High Dose Inspiratory Muscle Training in LOPD

NCT05431127 | Completed

• 1 other identifier: Pro00109392
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 1

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.

Conditions

Late-Onset Pompe Disease; Lysosomal Disease

Keywords

LOPD

Outcome Measures

Primary Outcomes (1)

• Change in maximum inspiratory pressure (MIP)

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

  Baseline, week 15, week 30

Secondary Outcomes (22)

• Change in maximum expiratory pressure (MEP)

  Baseline, week 15, week 30

• Change in inspiratory power curve (IPC)

  Baseline, week 15, week 30

• Change in inspiratory duration (ID)

  Baseline, week 15, week 30

• Change in fatigue index test score (FIT)

  Baseline, week 15, week 30

• Change in forced vital capacity (FVC)

  Baseline, week 15, week 30

Study Arms (1)

High Dose Inspiratory Muscle Training

OTHER

Inspiratory Muscle Training 3 times a week over 26 weeks

Device: IMT therapy using the Pr02 mobile device

Interventions

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.

High Dose Inspiratory Muscle Training

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Duke University: lead
• Genzyme, a Sanofi Company: collaborator

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Study Officials

• Harrison Jones, PhD

  Duke University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 21, 2022
• First Posted: June 24, 2022
• Study Start: July 19, 2022
• Primary Completion: January 1, 2025
• Study Completion: January 1, 2025
• Last Updated: October 1, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)