Duchenne Muscular Dystrophy (DMD)NCT06773988
Follow-up of NIV At Home in Patients with Duchenne Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy (DMD), run by Universitaire Ziekenhuizen KU Leuven at 1 site in Belgium.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- Monitoring of NIV at home
- How many people
- 17 participants
- Where
- 1 siteBelgium
- Study dates
- 6 Dec 2024to 31 Dec 2027
- How long will I take part?
- 3 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Patients are in follow-up at the Leuven University City for Sleep/wake disorders
Who may be able to join
- Patients with DMD > 18 years using NIV
- Signing the written informed consent
Who cannot join
- /
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Belgium1 site, 1 recruiting
- Recruiting
Universitaire Ziekenhuizen KU Leuven
Leuven, Vlaams-Brabant, 3000
Bart Vrijsen, PhD, 32 16 342522, bart.vrijsen@uzleuven.be
About this study
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.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- Monitoring of NIV at home
- Keywords
- Duchenne Muscular Dystrophy, Non-invasive ventilation, Feasibility, Home unattended full-video polysomnography, Transcutaneous capnography
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Monitoring of NIV at homeDiagnostic test
Monitoring of NIV at home
| Study group | Role | What they receive |
|---|---|---|
| Adult patients with Duchenne using NIV in follow-up at the Leuven University Center for Sleep/wake | Patients with Duchenne Muscular Dystrophy (DMD) on non-invasive ventilation (NIV) who need a yearly follow up by polysomnopgraphy Receives: Diagnostic Test: Monitoring of NIV at home |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
The primary endpoint is the feasibility of a home based full-video polysomnography for follow-up of DMD patients on NIV.
Measured: Yearly home polysomnopraphy and Transcutaneous CO2 measurement, for a period of 3 years
Secondary outcomes (7)
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)
Measured: 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)
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)
Measured: Cost-benifit analysis will be carried out at study completion, after 3 years.
Amount of technical failures, i.e. PSG signals, PtcCO2 measurement, O2 saturation measurement and video monitoring.
Checklist after each PSG
Measured: Performed immediately after each PSG or PtcCO2 measurement, during a period of 3 years.
Patient Reported Outcomes: Experience between in-hospital PSG vs PSG at home
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.
Measured: The PROM will be completed by the patient at each yearly PSG, during the 3 years of the ongoing study
Sleep quality questionnaire: The Pittsburgh Sleep Quality Index (PSQI)
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
Measured: The PSQI will be completed by the patient at each yearly PSG and at each yearly PtcCO2 measurment, during 3 years.
Home-NIV PROM: S3-NIV
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.
Measured: The S3-NIV questionnaire will be completed by the patient at each yearly PSG and at each yearly PTCO2 measurement, during 3 years.
Daytime sleepiness questionnaire: Epworth Sleepiness Scale (ESS)
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.
Measured: The ESS questionnaire will be completed by the patient at each yearly PSG and each yearly PtcCO2 measurement, during 3 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2025
- Study start6 Dec 2024
- Primary completion31 Oct 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies of the same condition, open and under way.
- More trials run by Universitaire Ziekenhuizen KU LeuvenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 17 participants (estimated)
- Target duration
- 3 Years
Sponsor and investigators
- Lead sponsor
- Universitaire Ziekenhuizen KU Leuven
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06773988
- Sponsor's protocol ID
- S68844
- Status last verified
- Oct 2024
- First submitted
- 4 Dec 2024
- Last updated
- 14 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 24388970Toussaint 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.
PubMed 17711917Bourke 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.
PubMed 24532751
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Follow-up of NIV At Home in Patients with Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT06773988
- Acronym / sponsor ID
- S68844
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- Duchenne Muscular Dystrophy, Non-invasive ventilation, Feasibility, Home unattended full-video polysomnography, Transcutaneous capnography
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 17 participants (estimated)
- Lead sponsor
- Universitaire Ziekenhuizen KU LeuvenUniversity, hospital or other
- Study start
- 6 Dec 2024 (actual)
- Primary completion
- 31 Oct 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 14 Jan 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 3 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Adult patients with Duchenne using NIV in follow-up at the Leuven University Center for Sleep/wake | – | Diagnostic Test: Monitoring of NIV at homePatients with Duchenne Muscular Dystrophy (DMD) on non-invasive ventilation (NIV) who need a yearly follow up by polysomnopgraphy |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients are in follow-up at the Leuven University City for Sleep/wake disorders
Key inclusion criteria (2 listed)
- Patients with DMD > 18 years using NIV
- Signing the written informed consent
Key exclusion criteria (1 listed)
- /
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Monitoring of NIV at homeMonitoring of NIV at home | Diagnostic test | Adult patients with Duchenne using NIV in follow-up at the Leuven University Center for Sleep/wake |
Primary outcome
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
Time frame: 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.
7 secondary outcomes
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)
Time frame: 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)
Time frame: Cost-benifit analysis will be carried out at study completion, after 3 years.
Amount of technical failures, i.e. PSG signals, PtcCO2 measurement, O2 saturation measurement and video monitoring.
Time frame: Performed immediately after each PSG or PtcCO2 measurement, during a period of 3 years.
Patient Reported Outcomes: Experience between in-hospital PSG vs PSG at home
Time frame: The PROM will be completed by the patient at each yearly PSG, during the 3 years of the ongoing study
Sleep quality questionnaire: The Pittsburgh Sleep Quality Index (PSQI)
Time frame: The PSQI will be completed by the patient at each yearly PSG and at each yearly PtcCO2 measurment, during 3 years.
Home-NIV PROM: S3-NIV
Time frame: The S3-NIV questionnaire will be completed by the patient at each yearly PSG and at each yearly PTCO2 measurement, during 3 years.
Daytime sleepiness questionnaire: Epworth Sleepiness Scale (ESS)
Time frame: The ESS questionnaire will be completed by the patient at each yearly PSG and each yearly PtcCO2 measurement, during 3 years.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2025.
- Background: PubMed 24388970Bruyneel 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.
- Background: PubMed 17711917Toussaint 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.
- Background: PubMed 24532751Bourke 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy (DMD), run by Universitaire Ziekenhuizen KU Leuven at 1 site in Belgium.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Dries Testelmans, MD PHD (+3216342522, dries.testelmans@uzleuven.be), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome 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 (time frame: Yearly home polysomnopraphy and Transcutaneous CO2 measurement, for a period of 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06773988, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jan 2025.