Home Airway Clearance in CF Patients

Study Overview
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Brief Summary
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Regular bronchial clearance is essential in patients with cystic fibrosis for their bronchial health. SIMEOX® (Physio-Assist, Aix en Provence, France) is an innovative medical device for the drainage of the bronchial tree. By changing the rheological properties of mucus, SIMEOX® helps to mobilize secretions and assists their transport to the upper airways. This technology is based on fundamental research on bronchial mucus rheology. At the present time, SIMEOX® device is mainly used over a short period at the time or after an exacerbation in healthcare structures (hospitals, physiotherapy practices, post-care, and rehabilitation units, etc…). The clinical effects observed in the short term encourages long-term autonomous use by the patients themselves. The overall objective of this study is to evaluate the efficiency and acceptability of SIMEOX® used at home by the patient himself for bronchial clearance in patients with cystic fibrosis.

Official Title
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Efficiency and Acceptability of SIMEOX® Used Autonomously at Home for Bronchial Clearance in Patients With Cystic Fibrosis: Prospective, Randomized Controlled Study

Conditions
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Pulmonary Cystic Fibrosis

Intervention / Treatment
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* Device: SIMEOX


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patient diagnosed with Cystic fibrosis Stabilization of the disease, defined by an at least 4 weeks period since the end of an exacerbation, stabilization will be let to investigator's judgment. Age >12 Requiring at least 1 bronchial clearing session each week (regardless of the technique) Patient (or patient's legal representative if applicable), capable to read and understand the procedure, and capable to express consent for the study protocol. Exclusion Criteria: Severe pneumothorax or hemoptysis (more than 30ml per 24 hours) within 6 weeks before the inclusion day. Patient placed on a transplant waiting list Any contraindication to an instrumental bronchial clearance technique Patients already own and use SIMEOX at home Unavailable patient or patient wishing to move to a different region within three months after inclusion. Patient currently participating or having participated to another interventional clinical research within a month prior inclusion date, that may impact the study, this impact is left to the investigator's judgment. Persons referred by the French Public Health Code to Articles L1121-5 to L1121-8 (includes all protected persons: pregnant women (confirmed by measurement of the serum human chorionic gonadotropin level for any woman wishing to enter the protocol and under childbearing age < 60), parturient woman, breastfeeding mother, everyone deprived of his liberty by judicial or administrative decision, everyone subject to a legal protection measure. Initiation of treatment with a CFTR modulator within the last 3 months

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Adaptive prospective, randomized, controlled, multicenter trial, with hierarchical sequential analysis.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: SIMEOX<br>Use the device for 3 months in addition to usual care | Device: SIMEOX<br>* Use without obligation, in autonomous and at home of the bronchial clearance device: SIMEOX<br>|
| No Intervention: Control<br>Usual care | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Respiratory score of CFQ-R questionnaire | Relative variation in respiratory score of the CFQ-R questionnaire (Cystic Fibrosis Questionnaire -revised version) in the SIMEOX® treated group versus control group. Score range from 0 to 100, with higher score indicating better health. | Change from baseline at 3 months | 
| Variation of forced expiratory volume in 1 second (FEV1) | Relative variation of the forced expiratory volume in 1 second (FEV1) in the SIMEOX® treated group versus control group | Change from baseline at 3 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Total score of CFQ-R questionnaire | Relative variation in the total score of the CFQ-R questionnaire (Cystic Fibrosis Questionnaire -revised version) in the SIMEOX® treated group versus control group.Score range from 0 to 100, with higher score indicating better health. | Change from baseline at 3 months | 
| Safety of use assessed by incidence of Treatment-Emergent Adverse Events | Take note and count all Treatment-Emergent Adverse Events to assess the safe use of SIMEOX® device autonomously at home | During 3 months of use | 
| Satisfaction assessed by Treatment Satisfaction Questionnaire for Medication(TSQM) questionnaire | Total score into a value ranging from the worst satisfaction 0 to the best satisfaction 100 of the TSQM questionnaire in the SIMEOX® treated group to assess the satisfaction | After 3 months of use | 
| Acceptability assessed by the number of uses of SIMEOX® device | Count the self-declared number of uses of the device SIMEOX by the patient in the SIMEOX® treated group to assess the acceptability of the device. | During 3 months of use | 
| Pulmonary forced vital capacity (FVC) | Relative variation of forced vital capacity in liter in the SIMEOX® treated group versus control group. | Change from baseline at 3 months | 
| FEV1/ FVC ratio | Relative variation of FEV1/ FVC ratio in % in the SIMEOX® treated group versus control group. | Change from baseline at 3 months | 
| Forced expiratory flow | Relative variation of forced expiratory flow in liter per second in the SIMEOX® treated group versus control group | Change from baseline at 3 months | 
| Static lung compliance in Liter | Relative variation of Static lung compliance in liter in the SIMEOX® treated group versus control group | Change from baseline at 3 months | 
| Static lung compliance in % of predicted value | Relative variation of Static lung compliance in % of predicted value in the SIMEOX® treated group versus control group | Change from baseline at 3 months | 
| Airway resistance | Relative variation of Airway resistance in cmH2O per liter per second in the SIMEOX® treated group versus control group | Change from baseline at 3 months | 
| Diffusion capacity for carbon monoxide | Relative variation of Diffusion capacity for carbon monoxide in % in the SIMEOX® treated group versus control group | Change from baseline at 3 months | 
| Quality of life assessed by EQ-5D 5L questionnaire : a standardized instrument for measuring generic health status | Relative variation of the scores of EQ-5D (a standardized instrument for measuring generic health status) questionnaire in the SIMEOX® treated group versus control group to asess the generic quality of life.Each 5 descriptive items have a value ranging from 1(best) to 5 (worst) and the visual analogic scale item should be scored from 0 (worst health) to 100 (best health). | Change from baseline at 3 months | 
| Physical activity assessed by step counts and moderate-to-vigorous physical activity | Compare the effect of SIMEOX autonomous treatment at home versus usual care on step counts and moderate-to-vigorous physical activity measured by actigraph device | Change from baseline at 3 months | 
| Sleep quality assessed by sleep efficiency and fragmentation | Compare the effect of SIMEOX autonomous treatment at home versus usual care on sleep efficiency and fragmentation measured by actigraph device | Change from baseline at 3 months | 
| Fatigue assessed by VAS (visual analogue scale) | Compare the effect of SIMEOX autonomous treatment at home versus usual care on fatigue experienced after a drainage session assessed by the VAS score between 0 and 10 | During 3 months of use | 
| Dyspnea assessed by mMRC (Modified Medical Research Council) score | Compare the effect of SIMEOX autonomous treatment at home versus usual care on mMRC (Modified Medical Research Council) score into a value ranging from the least severe dyspnea 0 to the most serious dyspnea 4 | Change from baseline at 3 months | 
| Exacerbation rate | Compare the effect of SIMEOX autonomous treatment at home versus usual care on respiratory exacerbation rate | After 3 months of use | 
| First exacerbation | Compare the effect of SIMEOX autonomous treatment at home versus usual care on the time to onset of the first exacerbation. | During 3 months of use | 
| Hospitalization rate | Compare the effect of SIMEOX autonomous treatment at home versus usual care on hospitalization rate for respiratory exacerbation | After 3 months of use | 
| Hospitalization duration | Compare the effect of SIMEOX autonomous treatment at home versus usual care on hospitalization duration for respiratory exacerbation | After 3 months of use | 
| Antibiotic therapy | Compare the effect of SIMEOX autonomous treatment at home versus usual care on antibiotic therapy rate (intravenous or oral) | After 3 months of use | 
| Adherence telemonitoring feasibility | Adherence telemonitoring feasibility is assessed by comparing self-declared data (number of treatment session) and electronically transmitted data. Electronically transmitted data will be transmitted by the SIMEOX-ANDROID application to an electronic secured platform. In the SIMEOX® treated group only. | After 3 months of use | 
| Acceptability of SIMEOX-ANDROID application assessed by self-questionnaire AES (acceptability E-SCALE) | Total score into a value ranging from the worst acceptability 6 to the best acceptability 30 of the self-questionnaire AES (acceptability E-SCALE) in the SIMEOX® treated group to assess the SIMEOX-ANDROID application acceptability | After 3 months of use | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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bronchial clearance, cystic fibrosis