Inspiratory Muscle Training for Adults with Pompe Disease
Efficacy and Feasibility of Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease: a Multicentre, Cross-over Randomized Control Trial (RCT)
This study is testing if a special breathing exercise program can help adults with Late-onset Pompe Disease breathe better and improve their quality of life.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Fondazione Don Carlo Gnocchi Onlus Academic / other |
| Locations | 1 site (Milan) |
| Trial ID | NCT05951790 on ClinicalTrials.gov |
What this trial studies
This multicentre, randomized, controlled cross-over trial aims to evaluate the efficacy of Inspiratory Muscle Training (IMT) in adults with Late-onset Pompe Disease (LOPD). Participants will undergo a specific treatment regimen that includes aerobic exercise and IMT, with assessments of respiratory function and quality of life conducted at multiple time points over a year. The study will measure various respiratory parameters and utilize questionnaires to evaluate dyspnea and overall well-being. The trial will compare the effects of IMT alone versus IMT combined with air-stacking.
Who should consider this trial
Good fit: Ideal candidates are adults diagnosed with Late-onset Pompe Disease who have been on Enzyme Replacement Therapy for at least 12 months and have specific respiratory function criteria.
Not a fit: Patients with severe cardiologic or respiratory comorbidities or those who are completely dependent on ventilation may not benefit from this study.
Why it matters
Potential benefit: If successful, this intervention could improve respiratory function and quality of life for patients with Late-onset Pompe Disease.
How similar studies have performed: Other studies have shown promising results with similar approaches in improving respiratory function in neuromuscular diseases, suggesting potential for success in this trial.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Late On-set Pompe Disease diagnosis already in Enzyme Replacement Therapy (ERT) since 12 months * Forced Vital Capacity \<80% predicted or Forced Vital Capacity \>80% but with Postural Drop \>25-30% Exclusion Criteria: * Severe Cardiologic or Respiratory comorbidities * Complete dependence on ventilation
Where this trial is running
Milan
- IRCCS S. Maria Nascente - Fondazione Don Carlo Gnocchi — Milan, Italy (Recruiting)
Study contacts
- Principal investigator: Paolo Banfi, MD — IRCCS Fondazione Don Carlo Gnocchi
- Study coordinator: Elena Compalati, RT
- Email: ecompalati@dongnocchi.it
- Phone: 3209727740
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.