Clinical trial NCT06773988: Follow-up of NIV At Home in Patients with Duchenne Muscular Dystrophy
Official title: Follow-up of NIV At Home in Patients with Duchenne Muscular Dystrophy
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Duchenne Muscular Dystrophy (DMD)
Keywords: Duchenne Muscular Dystrophy
Non-invasive ventilation
Feasibility
Home unattended full-video polysomnography
Transcutaneous capnography
Lead sponsor: Universitaire Ziekenhuizen KU Leuven
Sponsor class: OTHER
First posted: 2025-01-14
Enrollment: 17
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients are in follow-up at the Leuven University City for Sleep/wake disorders

Brief summary: The aim of this study is to verify whether full video home polysomnography (sleep study) with nocturnal transcutaneous CO2 monitoring is feasible in the follow-up of non-invasive ventilation (NIV) in patients with Duchenne muscular dystrophy. The researchers would like to investigate whether they could perform sufficient measurements in the home situation and see if correct decision for further treatment could be made. Furthermore, the researchers will make an analysis on the minimal requirements of measurements to make a correct decision for the treatment of these patients.


Eligibility criteria: Inclusion Criteria:

* Patients with DMD \> 18 years using NIV
* Signing the written informed consent

Exclusion Criteria:

* /

Primary outcomes:
measure: The feasibility of full video home polysomnography (PSG) and follow-up at home in patients with DMD on NIV, identical to the sophisticated PSG in-hospital
timeFrame: Yearly home polysomnopraphy and Transcutaneous CO2 measurement, for a period of 3 years
Secondary outcomes:
measure: Analysis of the minimal equipment to assure a correct follow-up of NIV at home (full video PSG vs PtcCO2) measurement combined with ventilator software data vs ventilator software data vs PtcCO2 measurement)
timeFrame: 3 years
measure: Cost-benefit analysis of follow-up of NIV at home as a preparation to create a file for the Belgian health insurance (registration of time and material)
timeFrame: Cost-benifit analysis will be carried out at study completion, after 3 years.
measure: Amount of technical failures, i.e. PSG signals, PtcCO2 measurement, O2 saturation measurement and video monitoring.
timeFrame: Performed immediately after each PSG or PtcCO2 measurement, during a period of 3 years.
measure: Patient Reported Outcomes: Experience between in-hospital PSG vs PSG at home
timeFrame: The PROM will be completed by the patient at each yearly PSG, during the 3 years of the ongoing study
measure: Sleep quality questionnaire: The Pittsburgh Sleep Quality Index (PSQI)
timeFrame: The PSQI will be completed by the patient at each yearly PSG and at each yearly PtcCO2 measurment, during 3 years.
measure: Home-NIV PROM: S3-NIV
timeFrame: The S3-NIV questionnaire will be completed by the patient at each yearly PSG and at each yearly PTCO2 measurement, during 3 years.
measure: Daytime sleepiness questionnaire: Epworth Sleepiness Scale (ESS)
timeFrame: The ESS questionnaire will be completed by the patient at each yearly PSG and each yearly PtcCO2 measurement, during 3 years.
Other outcomes:
Locations:
city: Leuven
country: Belgium
facility: Universitaire Ziekenhuizen KU Leuven
state: Vlaams-Brabant
Study officials:
References:
citation: Bruyneel M, Ninane V. Unattended home-based polysomnography for sleep disordered breathing: current concepts and perspectives. Sleep Med Rev. 2014 Aug;18(4):341-7. doi: 10.1016/j.smrv.2013.12.002. Epub 2013 Dec 12.
citation: Toussaint M, Chatwin M, Soudon P. Mechanical ventilation in Duchenne patients with chronic respiratory insufficiency: clinical implications of 20 years published experience. Chron Respir Dis. 2007;4(3):167-77. doi: 10.1177/1479972307080697.
citation: Bourke SC. Respiratory involvement in neuromuscular disease. Clin Med (Lond). 2014 Feb;14(1):72-5. doi: 10.7861/clinmedicine.14-1-72. No abstract available.