Neuromuscular DiseasesNCT02951572
Non-invasive Ventilation and Physical Activity
A study testing NIV in people with Neuromuscular Diseases, run by Universitaire Ziekenhuizen KU Leuven at 1 site in Belgium.
Trial summary
As fatigue and daytime sleepiness are typical symptoms of alveolar hypoventilation in patients with neuromuscular disorders and thoracic deformations, we hope, by starting non-invasive ventilation and improving these symptoms, to increase their physical activity. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Neuromuscular Diseases
- What is being tested?
- NIV
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteBelgium
- Study dates
- Feb 2013to Jun 2017
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- restrictive pulmonary function test AND
- symptoms of nocturnal alveolar hypoventilation or
- increased daytime pressure of arterial CO2 (PaCO2) (>45 mmHg) or
- >= 10 mmHg increase in PaCO2 during sleep in comparison to a normal awake supine value
Who cannot join
- patients < 16 years
- severe cardiac arrythmias or conduction disturbances
- severe mental disturbances
- amyotrophic lateral sclerosis
- total wheelchair-dependent patients
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Non-invasive Ventilation and Physical Activity in Patients With Neuromuscular Disorders and Thoracic Wall Disorders
As fatigue and daytime sleepiness are typical symptoms of alveolar hypoventilation in patients with neuromuscular disorders and thoracic deformations, we hope, by starting non-invasive ventilation and improving these symptoms, to increase their physical activity.
Read the detailed description
Patients with neuromuscular disorders and thoracic deformations develop alveolar hypoventilation during their disease progression. At that time, non-invasive ventilation(NIV) can be started as treatment.
Fatigue and daytime sleepiness are typical symptoms of alveolar hypoventilation. NIV has the intention to improve these symptoms but no research has been performed whether these improvements also objectively improve physical activity. Further on, no research has been performed on muscle strength and muscle fatigue.
Patients' physical activity, physical capacity and sleep quality will be measured before NIV initiation, after 3 months and 1 year of NIV use.
To start NIV, patients will be admitted to our sleep laboratory for 5 days/4 nights. Polysomnography will be used during NIV titration.
Physical activity will each time be measured by 2 activity monitors during 5 days.
- Conditions
- Neuromuscular Diseases
- Being tested
- NIV
- Keywords
- Noninvasive Ventilation, Physical activity
- Medical subject headings
- Neuromuscular Diseases, Motor Activity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NIVDevice
| Study group | Role | What they receive |
|---|---|---|
| Patients with RLD initiating NIV | Other | Patients with RLD initiating NIV according to Belgian Health guidelines are followed-up before and after NIV initiation Receives: Device: NIV |
What is being measured
The primary outcome is the main question the study is designed to answer.
Average daily walking time
Average daily walking time will be measured by the McRoberts physical activity monitor
Measured: Measured before initiation of NIV
Average daily walking time
Average daily walking time will be measured by the McRoberts physical activity monitor
Measured: Measured after 3 months of NIV
Average daily walking time
Average daily walking time will be measured by the McRoberts physical activity monitor
Measured: Measured after 1 year of NIV
Secondary outcomes (12)
Change in functional exercise capacity
Six minute walking test
Measured: Before NIV initiation
Change in muscle strength
Quadriceps muscle strength will be measured by magnetic stimulation. MicroFet measurements of wrist flexion/extension and ankle dorsiflexion/plantar flexion Respiratory muscle strength will be measured by maximal inspiratory pressure and sniff nasal inspiratory pressure
Measured: Before NIV initiation
Change in quadriceps fatiguability
Use of magnetic stimulation before and after performing fatigue test of the quadriceps muscle
Measured: Before NIV intiation
Change in sleep structure and quality of sleep
Full polysomnography will be used to get insight in sleep improvement
Measured: Before NIV intiation
Change in functional exercise capacity
Six minute walking test
Measured: After 3 months of NIV use
Change in functional exercise capacity
Six minute walking test
Measured: After 1 year NIV use
Change in muscle strength
Quadriceps muscle strength will be measured by magnetic stimulation. MicroFet measurements of wrist flexion/extension and ankle dorsiflexion/plantar flexion Respiratory muscle strength will be measured by maximal inspiratory pressure and sniff nasal inspiratory pressure
Measured: After 3 months of NIV use
Change in muscle strength
Quadriceps muscle strength will be measured by magnetic stimulation. MicroFet measurements of wrist flexion/extension and ankle dorsiflexion/plantar flexion Respiratory muscle strength will be measured by maximal inspiratory pressure and sniff nasal inspiratory pressure
Measured: After 1 year NIV use
Change in quadriceps fatiguability
Use of magnetic stimulation before and after performing fatigue test of the quadriceps muscle
Measured: After 3 months of NIV use
Change in quadriceps fatiguability
Use of magnetic stimulation before and after performing fatigue test of the quadriceps muscle
Measured: After 1 year NIV use
Change in sleep structure and quality of sleep
Full polysomnography will be used to get insight in sleep improvement
Measured: After 3 months of NIV use
Change in sleep structure and quality of sleep
Full polysomnography will be used to get insight in sleep improvement
Measured: After 1 year NIV use
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Nov 2016 (estimated)
- Study startFeb 2013
- Primary completionFeb 2017
- Study completionJun 2017
Why it stopped: Recruitment problem
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DiseasesStudies 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Universitaire Ziekenhuizen KU Leuven
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dr. Dries Testelmans, Universitaire Ziekenhuizen KU Leuven
- Investigators
- Dries Testelmans, MD, PhD, Principal investigator, Universitaire Ziekenhuizen KU LeuvenBertien Buyse, MD,PhD, Study director, Universitaire Ziekenhuizen KU Leuven
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02951572
- Sponsor's protocol ID
- S54220
- Status last verified
- Nov 2020
- First submitted
- 9 Dec 2013
- Last updated
- 23 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Non-invasive Ventilation and Physical Activity in Patients With Neuromuscular Disorders and Thoracic Wall Disorders
- ClinicalTrials.gov ID
- NCT02951572
- Acronym / sponsor ID
- S54220
- Conditions
- Neuromuscular Diseases
- Keywords
- Noninvasive Ventilation, Physical activity
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Recruitment problem
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Universitaire Ziekenhuizen KU LeuvenUniversity, hospital or other
- Study start
- Feb 2013
- Primary completion
- Feb 2017 (actual)
- Study completion
- Jun 2017 (actual)
- First posted
- 1 Nov 2016 (estimated)
- 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.
As fatigue and daytime sleepiness are typical symptoms of alveolar hypoventilation in patients with neuromuscular disorders and thoracic deformations, we hope, by starting non-invasive ventilation and improving these symptoms, to increase their physical activity.
Detailed description
Patients with neuromuscular disorders and thoracic deformations develop alveolar hypoventilation during their disease progression. At that time, non-invasive ventilation(NIV) can be started as treatment.
Fatigue and daytime sleepiness are typical symptoms of alveolar hypoventilation. NIV has the intention to improve these symptoms but no research has been performed whether these improvements also objectively improve physical activity. Further on, no research has been performed on muscle strength and muscle fatigue.
Patients' physical activity, physical capacity and sleep quality will be measured before NIV initiation, after 3 months and 1 year of NIV use.
To start NIV, patients will be admitted to our sleep laboratory for 5 days/4 nights. Polysomnography will be used during NIV titration.
Physical activity will each time be measured by 2 activity monitors during 5 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with RLD initiating NIV | Other | Device: NIVPatients with RLD initiating NIV according to Belgian Health guidelines are followed-up before and after NIV initiation |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- restrictive pulmonary function test AND
- symptoms of nocturnal alveolar hypoventilation or
- increased daytime pressure of arterial CO2 (PaCO2) (>45 mmHg) or
- >= 10 mmHg increase in PaCO2 during sleep in comparison to a normal awake supine value
Key exclusion criteria (5 listed)
- patients < 16 years
- severe cardiac arrythmias or conduction disturbances
- severe mental disturbances
- amyotrophic lateral sclerosis
- total wheelchair-dependent patients
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 |
|---|---|---|
| NIV | Device | Patients with RLD initiating NIV |
Primary outcome
Average daily walking time
Time frame: Measured before initiation of NIV
Average daily walking time will be measured by the McRoberts physical activity monitor
Average daily walking time
Time frame: Measured after 3 months of NIV
Average daily walking time will be measured by the McRoberts physical activity monitor
Average daily walking time
Time frame: Measured after 1 year of NIV
Average daily walking time will be measured by the McRoberts physical activity monitor
12 secondary outcomes
Change in functional exercise capacity
Time frame: Before NIV initiation
Change in muscle strength
Time frame: Before NIV initiation
Change in quadriceps fatiguability
Time frame: Before NIV intiation
Change in sleep structure and quality of sleep
Time frame: Before NIV intiation
Change in functional exercise capacity
Time frame: After 3 months of NIV use
Change in functional exercise capacity
Time frame: After 1 year NIV use
Change in muscle strength
Time frame: After 3 months of NIV use
Change in muscle strength
Time frame: After 1 year NIV use
Change in quadriceps fatiguability
Time frame: After 3 months of NIV use
Change in quadriceps fatiguability
Time frame: After 1 year NIV use
Change in sleep structure and quality of sleep
Time frame: After 3 months of NIV use
Change in sleep structure and quality of sleep
Time frame: After 1 year NIV use
Results and safety
The study was stopped early (reason given: Recruitment problem). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Nov 2020.
Related trials for Neuromuscular Diseases
RecruitingObservational study
Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
Frontotemporal Dementia, Amyotrophic Lateral Sclerosis
RecruitingPhase 4
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
Spinal Muscular Atrophy
RecruitingPhase 3
Evaluation of IV AMBTX-01 (Neridronate) for Treatment of CRPS Type 1 (CRPS-RISE)
Complex Regional Pain Syndrome Type I
Not yet recruitingPhase 3
A Study to Understand How Nerandomilast Works in People With Active Idiopathic Inflammatory Myopathies
Idiopathic Inflammatory Myopathies
Not yet recruitingEarly Phase 1
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
Spinal Muscular Atrophy (SMA)
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. This study is not recruiting at the moment.
Dries Testelmans, MD, PhD principal investigator
Universitaire Ziekenhuizen KU Leuven
Bertien Buyse, MD,PhD study director
Universitaire Ziekenhuizen KU Leuven
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing NIV in people with Neuromuscular Diseases, 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 4 inclusion and 5 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: stopped early, as of the sponsor's last update on 23 Nov 2020. 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?
The study was stopped early (reason given: Recruitment problem). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Average daily walking time (time frame: Measured before initiation of NIV). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 Nov 2020.