Late-Onset Pompe Disease Clinical Trial
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.
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. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Terminated |
NCT02191917 -
A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)
|
||
| Terminated |
NCT03347253 -
STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
|
||
| Terminated |
NCT01924845 -
BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
|
Phase 3 |