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Trial registered on ANZCTR
Registration number
ACTRN12624001405505
Ethics application status
Approved
Date submitted
11/11/2024
Date registered
28/11/2024
Date last updated
28/11/2024
Date data sharing statement initially provided
28/11/2024
Type of registration
Prospectively registered
Titles & IDs
Public title
Combined Hyperinflation and Expiratory Strength Training in People Living With Motor Neurone Disease (CHEST-MND: PRO)
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Scientific title
Prospective Controlled Run-in Study Evaluating Combined Respiratory Training on Cough and Respiratory Function in people living with Motor Neurone Disease
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Secondary ID [1]
313232
0
Nil known
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Universal Trial Number (UTN)
U1111-1314-8223
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Trial acronym
CHEST-MND: PRO
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Motor neurone disease
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Condition category
Condition code
Respiratory
332149
332149
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0
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Other respiratory disorders / diseases
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Physical Medicine / Rehabilitation
332169
332169
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0
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Physiotherapy
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Neurological
332150
332150
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0
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Neurodegenerative diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
All enrolled participants will commence a combined lung volume recruitment and expiratory muscle strength training exercise regimen (LVR and EMST), following a 5-week no-intervention lead-in period.
Intervention description: LVR uses a self-inflating bag (similar to a resuscitation bag) with a mouthpiece or mask to deliver consecutive insufflations (i.e., large “puffs” of air), to “stack” a larger volume of air than can be inspired spontaneously.
EMST uses a spring-loaded pressure threshold device to provide resistance as one breaths out.
This study combines these two devices, so that a person "stacks" a volume of air in using the LVR bag, and then exhales through the resistive training device.
Intervention dose: Participants will be instructed to complete 5 sets of 5 repetitions of combined LVR+EMST into a single device on 5 days per week for 5 weeks (total of 25 intervention sessions over a 5-week period). One repetition consists of an individually prescribed number of compressions via the LVR bag to reach the maximal tolerated inflation capacity, followed by a forceful expiration through the EMST device, set at 50% of the participant’s maximal expiratory pressure (MEP). Exercises are usually performed seated (chair or wheelchair), and take approximately 20 minutes to complete.
An experienced health care professional (e.g. physiotherapist with experience in the provision of respiratory care) will prescribe the therapy and ensure participants are trained in its appropriate use. This initial training will be performed in the participant's home, face-to-face.
Subsequent sessions will be conducted by the participant themselves, with the assistance of their caregiver (if required), in their home environment. Training technique will be reviewed by a member of the research team via telehealth weekly for the duration of the study.
Adherence to the intervention will be assessed via a participant diary, as well as via a LVR counter attached to the LVR kit.
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Intervention code [1]
329837
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Rehabilitation
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Comparator / control treatment
This study is a prospective controlled run-in trial.
Participants will undertake a 5-week no treatment period (dose of treatment = nil)
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Control group
Active
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Outcomes
Primary outcome [1]
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Peak cough flow
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Assessment method [1]
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Peak expiratory flow rate measured as the participant performs an unassisted cough into an oronasal mask
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Timepoint [1]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10). Also measured weekly via telehealth (Weeks 1-4 and Weeks 6-9).
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Secondary outcome [1]
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Side effects and adverse events during LVR+EMST
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Assessment method [1]
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Number of side effects and serious adverse events reported during study period. Events will be categorised according to severity (trivial, mild, moderate, severe), outcome (recovery, continuing, worsening, death) and likelihood of being related to intervention (unrelated, possible, probable, definite). Side effects include light-headedness, dizziness, dyspnoea, fatigue, aerophagia. Serious adverse events include prolonged hypotension, pneumothorax.
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Timepoint [1]
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Assessed weekly (question posed to participant) at Weeks 1-4 (via telehealth), Weeks 6-9 (via telehealth) and in-person Post LVR+EMST (Week 10). Events will be collated and reported per participant for the study period.
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Secondary outcome [2]
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Participant qualitative experience of intervention
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Assessment method [2]
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Qualitative data, obtained via semi-structured interviews with participants will be undertaken via Zoom or Microsoft Teams to explore participant experiences of the treatment. Interviews will be ~40 mins to 1 hour and conducted by researchers with qualitative methodology and interview experience. Interviews will explore patients’ experience of participating in the clinical trial, the treatment, barriers and facilitators to the use and prescription of LVR+EMST, and any perceived impacts on their treatment goals and outcomes. Interview analysis guided by the Theoretical Framework of Acceptability and the COM-B model.
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Timepoint [2]
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Post LVR+EMST (Week 10).
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Secondary outcome [3]
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Cough spirometry
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Assessment method [3]
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Participants will perform cough manoeuvres during voluntary unassisted and LVR-assisted cough manoeuvres, measured via a pneumotachograph to capture flow, volume and pressure. Cough metrics include: inspiratory peak flow, inspiratory phase duration, compression phase duration, expiratory peak flow (“peak cough flow”), peak expiratory rise time, cough volume acceleration, expiratory phase duration, pre-cough inspiratory volume, cough expiratory volume.
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Timepoint [3]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [4]
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Maximal expiratory pressure
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Assessment method [4]
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Measured in accordance with European Respiratory Society guidelines (using MicroRPM device or similar).
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Timepoint [4]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [5]
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Maximum phonation time
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Assessment method [5]
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Participants will be instructed to take a deep breath in and hold the sound “Ah” for as long as possible. Measured via stopwatch in seconds.
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Timepoint [5]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10). Also measured weekly via telehealth (Weeks 1-4 and Weeks 6-9).
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Secondary outcome [6]
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International Dysphagia Diet Standardisation Initiative Functional Diet Scale (IDDSI-FDS)
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Assessment method [6]
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The IDDSI-FDS provides a score (0-8) that classifies dysphagia severity according to the degree of diet limitation
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Timepoint [6]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [7]
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Lung insufflation capacity
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Assessment method [7]
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Lung insufflation capacity is the maximal, tolerable inflation capacity reached with lung volume recruitment (LVR), and is measured by recording the volume of air forcefully exhaled following assisted inflation with a LVR kit.
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Timepoint [7]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [8]
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Adherence to LVR+EMST
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Assessment method [8]
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Proportion of LVR+EMST sessions completed, measured by LVR counter attached to LVR+EMST device and participant exercise diaries.
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Timepoint [8]
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Post LVR+EMST (Week 10).
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Secondary outcome [9]
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Healthcare utilisation
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Assessment method [9]
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Participants will be asked to recall health care utilisation during weekly telehealth appointments, including hospital admissions, medication usage.
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Timepoint [9]
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Post LVR+EMST (Week 10).
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Secondary outcome [10]
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Maximal inspiratory pressure
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Assessment method [10]
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Measured in accordance with European Respiratory Society guidelines (using MicroRPM device or similar).
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Timepoint [10]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [11]
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Bulbar function - swallowing symptoms
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Assessment method [11]
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The Center for Neurological Study - Bulbar Function Scale (CNS-BFS) is a 21-item validated, patient-reported scale indexing degree of bulbar dysfunction in the domains of speech, salivation, and swallowing. The individual CNS-BFS swallowing score will be used to assess the swallowing domain of bulbar function.
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Timepoint [11]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [12]
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Bulbar function, assessed as a composite measure
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Assessment method [12]
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The Center for Neurological Study - Bulbar Function Scale (CNS-BFS) is a 21-item validated, patient-reported scale indexing degree of bulbar dysfunction in the domains of speech, salivation, and swallowing. The CNS-BFS total score will be used to assess overall bulbar function as a composite measure. The individual domains of speech, swallowing and salivation will also be explored using the domain scores (see items below).
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Timepoint [12]
441052
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [13]
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Dyspnea
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Assessment method [13]
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The amyotrophic lateral sclerosis (ALS) respiratory symptom scale (ARES) is a 12-item patient-report scale indexing the degree of dyspnea with each question rated on a scale from 0 (no dyspnea) to 10 (severe dyspnea), and total score ranging from 0 (no dyspnea) to 120 (severe dyspnea).
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Timepoint [13]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [14]
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Sniff nasal inspiratory pressure
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Assessment method [14]
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Measured in accordance with European Respiratory Society guidelines (using MicroRPM device or similar).
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Timepoint [14]
441049
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [15]
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Global rating of change: cough effectiveness
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Assessment method [15]
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A 15-point global rating of change scale (GRC) shall be used to assess participant-perceived change in cough effectiveness.
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Timepoint [15]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10). Also measured weekly via telehealth (Weeks 1-4 and Weeks 6-9).
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Secondary outcome [16]
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Forced vital capacity
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Assessment method [16]
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The volume of air forcefully exhaled following a maximal active inspiration. Measured in accordance with American Thoracic Society guidelines.
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Timepoint [16]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [17]
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Care giver burden, assessed as a composite outcome
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Assessment method [17]
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The Zarit Burden Interview-7 (ZBI-7) is a 7-item questionnaire assessing the degree of the caregiver’s physical and psychosocial burden on a five-point likert scale ranging from 0 (never, not at all burdened) to 4 (nearly always, extremely burdened). Total scores range from 0 (no burden) to 28 (extremely burdened).
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Timepoint [17]
441056
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [18]
441055
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Severe Respiratory Insufficiency Questionnaire (SRI)
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Assessment method [18]
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The SRI is a disease-specific multi-dimensional QoL questionnaire, designed for people with chronic respiratory failure. It consists of seven sub-scales covering 49 items summarized to one summary scale (SRI-SS) between 0 and 100, with higher scores indicating a better QoL.
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Timepoint [18]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [19]
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Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
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Assessment method [19]
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A validated instrument for monitoring the progression of functional disability in people with ALS/MND, and marker of disease severity. The ALSFRS-R is a 12-item scale, with questions across upper limb, lower limb, bulbar and respiratory domains rated on a scale from 0 to 4.
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Timepoint [19]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [20]
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Bulbar function - salivation symptoms
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Assessment method [20]
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The Center for Neurological Study - Bulbar Function Scale (CNS-BFS) is a 21-item validated, patient-reported scale indexing degree of bulbar dysfunction in the domains of speech, salivation, and swallowing. The individual CNS-BFS sialorrhea score will be used to assess the salivation domain of bulbar function.
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Timepoint [20]
441966
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [21]
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Bulbar function - speech symptoms
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Assessment method [21]
441964
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The Center for Neurological Study - Bulbar Function Scale (CNS-BFS) is a 21-item validated, patient-reported scale indexing degree of bulbar dysfunction in the domains of speech, salivation, and swallowing. The individual CNS-BFS speech score will be used to assess the speech domain of bulbar function.
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Timepoint [21]
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Measured at Baseline 1 (Week 0), Baseline 2 (Week 5), and Post LVR+EMST (Week 10).
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Secondary outcome [22]
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Exercise therapy burden, assessed as a composite outcome
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Assessment method [22]
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The Exercise Therapy Burden Questionnaire (ETBQ) is a 10-item questionnaire specifically assessing the global burden related to the prescribed exercise regimen in chronic health conditions. Each question addresses different aspects of the exercise program (i.e., pain, fatigue, difficulty, time) and is rated on a scale from 0 (totally disagree) to 10 (totally agree), for a total score of 0 (no burden) to 100 (significant burden). The ETBQ score can be used to assess global burden, and an individualised analysis of responses can also be performed to explore domains.
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Timepoint [22]
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Post LVR+EMST (Week 10).
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Eligibility
Key inclusion criteria
A diagnosis of MND/ALS, according to the revised El Escorial criteria (possible, probable laboratory supported, probable, or definite).
Forced vital capacity (FVC) greater than or equal to 50% predicted normal reference values.
Naïve to lung volume recruitment (LVR) and expiratory muscle strength training (EMST).
Provided a signed and dated informed consent form (or witnessed signed form if unable to write),
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Minimum age
18
Years
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Maximum age
90
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
Medical instability (e.g., acute medical issues/hospitalisation).
History of stroke, head and neck cancer or other concomitant disorder that might contribute to dysphagia or respiratory impairment.
Diagnosis of frontotemporal dementia or advanced cognitive impairment.
Use of prescription mechanical insufflation-exsufflation or non-invasive ventilation devices.
Tracheostomy / use of invasive mechanical ventilation.
Enrolment in another research investigation that might impact cough or respiratory function.
Contraindications for respiratory training (i.e., history of pneumothorax, severe chronic obstructive pulmonary disease).
Unable to physically perform or can’t tolerate LVR.
Home visit safety risk.
Unable to provide informed consent.
Limited English proficiency.
Resides more than 2 hours travel time from Austin Health.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Crossover
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Other design features
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Phase
Not Applicable
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Type of endpoint/s
Safety/efficacy
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Statistical methods / analysis
This study is a pilot and feasibility study and as such is not powered to detect a difference in the primary outcome. To adequately evaluate feasibility and acceptability, 16 participants with MND will be sought for this study. This sample size exceeds the minimum recommended to achieve saturation for qualitative research of this nature (10-13 participants),(Francis et al 2010) and the “12 participant rule of thumb” for pilot studies.(Julious 2005) Moreover, it is feasible based on conservative estimates of diagnosis, with approximately two people diagnosed with MND per day in Australia, with an estimated 10 people/month residing in Victoria. It is anticipated approximately 2 participants will be recruited per month during the 12-month available for enrolment.
Consultation with a biostatistician will be undertaken through the University of Melbourne. The results of the study will be analysed in accordance with the intention to treat principle using linear models. Change over time for the period Baseline 1 to Baseline 2 will be compared to the period Baseline 2 to Post LVR+EMST. Quantitative data analysis will be undertaken using STATA and/or R software. Qualitative data will be primarily deductive. Data will be coded into 8 domains of the TFA and 6 domains of the COM-B. Sub-themes within these domains will be identified. Any themes that do not naturally fit within the TFA or COM-B will be inductively identified and included. A proportion of the interviews will be double-coded to ensure consistency.
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Recruitment
Recruitment status
Not yet recruiting
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Date of first participant enrolment
Anticipated
12/12/2024
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Actual
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
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Sample size
Target
16
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Calvary Health Care Bethlehem Ltd - Caulfield
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Recruitment hospital [2]
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Austin Health - Austin Hospital - Heidelberg
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Recruitment hospital [3]
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The Northern Hospital - Epping
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Recruitment postcode(s) [1]
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3162 - Caulfield
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Recruitment postcode(s) [2]
43374
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3084 - Heidelberg
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Recruitment postcode(s) [3]
43405
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3076 - Epping
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Funding & Sponsors
Funding source category [1]
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Charities/Societies/Foundations
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Name [1]
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FightMND
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Address [1]
317674
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Country [1]
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Australia
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Primary sponsor type
Other Collaborative groups
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Name
Institute for Breathing and Sleep (IBAS)
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Address
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Country
Australia
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Secondary sponsor category [1]
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None
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Name [1]
319992
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Address [1]
319992
0
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Country [1]
319992
0
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Ethics approval
Ethics application status
Approved
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Ethics committee name [1]
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Austin Health Human Research Ethics Committee
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Ethics committee address [1]
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https://www.austin.org.au/Office-for-Research/
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Ethics committee country [1]
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Australia
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Date submitted for ethics approval [1]
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15/07/2024
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Approval date [1]
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02/10/2024
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Ethics approval number [1]
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HREC/110306/Austin-2024
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Summary
Brief summary
Breathing and coughing difficulties are extremely distressing for people living with MND and therapies that improve breathing capacity and related symptoms are needed. Our research team has previously shown that breathing exercises using a hand-held device to help people take a bigger breath (lung volume recruitment) can produce important changes in lung capacity. Separately, our collaborators in the United States have found that exercises using a different hand-held device (expiratory muscle strength training) can improve expiratory muscle strength in people with MND. This research project will evaluate whether combining these two therapies together has added benefit. We will test whether five weeks of combined respiratory training is feasible and acceptable to people living with MND and their carers, and whether it improves breathing and cough strength, symptoms and quality of life in people affected by MND.
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Trial website
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Dr Nicole Sheers
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Address
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Institute for Breathing and Sleep; Level 5, Harold Stokes Building; Austin Hospital, 145 Studley Road; Heidelberg, VIC, 3084
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Country
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Australia
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Phone
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+61 468 862 693
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Fax
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Email
137690
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[email protected]
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Contact person for public queries
Name
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Nicole Sheers
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Address
137691
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Institute for Breathing and Sleep; Level 5, Harold Stokes Building; Austin Hospital, 145 Studley Road; Heidelberg, VIC, 3084
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Country
137691
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Australia
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Phone
137691
0
+61 468 862 693
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Fax
137691
0
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Email
137691
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[email protected]
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Contact person for scientific queries
Name
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Nicole Sheers
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Address
137692
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Institute for Breathing and Sleep; Level 5, Harold Stokes Building; Austin Hospital, 145 Studley Road; Heidelberg, VIC, 3084
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Country
137692
0
Australia
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Phone
137692
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+61 468 862 693
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Fax
137692
0
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Email
137692
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[email protected]
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Data sharing statement
Will the study consider sharing individual participant data?
Yes
Will there be any conditions when requesting access to individual participant data?
Persons/groups eligible to request access:
•
Principal Investigator Lauren Tabor Gray: ClinicalTrials.gov Identifier: NCT05913882.
Additionally, investigators whose proposed use of the individual participant data has been approved by an independent review committee will be eligible to request individual participant data. Data requesters will need to sign a data access agreement.
Conditions for requesting access:
•
-
What individual participant data might be shared?
•
This research trial has an a priori plan to conduct an individual patient data meta-analysis with data from a similar pilot study currently underway in Florida, USA (Principal Investigator Lauren Tabor Gray: ClinicalTrials.gov Identifier: NCT05913882). Additionally, de-identified data will be made available to selected Australian and international researchers upon approval of written requests.
What types of analyses could be done with individual participant data?
•
For IPD meta-analysis and de-identified individual data.
When can requests for individual participant data be made (start and end dates)?
From:
Start date: Immediately following publication
End date: No end date determined
To:
-
Where can requests to access individual participant data be made, or data be obtained directly?
•
Proposals to analyse individual participant data should be directed to the principal investigator, Dr Nicole Sheers (via email to:
[email protected]
)
Are there extra considerations when requesting access to individual participant data?
No
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
Documents added manually
No documents have been uploaded by study researchers.
Documents added automatically
No additional documents have been identified.
Download to PDF