CompletedNot Applicable

Multisystem and Autonomic Complications of NMD on Long-term NIV

United Kingdom35 participantsStarted 2019-10-25

Plain-language 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.

Who can participate

Age range

16 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: 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 Exclusion Criteria: * 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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Heart rate variability (HRV)

Timeframe: 1 day

Trial details

NCT IDNCT04100993

SponsorRoyal Brompton & Harefield NHS Foundation Trust

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2021-05-20

View on ClinicalTrials.gov More Neuromuscular Diseases trials