Multisystem and Autonomic Complications of NMD on Long-term NIV

NCT04100993 | Completed

• 1 other identifier: 266270
• Study Type: observational
• Target: 35
• Locations: 1 country
• Sites: 1

Brief Summary

This exploratory observational study evaluates autonomic function using measures including heart rate variability in adults with childhood-onset neuromuscular and neurological disorders (NMD) who use non-invasive ventilation (NIV) as part of their long-term care. Autonomic function will be correlated with measures of symptom burden, disease severity and complications.

Conditions

Neuromuscular Diseases; Neurologic Disorder

Outcome Measures

Primary Outcomes (1)

• Heart rate variability (HRV)

  HRV will be analysed in time and frequency domains from short-term (5 minute) artefact free ECG recordings. Normal parameters will be determined from an age- and sex-matched exploratory reference group of healthy adults.

  1 day

Secondary Outcomes (22)

• Baroreflex sensitivity (BRS)

  1 day

• Composite Autonomic Symptom Scale 31 questionnaire

  1 day

• Sydney Swallow Questionnaire

  1 day

• The European Quality of Life - 5 Dimensions questionnaire

  1 day

• Patient Assessment of Constipation Symptoms questionnaire

  1 day

Study Arms (2)

Patient group

60 participants ≥16 years of age with a confirmed diagnosis of a childhood-onset NMD

Other: No intervention used

Exploratory reference group

20 healthy adults ≥16 years of age

Other: No intervention used

Interventions

No intervention used OTHER

No intervention used

Exploratory reference group; Patient group

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with a relevant NMD who attend adult Sleep and Ventilation services at the study site will be identified by the direct care team. Healthy age- and sex-matched adults from the local community will be invited to participate by the research team.

You may qualify if:

• Patient group:
• Age ≥16 years
• Confirmed diagnosis of childhood-onset NMD
• Able and willing to provide informed consent
• Requires non-invasive ventilation on an intermittent or continuous basis to manage respiratory failure
• Reference group:
• Age ≥16 years
• No significant co-morbidities
• Not on any regular medications
• Able and willing to provide informed consent

You may not qualify if:

• Inability to travel to hospital site
• Clinical instability or acute deterioration requiring hospitalisation or changes to medication in the previous 4 weeks
• A female of child-bearing potential who is pregnant or intends to become pregnant during study involvement

Sponsors & Collaborators

• Royal Brompton & Harefield NHS Foundation Trust: lead

Study Sites (1)

Royal Brompton Hospital

London, SW3 6HP, United Kingdom

Location

MeSH Terms

Conditions

Neuromuscular Diseases; Nervous System Diseases

Study Officials

• Anita K Simonds, MD MBBS

  Royal Brompton & Harefield NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 31, 2019
• First Posted: September 24, 2019
• Study Start: October 25, 2019
• Primary Completion: May 20, 2021
• Study Completion: May 20, 2021
• Last Updated: October 16, 2024

Record last verified: 2024-10

Locations

GB (1)