Phase 4
N=70
Stacking Exercises Aid the Decline in FVC and Sick Time
Duchenne Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT01999075 ↗Enrolled (actual)
70
Serious AEs
0.0%
Results posted
Jan 2025
Primary outcome: Primary: Change in FVC (%-Predicted) From Baseline to 2 Years. — -9.2; -6.4 Mean difference in FVC %-predicted — p=0.68
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Lung Volume Recruitment (LVR) (Device); Conventional Treatment (Other)
- Age
- Pediatric · 6+ yrs
- Sex
- All
- Sponsor
- Children's Hospital of Eastern Ontario
- Primary completion
- Nov 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in FVC (%-Predicted) From Baseline to 2 Years. |
-9.2; -6.4 | 0.68 |
| SECONDARY FVC Decline of 10% of Predicted |
19.14; 21.70 | — |
| SECONDARY Number of Participants Prescribed Outpatient Oral Antibiotic Courses Between Baseline and 2 Years |
1; 2 | — |
| SECONDARY Health-related Quality of Life From Baseline to 2 Years |
2.0; 3.8 | — |
| SECONDARY Change in Difference Between Assisted and Unassisted Peak Cough Flow (PCF) From Baseline to 2 Years |
-2.6; 44.9 | — |
| SECONDARY Change in Maximal Insufflation Capacity (MIC)-Vital Capacity (VC) From Baseline to 2 Years |
0.1; 0.0 | 0.79 |
| SECONDARY Change in Maximum Inspiratory Pressures (MIP), From Baseline to 2 Years |
-0.5; -4.6 | 1.00 |
| SECONDARY Change in Maximal Expiratory Pressures (MEP), From Baseline to 2 Years |
5.5; 12.9 | 0.98 |
Summary
Duchenne Muscular Dystrophy is complicated by weak breathing muscles and lung infections. "Lung volume recruitment" is a technique performed using a face mask or mouthpiece and a hand-held resuscitation bag to stack breaths, inflate the lungs and help clear the airways of secretions by increasing the forcefulness of a cough. We believe this will slow down the steady loss of lung function, prevent lung infection, and improve quality of life. Our aim is to compare the outcome of a group of individuals with DMD treated with standard care to another group that also receives lung volume recruitment. If effective, this study will change clinical practice by including twice-daily treatment as part of the standard of care for individuals with DMD, in order to improve their lung health and quality of life.
Eligibility Criteria
Inclusion Criteria
- Age 6-16 years - This age range was selected as there are accepted normative pulmonary function data and children 6 years of age and older are generally able to reliably perform pulmonary function tests. Children are followed in participating centres until they reach 18 years of age (allowing two years of follow-up).
- Clinical phenotypic features consistent with DMD and confirmed by either: (1) Muscle biopsy showing complete dystrophin deficiency; (2) Genetic test positive for deletion or duplication in the dystrophin gene resulting in an 'out-of-frame' mutation; or (3) Dystrophin gene sequencing showing a mutation associated with DMD.
- FVC ≥ 30% predicted - This range of pulmonary function was selected to exclude those with severe restrictive respiratory impairment, who are less likely to be able to reliably perform pulmonary function testing over a two year period.
- A caregiver willing to provide the therapy
- Fluency in English or French
Exclusion Criteria
- Unable to perform pulmonary function tests and/or LVR manoeuvre
- Presence of an endotracheal or tracheostomy tube
- Already using LVR and/or the Respironics in-exsufflator between and during respiratory infections
- Known susceptibility to pneumothorax or pneumomediastinum
- Uncontrolled asthma or other obstructive lung disease
- Symptomatic cardiomyopathy (ejection fraction less than 50% )
Data sourced from ClinicalTrials.gov (NCT01999075). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.