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| Name | Class |
|---|---|
| Genzyme, a Sanofi Company | INDUSTRY |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| High Dose Inspiratory Muscle Training | Other | Inspiratory Muscle Training 3 times a week over 26 weeks |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| IMT therapy using the Pr02 mobile device | Device | Inspiratory Muscle Training using a device used to measure and increase respiratory strength and performance through resisted breathing exercises. |
| Measure | Description | Time Frame |
|---|---|---|
| Change in maximum inspiratory pressure (MIP) | Change pre-test to post test, measured in cm H20 | Baseline, week 15, week 30 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in maximum expiratory pressure (MEP) | Change pre-test to post test, measured in cm H20 | Baseline, week 15, week 30 |
| Change in inspiratory power curve (IPC) | Change pre-test to post test, measured in pressure-time units (PTUs) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Harrison Jones, PhD | Duke University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Duke University Medical Center | Durham | North Carolina | 27705 | United States |
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| Baseline, week 15, week 30 |
| Change in inspiratory duration (ID) | Change pre-test to post test, measured by duration in seconds | Baseline, week 15, week 30 |
| 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 | Baseline, week 15, week 30 |
| Change in forced vital capacity (FVC) | Change pre-test to post-test, measured in liters using a portable hand-held spirometer | Baseline, week 15, week 30 |
| 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 | Baseline, week 15, week 30 |
| Change in peak expiratory flow (PEF) | change pre-test to post-test, measured in liters per second using portable hand-held spirometer | Baseline, week 15, week 30 |
| Change in inspiratory phase duration (IPD) | change pre-test to post-test, measured in seconds | Baseline, week 15, week 30 |
| Change in inspiratory peak flow (IPF) | Change pre-test to post-test, measured in liters per second | Baseline, week 15, week 30 |
| Change in compression phase duration (CPD) | Change pre-test to post-test, measured in seconds | Baseline, week 15, week 30 |
| Change in expiratory phase rise time (EPRT) | Change pre-test to post-test, measured in liters per second | Baseline, week 15, week 30 |
| Change in cough volume acceleration (CVA) | Change pre-test to post-test, measured by EPF/EPRT | Baseline, week 15, week 30 |
| 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. | Baseline, week 15, week 30 |
| 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. | Baseline, week 15, week 30 |
| 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. | Baseline, week 15, week 30 |
| 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. | Baseline, week 15, week 30 |
| 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. | Baseline, week 15, week 30 |
| 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. | Baseline, week 15, week 30 |
| 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. | Baseline, week 15, week 30 |
| 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. | Baseline, week 15, week 30 |
| 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. | Baseline, week 15, week 30 |
| 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. | Baseline, week 15, week 30 |