Status:
COMPLETED
High Dose Inspiratory Muscle Training in LOPD
Lead Sponsor:
Duke University
Collaborating Sponsors:
Genzyme, a Sanofi Company
Conditions:
Late-Onset Pompe Disease
Lysosomal Disease
Eligibility:
All Genders
18+ years
Phase:
NA
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 ...
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 int...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
July 19 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2025
Estimated Enrollment :
34 Patients enrolled
Trial Details
Trial ID
NCT05431127
Start Date
July 19 2022
End Date
January 1 2025
Last Update
October 1 2025
Active Locations (1)
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1
Duke University Medical Center
Durham, North Carolina, United States, 27705