Follow-up of NIV At Home in Patients with Duchenne Muscular Dystrophy

Recruiting

• Conditions: Duchenne Muscular Dystrophy (DMD)

• Registration Number: NCT06773988
• Lead Sponsor: Universitaire Ziekenhuizen KU Leuven

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-10
• Study First Submitted: 2024-12-04
• Study Start: 2024-12-06
• Study First Submitted QC: 2025-01-08
• Last Update Submitted: 2025-01-08
• Study First Posted: 2025-01-14
• Last Update Posted: 2025-01-14
• Primary Completion: 2027-10-31
• Study Completion: 2027-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 17

Inclusion Criteria

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

Exclusion Criteria

• /

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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	Yearly home polysomnopraphy and Transcutaneous CO2 measurement, for a period of 3 years	The primary endpoint is the feasibility of a home based full-video polysomnography for follow-up of DMD patients on NIV.

Secondary Outcome Measures

Name	Time	Method
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)	3 years
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)	Cost-benifit analysis will be carried out at study completion, after 3 years.	During 3 years, the costs for performing a home based full-video PSG (relocation costs, material, physiotherapist wage) will be weighed against the benifits (patient comfort, relieving hospital ward occupation)
Amount of technical failures, i.e. PSG signals, PtcCO2 measurement, O2 saturation measurement and video monitoring.	Performed immediately after each PSG or PtcCO2 measurement, during a period of 3 years.	Checklist after each PSG
Patient Reported Outcomes: Experience between in-hospital PSG vs PSG at home	The PROM will be completed by the patient at each yearly PSG, during the 3 years of the ongoing study	Both patient and caregiver report on a 10 point likert scale, ranging from totally not to very, how mor comfortable the PSG at home was compared to the hospital PSG.
Sleep quality questionnaire: The Pittsburgh Sleep Quality Index (PSQI)	The PSQI will be completed by the patient at each yearly PSG and at each yearly PtcCO2 measurment, during 3 years.	The Pittsburgh Sleep Quality Index (PSQI) is a widely used self-report questionnaire that assesses sleep quality over a one-month time interval. Each component score of the PSQI ranges from 0 to 3, with 3 indicating the greatest dysfunction or disturbance. The seven component scores are then summed to obtain a global PSQI score, which ranges from 0 to 21. Higher scores indicate poorer sleep quality, with a score greater than 5 suggesting significant sleep difficulties
Home-NIV PROM: S3-NIV	The S3-NIV questionnaire will be completed by the patient at each yearly PSG and at each yearly PTCO2 measurement, during 3 years.	The S3-NIV questionnaire is a short, valid and repeatable self-completed tool for the routine clinical assessment of patients undergoing home NIV. The S3-NIV questionnaire provides clinicians and patients with a simple and reliable tool to assess important domains (symptoms, sleep quality and NIV-related side effects) as a complement to physiological monitoring. The S3-NIV questionnaire has 11 items. Each item is scored using a 5-point Likert scale from "always true" to "never true". The total score and the two sub scores range from 0 to 10 points. Lower scores represent a higher, negative impact of the disease and NIV on the individual.
Daytime sleepiness questionnaire: Epworth Sleepiness Scale (ESS)	The ESS questionnaire will be completed by the patient at each yearly PSG and each yearly PtcCO2 measurement, during 3 years.	The ESS is a self-administered questionnaire with 8 questions. Respondents are asked to rate, on a 4-point scale (0-3), their usual chances of dozing off or falling asleep while engaged in eight different activities.

Trial Locations

Locations (1)

Universitaire Ziekenhuizen KU Leuven; 🇧🇪 Leuven, Vlaams-Brabant, Belgium