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Late-Onset Pompe DiseaseNCT05431127

High Dose Inspiratory Muscle Training in LOPD

A study testing IMT therapy using the Pr02 mobile device in people with Late-Onset Pompe Disease and Lysosomal Disease, run by Duke University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Late-Onset Pompe Disease, Lysosomal Disease
What is being tested?
IMT therapy using the Pr02 mobile device
Type of treatment
Medical device
Could I receive a placebo?
No, everyone receives an active treatment
How many people
34 participants
Where
1 siteUnited States
Study dates
19 Jul 2022to 1 Jan 2025
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Duke University Medical Center

    Durham, North Carolina, 27705

Other trials in United States

About this study

Official title: High-dose Inspiratory Muscle Training (IMT) in Late-onset Pompe Disease (LOPD)

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.

Read the 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.

Being tested
IMT therapy using the Pr02 mobile device
Keywords
LOPD

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • IMT therapy using the Pr02 mobile deviceDevice

    Inspiratory Muscle Training using a device used to measure and increase respiratory strength and performance through resisted breathing exercises.

Study groups in NCT05431127
Study groupRoleWhat they receive
High Dose Inspiratory Muscle TrainingOtherInspiratory Muscle Training 3 times a week over 26 weeks
Receives: Device: IMT therapy using the Pr02 mobile device

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in maximum inspiratory pressure (MIP)

    Change pre-test to post test, measured in cm H20

    Measured: Baseline, week 15, week 30

Secondary outcomes (22)
  1. Change in maximum expiratory pressure (MEP)

    Change pre-test to post test, measured in cm H20

    Measured: Baseline, week 15, week 30

  2. Change in inspiratory power curve (IPC)

    Change pre-test to post test, measured in pressure-time units (PTUs)

    Measured: Baseline, week 15, week 30

  3. Change in inspiratory duration (ID)

    Change pre-test to post test, measured by duration in seconds

    Measured: Baseline, week 15, week 30

  4. 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

    Measured: Baseline, week 15, week 30

  5. Change in forced vital capacity (FVC)

    Change pre-test to post-test, measured in liters using a portable hand-held spirometer

    Measured: Baseline, week 15, week 30

  6. 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

    Measured: Baseline, week 15, week 30

  7. Change in peak expiratory flow (PEF)

    change pre-test to post-test, measured in liters per second using portable hand-held spirometer

    Measured: Baseline, week 15, week 30

  8. Change in inspiratory phase duration (IPD)

    change pre-test to post-test, measured in seconds

    Measured: Baseline, week 15, week 30

  9. Change in inspiratory peak flow (IPF)

    Change pre-test to post-test, measured in liters per second

    Measured: Baseline, week 15, week 30

  10. Change in compression phase duration (CPD)

    Change pre-test to post-test, measured in seconds

    Measured: Baseline, week 15, week 30

  11. Change in expiratory phase rise time (EPRT)

    Change pre-test to post-test, measured in liters per second

    Measured: Baseline, week 15, week 30

  12. Change in cough volume acceleration (CVA)

    Change pre-test to post-test, measured by EPF/EPRT

    Measured: Baseline, week 15, week 30

  13. 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.

    Measured: Baseline, week 15, week 30

  14. 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.

    Measured: Baseline, week 15, week 30

  15. 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.

    Measured: Baseline, week 15, week 30

  16. 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.

    Measured: Baseline, week 15, week 30

  17. 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.

    Measured: Baseline, week 15, week 30

  18. 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.

    Measured: Baseline, week 15, week 30

  19. 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.

    Measured: Baseline, week 15, week 30

  20. 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.

    Measured: Baseline, week 15, week 30

  21. 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.

    Measured: Baseline, week 15, week 30

  22. 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.

    Measured: Baseline, week 15, week 30

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Jun 2022
  2. Study start19 Jul 2022
  3. Primary completion1 Jan 2025
  4. Study completion1 Jan 2025

Resources

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
Supportive care
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
34 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05431127
Sponsor's protocol ID
Pro00109392
Status last verified
May 2025
First submitted
21 Jun 2022
Last updated
1 Oct 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A study testing IMT therapy using the Pr02 mobile device in people with Late-Onset Pompe Disease and Lysosomal Disease, run by Duke University at 1 site in United States.

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 6 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: finished, as of the sponsor's last update on 1 Oct 2025. 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 United States. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Change in maximum inspiratory pressure (MIP) (time frame: Baseline, week 15, week 30). 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 1 Oct 2025.

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