High Dose Inspiratory Muscle Training in LOPD
Primary Purpose
Late-Onset Pompe Disease, Lysosomal Disease
Status
Recruiting
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
IMT therapy using the Pr02 mobile device
Sponsored by
About this trial
This is an interventional supportive care trial for Late-Onset Pompe Disease focused on measuring LOPD
Eligibility Criteria
Inclusion Criteria:
- Age ≥ 18 years
- Confirmed diagnosis of LOPD
- MIP >50% of predicted for sex and age
- Stable on current Pompe disease treatment regimen >6 months
- Able to follow directions for study participation
- Access to computer and smartphone/tablet with reliable internet connection for video visits and sensor-based respiratory technologies
Exclusion Criteria:
- Presence of medical comorbidities that prevent meaningful study participation (e.g., COPD GOLD III-IV, significant mental illness, dementia)
- Use of continuous invasive or non-invasive ventilation while awake
- Prior history of gene therapy for LOPD
- Inability to give legally effective consent
- Inability to read and understand English
Sites / Locations
- Duke University Medical CenterRecruiting
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
High Dose Inspiratory Muscle Training
Arm Description
Inspiratory Muscle Training 3 times a week over 26 weeks
Outcomes
Primary Outcome Measures
Change in maximum inspiratory pressure (MIP)
Change pre-test to post test, measured in cm H20
Secondary Outcome Measures
Change in maximum expiratory pressure (MEP)
Change pre-test to post test, measured in cm H20
Change in inspiratory power curve (IPC)
Change pre-test to post test, measured in pressure-time units (PTUs)
Change in inspiratory duration (ID)
Change pre-test to post test, measured by duration in seconds
Change in fatigue index test score (FIT)
Change pre-test to post test, a proprietary measure which quantifies propensity to inspiratory muscle fatigue based upon the relationship between inspiratory capacity and demand using the following equation: (IPC [in Watts] x MID) / (Power500 x T500), where Power500 = power expended to inspire a mass of 500 mL air and T500 = time when mass of inspired air=500 mL at sea level
Change in forced vital capacity (FVC)
Change pre-test to post-test, measured in liters using a portable hand-held spirometer
Change in forced expiratory volume over 1 second (FEV1)
change pre-test to post-test, measured in liters per second using a portable hand-held spirometer
Change in peak expiratory flow (PEF)
change pre-test to post-test, measured in liters per second using portable hand-held spirometer
Change in inspiratory phase duration (IPD)
change pre-test to post-test, measured in seconds
Change in inspiratory peak flow (IPF)
Change pre-test to post-test, measured in liters per second
Change in compression phase duration (CPD)
Change pre-test to post-test, measured in seconds
Change in expiratory phase rise time (EPRT)
Change pre-test to post-test, measured in liters per second
Change in cough volume acceleration (CVA)
Change pre-test to post-test, measured by EPF/EPRT
Change in fatigue
Change pre-test to post-test, measured by Fatigue Severity Scale (FSS) survey completion, using a 7 point ordinal scale where a rating of 1 indicates strong disagreement and a rating of 7 indicates strong agreement.
Change in impact of fatigue on quality of life (QOL)
Change pre-test to post-test, measured by Modified Fatigue Impact Scale (MFIS) survey completion using a score of 0 (never affected) to 4 (almost always affected). The total score ranges from 0 to a maximum of 84. Higher scores indicate greater impact of fatigue on quality of life.
Change in daytime sleepiness
Change pre-test to post-test, measured by Epworth Sleepiness Scale (ESS) survey completion using a 0 to 3 ordinal scale in which 0=no chance of dozing, 1=slight chance of dozing, 2= moderate chance of dozing, and 3=high chance of dozing. Scores are summed to obtain total ESS score where a score >10 reflects excessive daytime sleepiness.
Change in sleep quality
Change pre-test to post-test, measured by Pittsburgh Sleep Quality Index (PSQI) survey completion. The PSQI is a 9-question, 19-item instrument. Items 1 to 4 are open-ended questions (customary bedtime, length of time to fall asleep). Items 5 to 8 (including the 10 questions comprising item 5) are sleep symptoms which are rated as to their frequency using an ordinal scale: 0=not occurring in the last month, 1=less than once a week, 2=once or twice a week, and 3=three or more times a week. Item 9 is a rating of overall sleep quality over the past month using a 0 to 3 ordinal scale: 0=very good; 1=fairly good; 2= fairly bad; and 3=very bad. Scores from the 19-items are combined according to standard scoring criteria to obtain a Global PSQI score. Scores >5 indicate reduced sleep quality.
Change in respiratory symptoms
Change pre-test to post-test, measured by Respiratory Symptoms Questionnaire (RSQ) completion using a 0-3 scale where a higher score indicates worse symptoms.
Change in motor performance
Change pre-test to post-test, measured by Rotterdam Handicap Scale (RHS) survey completion using a ranging scale of 1 - 4 where 1 = unable to fulfil the task or activity and 4 = complete fulfillment of the task or activity. Scores are summed and range from 9-36.
Change in health-related quality of life
Change pre-test to post-test, measured by Short Form 36 (SF-36) survey completion where physical and mental component summary scales (PCS and MCS) are calculated from the subscales and transformed to a normalized T-score with a mean of 50 and a standard deviation of 10. Higher scores represent better health-related quality of life.
Change in ability to communicate
Change pre-test to post-test, measured by Communicative Participation Item Bank-Short From (CPIB-10) survey completion using a 4-point scale (not at all=3, a little=2, quite a bit=1, very much=0). Item scores are added to obtain the summary score which ranges from 0-30. This can be transformed into a standard T score (mean=50, SD=10). Higher scores represent less interference in communication participation.
Change in voice quality
Change pre-test to post-test, measured by Voice Handicap Index (VHI-10) survey completion using a 5-point scale (0=never, 1=almost never, 2=sometimes, 3=almost always, 4=always). Item responses are added to obtain a total score (values >11 abnormal) with higher scores indicating greater perception of voice-related handicap.
Change in swallowing symptoms
Change pre-test to post-test, measured by Eating Assessment Tool (EAT-10) survey completion using a 5-point scale (0=no problem, 4=severe problem). Item responses are added to obtain a total score (values >3 abnormal) with higher scores indicating greater severity of swallowing symptoms.
Full Information
NCT ID
NCT05431127
First Posted
June 21, 2022
Last Updated
July 6, 2023
Sponsor
Duke University
Collaborators
Genzyme, a Sanofi Company
1. Study Identification
Unique Protocol Identification Number
NCT05431127
Brief Title
High Dose Inspiratory Muscle Training in LOPD
Official Title
High-dose Inspiratory Muscle Training (IMT) in Late-onset Pompe Disease (LOPD)
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 19, 2022 (Actual)
Primary Completion Date
August 2024 (Anticipated)
Study Completion Date
August 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Duke University
Collaborators
Genzyme, a Sanofi Company
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Study Objectives: 1) assess the safety and feasibility of high-dose inspiratory muscle training (IMT) delivered remotely in Late-onset Pompe Disease (LOPD) and 2) determine its effects on respiratory and patient-reported outcomes.
Detailed Description
This study aims to develop treatments that enhance respiratory strength and function to provide meaningful clinical improvements for people with LOPD. Identification of a cost-effective adjunctive intervention to address respiratory weakness remains critical to reduce disease burden, ease activity limitations and participation restrictions, and improve health-related quality of life. The proposed study will provide a high-dose inspiratory muscle training (IMT) stimulus to enhance treatment efficacy and efficiency. Our hypothesis is that high-dose IMT is necessary to produce meaningful changes in respiratory muscle strength and other outcomes in participants with LOPD.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Late-Onset Pompe Disease, Lysosomal Disease
Keywords
LOPD
7. Study Design
Primary Purpose
Supportive Care
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
29 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
High Dose Inspiratory Muscle Training
Arm Type
Other
Arm Description
Inspiratory Muscle Training 3 times a week over 26 weeks
Intervention Type
Device
Intervention Name(s)
IMT therapy using the Pr02 mobile device
Intervention Description
Inspiratory Muscle Training using a device used to measure and increase respiratory strength and performance through resisted breathing exercises.
Primary Outcome Measure Information:
Title
Change in maximum inspiratory pressure (MIP)
Description
Change pre-test to post test, measured in cm H20
Time Frame
Baseline, week 15, week 30
Secondary Outcome Measure Information:
Title
Change in maximum expiratory pressure (MEP)
Description
Change pre-test to post test, measured in cm H20
Time Frame
Baseline, week 15, week 30
Title
Change in inspiratory power curve (IPC)
Description
Change pre-test to post test, measured in pressure-time units (PTUs)
Time Frame
Baseline, week 15, week 30
Title
Change in inspiratory duration (ID)
Description
Change pre-test to post test, measured by duration in seconds
Time Frame
Baseline, week 15, week 30
Title
Change in fatigue index test score (FIT)
Description
Change pre-test to post test, a proprietary measure which quantifies propensity to inspiratory muscle fatigue based upon the relationship between inspiratory capacity and demand using the following equation: (IPC [in Watts] x MID) / (Power500 x T500), where Power500 = power expended to inspire a mass of 500 mL air and T500 = time when mass of inspired air=500 mL at sea level
Time Frame
Baseline, week 15, week 30
Title
Change in forced vital capacity (FVC)
Description
Change pre-test to post-test, measured in liters using a portable hand-held spirometer
Time Frame
Baseline, week 15, week 30
Title
Change in forced expiratory volume over 1 second (FEV1)
Description
change pre-test to post-test, measured in liters per second using a portable hand-held spirometer
Time Frame
Baseline, week 15, week 30
Title
Change in peak expiratory flow (PEF)
Description
change pre-test to post-test, measured in liters per second using portable hand-held spirometer
Time Frame
Baseline, week 15, week 30
Title
Change in inspiratory phase duration (IPD)
Description
change pre-test to post-test, measured in seconds
Time Frame
Baseline, week 15, week 30
Title
Change in inspiratory peak flow (IPF)
Description
Change pre-test to post-test, measured in liters per second
Time Frame
Baseline, week 15, week 30
Title
Change in compression phase duration (CPD)
Description
Change pre-test to post-test, measured in seconds
Time Frame
Baseline, week 15, week 30
Title
Change in expiratory phase rise time (EPRT)
Description
Change pre-test to post-test, measured in liters per second
Time Frame
Baseline, week 15, week 30
Title
Change in cough volume acceleration (CVA)
Description
Change pre-test to post-test, measured by EPF/EPRT
Time Frame
Baseline, week 15, week 30
Title
Change in fatigue
Description
Change pre-test to post-test, measured by Fatigue Severity Scale (FSS) survey completion, using a 7 point ordinal scale where a rating of 1 indicates strong disagreement and a rating of 7 indicates strong agreement.
Time Frame
Baseline, week 15, week 30
Title
Change in impact of fatigue on quality of life (QOL)
Description
Change pre-test to post-test, measured by Modified Fatigue Impact Scale (MFIS) survey completion using a score of 0 (never affected) to 4 (almost always affected). The total score ranges from 0 to a maximum of 84. Higher scores indicate greater impact of fatigue on quality of life.
Time Frame
Baseline, week 15, week 30
Title
Change in daytime sleepiness
Description
Change pre-test to post-test, measured by Epworth Sleepiness Scale (ESS) survey completion using a 0 to 3 ordinal scale in which 0=no chance of dozing, 1=slight chance of dozing, 2= moderate chance of dozing, and 3=high chance of dozing. Scores are summed to obtain total ESS score where a score >10 reflects excessive daytime sleepiness.
Time Frame
Baseline, week 15, week 30
Title
Change in sleep quality
Description
Change pre-test to post-test, measured by Pittsburgh Sleep Quality Index (PSQI) survey completion. The PSQI is a 9-question, 19-item instrument. Items 1 to 4 are open-ended questions (customary bedtime, length of time to fall asleep). Items 5 to 8 (including the 10 questions comprising item 5) are sleep symptoms which are rated as to their frequency using an ordinal scale: 0=not occurring in the last month, 1=less than once a week, 2=once or twice a week, and 3=three or more times a week. Item 9 is a rating of overall sleep quality over the past month using a 0 to 3 ordinal scale: 0=very good; 1=fairly good; 2= fairly bad; and 3=very bad. Scores from the 19-items are combined according to standard scoring criteria to obtain a Global PSQI score. Scores >5 indicate reduced sleep quality.
Time Frame
Baseline, week 15, week 30
Title
Change in respiratory symptoms
Description
Change pre-test to post-test, measured by Respiratory Symptoms Questionnaire (RSQ) completion using a 0-3 scale where a higher score indicates worse symptoms.
Time Frame
Baseline, week 15, week 30
Title
Change in motor performance
Description
Change pre-test to post-test, measured by Rotterdam Handicap Scale (RHS) survey completion using a ranging scale of 1 - 4 where 1 = unable to fulfil the task or activity and 4 = complete fulfillment of the task or activity. Scores are summed and range from 9-36.
Time Frame
Baseline, week 15, week 30
Title
Change in health-related quality of life
Description
Change pre-test to post-test, measured by Short Form 36 (SF-36) survey completion where physical and mental component summary scales (PCS and MCS) are calculated from the subscales and transformed to a normalized T-score with a mean of 50 and a standard deviation of 10. Higher scores represent better health-related quality of life.
Time Frame
Baseline, week 15, week 30
Title
Change in ability to communicate
Description
Change pre-test to post-test, measured by Communicative Participation Item Bank-Short From (CPIB-10) survey completion using a 4-point scale (not at all=3, a little=2, quite a bit=1, very much=0). Item scores are added to obtain the summary score which ranges from 0-30. This can be transformed into a standard T score (mean=50, SD=10). Higher scores represent less interference in communication participation.
Time Frame
Baseline, week 15, week 30
Title
Change in voice quality
Description
Change pre-test to post-test, measured by Voice Handicap Index (VHI-10) survey completion using a 5-point scale (0=never, 1=almost never, 2=sometimes, 3=almost always, 4=always). Item responses are added to obtain a total score (values >11 abnormal) with higher scores indicating greater perception of voice-related handicap.
Time Frame
Baseline, week 15, week 30
Title
Change in swallowing symptoms
Description
Change pre-test to post-test, measured by Eating Assessment Tool (EAT-10) survey completion using a 5-point scale (0=no problem, 4=severe problem). Item responses are added to obtain a total score (values >3 abnormal) with higher scores indicating greater severity of swallowing symptoms.
Time Frame
Baseline, week 15, week 30
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Age ≥ 18 years
Confirmed diagnosis of LOPD
MIP >50% of predicted for sex and age
Stable on current Pompe disease treatment regimen >6 months
Able to follow directions for study participation
Access to computer and smartphone/tablet with reliable internet connection for video visits and sensor-based respiratory technologies
Exclusion Criteria:
Presence of medical comorbidities that prevent meaningful study participation (e.g., COPD GOLD III-IV, significant mental illness, dementia)
Use of continuous invasive or non-invasive ventilation while awake
Prior history of gene therapy for LOPD
Inability to give legally effective consent
Inability to read and understand English
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Kelly Crisp, MA, CCC-SLP
Phone
919-681-1852
Email
kelly.crisp@duke.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Victoria Eifert
Phone
919-681-4595
Email
victoria.a.johnson@duke.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Harrison Jones, PhD
Organizational Affiliation
Duke University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Harrison Jones, PhD
Phone
919-681-1852
Email
harrison.jones@duke.edu
First Name & Middle Initial & Last Name & Degree
Kelly Crisp
Email
kelly.crisp@duke.edu
First Name & Middle Initial & Last Name & Degree
Harrison Jones, PhD
12. IPD Sharing Statement
Plan to Share IPD
No
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High Dose Inspiratory Muscle Training in LOPD
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