Clinical trial NCT04224961: Respiratory Muscle Training (RMT) in ALS
Official title: Moderate Intensity Respiratory Muscle Training (RMT) in ALS
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: ALS
Lead sponsor: Duke University
Sponsor class: OTHER
First posted: 2020-01-13
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

Brief summary: Twelve adults with ALS will participate in a study involving four 3-week cycles of progressive respiratory muscle training (RMT). The investigators will measure maximum inspiratory pressure (MIP) and maximum expiratory pressure (MEP) weekly. The investigators will obtain the majority of measurements of MIP and MEP during web-based telehealth visits. Participants will be stratified into 2 groups based on baseline inspiratory muscle strength as determined by maximum inspiratory pressure (MIP): six participants with minimal to no respiratory weakness (i.e., MIP ≥ 70% predicted) and six participants with mild to moderate inspiratory weakness (i.e., MIP 40-70% predicted).

Detailed description: Twelve adults will participate in a single- subject A1-B-A2 study involving 4 visits to Duke over 24 weeks. The 6 week delayed start period (A1) will establish baseline status prior to initiation of RMT (B) and the follow-up period (A2) will determine detraining effects over 6 weeks of RMT withdrawal. Participants will complete four 3-week cycles of progressive RMT during the B phase. The investigators will measure MIP and MEP weekly. To reduce participant burden and enhance our ability to obtain repeated measures, the investigators will obtain 21 of 25 measurements of MIP and MEP during web-based telehealth visits.

Participants will complete a full assessment during the first study visit (M1) and receive training/instruction for obtaining MIP and MEP measurements at home with a portable, battery-powered digital pressure gauge manometer (MicroRPM Pressure Meter; Micro Direct). They will return to Duke for the pretest visit (M7), at which time a full assessment will be completed and RMT will commence. During the B phase, participants will complete 12-weeks of moderate intensity RMT, divided into four 3-week cycles, and participate in weekly web-based RMT therapy visits (M8-M18). Post-test assessment (M19) will occur immediately following completion of the 12-week RMT regimen. The investigators will measure MIP and MEP during RMT withdrawal via web-based telehealth visits (M20-24). Following the fourth and final on-site full assessment visit (M25), participants will return all equipment and will be offered the opportunity to initiate a clinical RMT regimen and receive follow-up care by Speech Pathology during future ALS Clinic visits.

Participants will be stratified into 2 groups based on baseline inspiratory muscle strength as determined by maximum inspiratory pressure (MIP): six participants with minimal to no respiratory weakness (i.e., MIP ≥ 70% predicted) and six participants with mild to moderate inspiratory weakness (i.e., MIP 40-70% predicted).

The primary outcomes are MIP and MEP. Secondary outcomes include peak cough flow (PCF) and sniff nasal inspiratory pressure (SNIP). Exploratory outcomes include the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS), Eating Assessment Tool - 10 Item (EAT-10), 36-Item Short Form Survey (SF36), World Health Organization Quality of Life-BREF (WHOQOL-BREF), and Communicative Effectiveness Survey-Revised (CES-R). Clinical spirometry data will be extracted from the participant's electronic medical record if available from a coinciding ALS Clinic visit that occurs +/- 5 days from the assessment visit.

Eligibility criteria: Inclusion Criteria:

* Age ≥ 18 years
* Confirmed diagnosis of ALS
* Ability to follow directions for study participation
* Ability to successfully and independently complete RMT repetitions at a minimum pressure-threshold target equal to 30% of MIP or MEP
* Ability to complete telehealth visits using a smartphone or computer with video capabilities
* Ability to complete a home-RMT regimen

Exclusion Criteria:

* MIP \< 40 cmH20
* Presence of a tracheostomy
* Use of non-invasive or invasive ventilation when awake
* Participant or caregiver(s) inability to manipulate respiratory pressure meter, the RMT device, or calibration equipment for home training
* Inability to complete RMT repetitions successfully
* Concomitant neurologic or neurodegenerative conditions (e.g. stroke, dementia) or other serious conditions that would prevent meaningful study participation as determined at the discretion of the principal investigator
* Inability to give legally effective consent
* Inability to read and understand English

Primary outcomes:
measure: Percentage of Patients with Safe, Well-tolerated Moderate Intensity Respiratory Muscle Training (RMT)
timeFrame: 12 weeks
measure: Percentage of Patients Able to Complete Respiratory Muscle Training (RMT)
timeFrame: 12 weeks
measure: Magnitude of Change in Maximum Expiratory Pressure from Pre-test to Post-test
timeFrame: Baseline, 12 weeks
measure: Magnitude of Change in Maximum Expiratory Pressure from Baseline to Withdrawal
timeFrame: Baseline, 24 weeks
measure: Magnitude of Change in Maximum Inspiratory Pressure from Pre-test to Post-test
timeFrame: Baseline, 12 weeks
Secondary outcomes:
measure: Change in Peak Cough Flow in L/min
timeFrame: Baseline, 7 weeks, 18 weeks, 24 weeks
measure: Change in Sniff Nasal Inspiratory Pressure
timeFrame: Baseline, 7 weeks, 18 weeks, 24 weeks
Other outcomes:
Locations:
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
Study officials:
affiliation: Duke University
name: Harrison Jones, PhD
role: PRINCIPAL_INVESTIGATOR
References: