Clinical trial NCT04793724: Respiratory Physiotherapy Performed by Simeox In Patients With Primary Ciliary Dyskinesia
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
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
Lead sponsor: University Hospital, Motol
Sponsor class: OTHER
First posted: 2021-03-11
Enrollment: 30
Sex: ALL
Minimum age (years): 4
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention 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)
Primary purpose: SUPPORTIVE_CARE

Brief 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 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.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* inability to undergo the assessment and intervention
* noncompliance and/or nonadherence

Primary outcomes:
measure: Change in Lung Clearance Index
timeFrame: Through study completion, an average of 1 year
Secondary outcomes:
measure: Change in Forced Residual Capacity (FRC)
timeFrame: Through study completion, an average of 1 year
measure: Change in Forced Vital Capacity (FVC)
timeFrame: Through study completion, an average of 1 year
measure: Change in Forced Expiratory Volume in 1 second (FEV1)
timeFrame: Through study completion, an average of 1 year
measure: Change in Maximal Expiratory Flow (MEF25-75)
timeFrame: Through study completion, an average of 1 year
measure: Change in Acinar airway inhomogeneity (Sacin)
timeFrame: Through study completion, an average of 1 year
measure: Change in Conductive airway inhomogeneity (Scond)
timeFrame: Through study completion, an average of 1 year
Other outcomes:
measure: Change in Global Inhomogeneity (GI) Index
timeFrame: Through study completion, an average of 1 year
measure: Change in chest expansion measurement
timeFrame: Through study completion, an average of 1 year
Locations:
city: Praha
country: Czechia
facility: Motol University Hospital
state: Praha 5
Study officials:
affiliation: Department of Rehabilitation and Sports Medicine, 2nd Faculty of Medicine
name: Anna Chmelarova, MD
role: PRINCIPAL_INVESTIGATOR
References: