High-dose inspiratory muscle training for Late-Onset Pompe Disease
High-dose Inspiratory Muscle Training (IMT) in Late-onset Pompe Disease (LOPD)
This study is testing if high-dose breathing exercises done at home can help people with Late-Onset Pompe Disease breathe better and improve their overall quality of life.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 29 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Duke University Academic / other |
| Locations | 1 site (Durham, North Carolina) |
| Trial ID | NCT05431127 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the safety and feasibility of high-dose inspiratory muscle training (IMT) delivered remotely to individuals with Late-Onset Pompe Disease (LOPD). The focus is on enhancing respiratory strength and function through a cost-effective intervention that addresses respiratory weakness. Participants will engage in IMT using the Pr02 mobile device, with the goal of improving respiratory muscle strength and overall health-related quality of life. The study will evaluate both respiratory outcomes and patient-reported outcomes to determine the efficacy of this approach.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 and older with a confirmed diagnosis of LOPD who are stable on their current treatment regimen.
Not a fit: Patients with severe comorbidities, those requiring continuous ventilation while awake, or those who cannot provide informed consent may not benefit from this study.
Why it matters
Potential benefit: If successful, this intervention could significantly improve respiratory function and quality of life for patients with LOPD.
How similar studies have performed: While the approach of high-dose IMT is innovative, similar studies have shown promise in enhancing respiratory function in other conditions, suggesting potential for success in this context.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Criteria: * 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
Where this trial is running
Durham, North Carolina
- Duke University Medical Center — Durham, North Carolina, United States (Recruiting)
Study contacts
- Principal investigator: Harrison Jones, PhD — Duke University
- Study coordinator: Kelly Crisp, MA, CCC-SLP
- Email: kelly.crisp@duke.edu
- Phone: 919-681-1852
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.