O PEP intervention. |
| PARI O PEP first | Active comparator | Device: PARI O PEPPatients who will undergo PARI O PEP intervention first. After three months cross-over to Simeox intervention. |
Who may qualify
- Ages
- 4 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- diagnosis of PCD confirmed by Transmission Electron Microscopy (TEM) analysis of ciliary ultrastructure showing clear structural axonemal defect and/or positive genetic testing for one (autosomal dominant) or two (autosomal recesive) PCD-causing mutations;
- age range 4 - 18 years;
- established chest physiotherapy with PARI O PEP
Key exclusion criteria (2 listed)
- inability to undergo the assessment and intervention
- noncompliance and/or nonadherence
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 |
|---|---|---|
| SimeoxA respiratory physiotherapy session with the Simeox device will be performed in accordance with the official PhysioAssist recommendations, which are; * clearance of the upper airways before the physiotherapy session * correct position of the mouthpiece in patient's mouth, with the lips placed on the thinnest part of the mouthpiece and the tongue positioned underneath the mouthpiece * slow nasal inhalation without too much recruitment of the inspiratory residual volume (IRV) and an exhalation with real deflation of the thorax * step-by-step shifting of the patient's tidal volume towards the expiratory residual volume (ERV), in order to target the most distal regions of the lungs * comfortable, relaxed sitting position with straightened spine * controlled cough, which will be encouraged only when it is productive | Device | Simeox first |
| PARI O PEPACT with PARI O-PEP will be performed in an upright sitting position, using four positions of the device for the most effective clearance of mucus from the lungs. These positions will be: * horizontal; * low; * transition of the device from the low position to the horizontal position; * upside down. The mouthpiece will be placed between the patient's teeth as per standard procedure, and enclosed properly within the lips. Patients will perform slow and deep inhalation via the nose, then hold their breath for about 1 to 2 seconds at the end of inhalation. Exhalation will be performed slowly and completely into the PARI O-PEP through the patient's mouth. At the end of the session, the patient will perform "huffing" with the PARI O-PEP device in the upside down position in order to maintain open airways, and, if possible, expectoration sputum. | Device | PARI O PEP first |
Primary outcome
Change in Lung Clearance Index
Time frame: Through study completion, an average of 1 year
Ventilation Inhomogenity Assesment
6 secondary outcomes
Change in Forced Residual Capacity (FRC)
Time frame: Through study completion, an average of 1 year
Change in Forced Vital Capacity (FVC)
Time frame: Through study completion, an average of 1 year
Change in Forced Expiratory Volume in 1 second (FEV1)
Time frame: Through study completion, an average of 1 year
Change in Maximal Expiratory Flow (MEF25-75)
Time frame: Through study completion, an average of 1 year
Change in Acinar airway inhomogeneity (Sacin)
Time frame: Through study completion, an average of 1 year
Change in Conductive airway inhomogeneity (Scond)
Time frame: Through study completion, an average of 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2023.
- Individual participant dataSharing planned: No
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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.
Anna Chmelarova, MD principal investigator
Department of Rehabilitation and Sports Medicine, 2nd Faculty of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Simeox and PARI O PEP in people with Primary Ciliary Dyskinesia, run by University Hospital, Motol at 1 site in Czechia.
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 3 inclusion and 2 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: finished, as of the sponsor's last update on 2 Mar 2023. 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 Czechia. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Lung Clearance Index (time frame: Through study completion, an average of 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 2 Mar 2023.