Pulmonary Cystic Fibrosis
Conditions
Keywords
bronchial clearance, cystic fibrosis
Brief summary
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.
Interventions
DEVICESIMEOX
Use without obligation, in autonomous and at home of the bronchial clearance device: SIMEOX
Sponsors
Icadom
Physio-Assist
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Adaptive prospective, randomized, controlled, multicenter trial, with hierarchical sequential analysis.
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Respiratory score of CFQ-R questionnaire | Change from baseline at 3 months | 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. |
| Variation of forced expiratory volume in 1 second (FEV1) | Change from baseline at 3 months | Relative variation of the forced expiratory volume in 1 second (FEV1) in the SIMEOX® treated group versus control group |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Satisfaction assessed by Treatment Satisfaction Questionnaire for Medication(TSQM) questionnaire | After 3 months of use | 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 |
| Acceptability assessed by the number of uses of SIMEOX® device | During 3 months of use | 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. |
| Pulmonary forced vital capacity (FVC) | Change from baseline at 3 months | Relative variation of forced vital capacity in liter in the SIMEOX® treated group versus control group. |
| FEV1/ FVC ratio | Change from baseline at 3 months | Relative variation of FEV1/ FVC ratio in % in the SIMEOX® treated group versus control group. |
| Forced expiratory flow | Change from baseline at 3 months | Relative variation of forced expiratory flow in liter per second in the SIMEOX® treated group versus control group |
| Static lung compliance in Liter | Change from baseline at 3 months | Relative variation of Static lung compliance in liter in the SIMEOX® treated group versus control group |
| Static lung compliance in % of predicted value | Change from baseline at 3 months | Relative variation of Static lung compliance in % of predicted value in the SIMEOX® treated group versus control group |
| Airway resistance | Change from baseline at 3 months | Relative variation of Airway resistance in cmH2O per liter per second in the SIMEOX® treated group versus control group |
| Diffusion capacity for carbon monoxide | Change from baseline at 3 months | Relative variation of Diffusion capacity for carbon monoxide in % in the SIMEOX® treated group versus control group |
| Quality of life assessed by EQ-5D 5L questionnaire : a standardized instrument for measuring generic health status | Change from baseline at 3 months | 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). |
| Total score of CFQ-R questionnaire | Change from baseline at 3 months | 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. |
| Sleep quality assessed by sleep efficiency and fragmentation | Change from baseline at 3 months | Compare the effect of SIMEOX autonomous treatment at home versus usual care on sleep efficiency and fragmentation measured by actigraph device |
| Fatigue assessed by VAS (visual analogue scale) | During 3 months of use | 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 |
| Dyspnea assessed by mMRC (Modified Medical Research Council) score | Change from baseline at 3 months | 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 |
| Exacerbation rate | After 3 months of use | Compare the effect of SIMEOX autonomous treatment at home versus usual care on respiratory exacerbation rate |
| First exacerbation | During 3 months of use | Compare the effect of SIMEOX autonomous treatment at home versus usual care on the time to onset of the first exacerbation. |
| Hospitalization rate | After 3 months of use | Compare the effect of SIMEOX autonomous treatment at home versus usual care on hospitalization rate for respiratory exacerbation |
| Hospitalization duration | After 3 months of use | Compare the effect of SIMEOX autonomous treatment at home versus usual care on hospitalization duration for respiratory exacerbation |
| Antibiotic therapy | After 3 months of use | Compare the effect of SIMEOX autonomous treatment at home versus usual care on antibiotic therapy rate (intravenous or oral) |
| Adherence telemonitoring feasibility | After 3 months of use | 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. |
| Acceptability of SIMEOX-ANDROID application assessed by self-questionnaire AES (acceptability E-SCALE) | After 3 months of use | 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 |
| Physical activity assessed by step counts and moderate-to-vigorous physical activity | Change from baseline at 3 months | 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 |
| Safety of use assessed by incidence of Treatment-Emergent Adverse Events | During 3 months of use | Take note and count all Treatment-Emergent Adverse Events to assess the safe use of SIMEOX® device autonomously at home |
Countries
France
Outcome results
None listed