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

NCT06773988 | Recruiting

• 1 other identifier: S68844
• Study Type: observational
• Target: 17
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

Duchenne Muscular Dystrophy; Non-invasive ventilation; Feasibility; Home unattended full-video polysomnography; Transcutaneous capnography

Outcome Measures

Primary Outcomes (1)

• 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.

  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)

  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.

• 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.

• 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

• 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.

Study Arms (1)

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

Diagnostic Test: Monitoring of NIV at home

Interventions

Monitoring of NIV at home DIAGNOSTIC_TEST

Monitoring of NIV at home

Adult patients with Duchenne using NIV in follow-up at the Leuven University Center for Sleep/wake

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients are in follow-up at the Leuven University City for Sleep/wake disorders

You may qualify if:

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

Sponsors & Collaborators

• Universitaire Ziekenhuizen KU Leuven: lead

Study Sites (1)

Universitaire Ziekenhuizen KU Leuven

Leuven, Vlaams-Brabant, 3000, Belgium

RECRUITING

Related Publications (3)

• 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.

  PMID: 24388970 BACKGROUND

• 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.

  PMID: 17711917 BACKGROUND

• 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.

  PMID: 24532751 BACKGROUND

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Dries Testelmans, MD PHD

CONTACT

+3216342522; dries.testelmans@uzleuven.be

Bart Vrijsen, PT PHD

CONTACT

+3216342522; bart.vrijsen@uzleuven.be

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 3 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 4, 2024
• First Posted: January 14, 2025
• Study Start: December 6, 2024
• Primary Completion (Estimated): October 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: January 14, 2025

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1)