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.
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
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.
- Conditions
- Late-Onset Pompe Disease, Lysosomal Disease
- 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 group | Role | What they receive |
|---|---|---|
| High Dose Inspiratory Muscle Training | Other | Inspiratory 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.
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)
Change in maximum expiratory pressure (MEP)
Change pre-test to post test, measured in cm H20
Measured: Baseline, week 15, week 30
Change in inspiratory power curve (IPC)
Change pre-test to post test, measured in pressure-time units (PTUs)
Measured: Baseline, week 15, week 30
Change in inspiratory duration (ID)
Change pre-test to post test, measured by duration in seconds
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: Baseline, week 15, week 30
Change in inspiratory phase duration (IPD)
change pre-test to post-test, measured in seconds
Measured: Baseline, week 15, week 30
Change in inspiratory peak flow (IPF)
Change pre-test to post-test, measured in liters per second
Measured: Baseline, week 15, week 30
Change in compression phase duration (CPD)
Change pre-test to post-test, measured in seconds
Measured: Baseline, week 15, week 30
Change in expiratory phase rise time (EPRT)
Change pre-test to post-test, measured in liters per second
Measured: Baseline, week 15, week 30
Change in cough volume acceleration (CVA)
Change pre-test to post-test, measured by EPF/EPRT
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
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.
- First posted24 Jun 2022
- Study start19 Jul 2022
- Primary completion1 Jan 2025
- Study completion1 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Late-Onset Pompe DiseaseStudies of the same condition, open and under way.
- More trials run by Duke UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Responsible party
- Sponsor
- Investigators
- Harrison Jones, PhD, Principal investigator, Duke University
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
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- High-dose Inspiratory Muscle Training (IMT) in Late-onset Pompe Disease (LOPD)
- ClinicalTrials.gov ID
- NCT05431127
- Acronym / sponsor ID
- Pro00109392
- Conditions
- Late-Onset Pompe Disease, Lysosomal Disease
- Keywords
- LOPD
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Study start
- 19 Jul 2022 (actual)
- Primary completion
- 1 Jan 2025 (actual)
- Study completion
- 1 Jan 2025 (actual)
- First posted
- 24 Jun 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| High Dose Inspiratory Muscle Training | Other | Device: IMT therapy using the Pr02 mobile deviceInspiratory Muscle Training 3 times a week over 26 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IMT therapy using the Pr02 mobile deviceInspiratory Muscle Training using a device used to measure and increase respiratory strength and performance through resisted breathing exercises. | Device | High Dose Inspiratory Muscle Training |
Primary outcome
Change in maximum inspiratory pressure (MIP)
Time frame: Baseline, week 15, week 30
Change pre-test to post test, measured in cm H20
22 secondary outcomes
Change in maximum expiratory pressure (MEP)
Time frame: Baseline, week 15, week 30
Change in inspiratory power curve (IPC)
Time frame: Baseline, week 15, week 30
Change in inspiratory duration (ID)
Time frame: Baseline, week 15, week 30
Change in fatigue index test score (FIT)
Time frame: Baseline, week 15, week 30
Change in forced vital capacity (FVC)
Time frame: Baseline, week 15, week 30
Change in forced expiratory volume over 1 second (FEV1)
Time frame: Baseline, week 15, week 30
Change in peak expiratory flow (PEF)
Time frame: Baseline, week 15, week 30
Change in inspiratory phase duration (IPD)
Time frame: Baseline, week 15, week 30
Change in inspiratory peak flow (IPF)
Time frame: Baseline, week 15, week 30
Change in compression phase duration (CPD)
Time frame: Baseline, week 15, week 30
Change in expiratory phase rise time (EPRT)
Time frame: Baseline, week 15, week 30
Change in cough volume acceleration (CVA)
Time frame: Baseline, week 15, week 30
Change in fatigue
Time frame: Baseline, week 15, week 30
Change in impact of fatigue on quality of life (QOL)
Time frame: Baseline, week 15, week 30
Change in daytime sleepiness
Time frame: Baseline, week 15, week 30
Change in sleep quality
Time frame: Baseline, week 15, week 30
Change in respiratory symptoms
Time frame: Baseline, week 15, week 30
Change in motor performance
Time frame: Baseline, week 15, week 30
Change in health-related quality of life
Time frame: Baseline, week 15, week 30
Change in ability to communicate
Time frame: Baseline, week 15, week 30
Change in voice quality
Time frame: Baseline, week 15, week 30
Change in swallowing symptoms
Time frame: Baseline, week 15, week 30
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2025.
- Individual participant dataSharing planned: No
Related trials for Late-Onset Pompe Disease
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingObservational study
Pompe Disease Registry Protocol
Glycogen Storage Disease Type II, Pompe Disease
Not yet recruitingPhase 2
An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
Pompe Disease
RecruitingPhase 1/2
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
Pompe Disease (Late-onset), Pompe Disease Late-Onset
Active, not recruiting
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
Pompe Disease (Late-onset)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Harrison Jones, PhD principal investigator
Duke University
1 site in 1 country: see every site and its status.
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.