Respiratory Physiotherapy Performed by Simeox In Patients With Primary Ciliary Dyskinesia

NCT04793724 | Completed

• 1 other identifier: PCD-375.2/20
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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.

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

Outcome Measures

Primary Outcomes (1)

• Change in Lung Clearance Index

  Ventilation Inhomogenity Assesment

  Through study completion, an average of 1 year

Secondary Outcomes (6)

• Change in Forced Residual Capacity (FRC)

  Through study completion, an average of 1 year

• Change in Forced Vital Capacity (FVC)

  Through study completion, an average of 1 year

• Change in Forced Expiratory Volume in 1 second (FEV1)

  Through study completion, an average of 1 year

• Change in Maximal Expiratory Flow (MEF25-75)

  Through study completion, an average of 1 year

• Change in Acinar airway inhomogeneity (Sacin)

  Through study completion, an average of 1 year

Other Outcomes (2)

• Change in Global Inhomogeneity (GI) Index

  Through study completion, an average of 1 year

• Change in chest expansion measurement

  Through study completion, an average of 1 year

Study Arms (2)

Simeox first

EXPERIMENTAL

Patients who will undergo Simeox intervention first. After three months cross-over to PARI O PEP intervention.

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.

Device: PARI O PEP

Interventions

Simeox DEVICE

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

Simeox first

PARI O PEP DEVICE

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.

PARI O PEP first

Eligibility Criteria

• Age: 4 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• University Hospital, Motol: lead

Study Sites (1)

Motol University Hospital

Prague, Praha 5, 150 06, Czechia

Location

MeSH Terms

Conditions

Ciliary Motility Disorders

Condition Hierarchy (Ancestors)

Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Officials

• Anna Chmelarova, MD

  Department of Rehabilitation and Sports Medicine, 2nd Faculty of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: 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)

Study Record Dates

• First Submitted: February 22, 2021
• First Posted: March 11, 2021
• Study Start: July 15, 2019
• Primary Completion: September 30, 2022
• Study Completion: February 28, 2023
• Last Updated: March 2, 2023

Record last verified: 2023-02

Data Sharing

• IPD Sharing: Will not share

Locations

CZ (1)