Stacking Exercises Aid the Decline in FVC and Sick Time

NCT01999075 | Completed Phase 4 Results DMC

• 1 other identifier: 12/26E
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 9

Brief 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.

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne Muscular Dystrophy; Pulmonary complications; Lung volume recruitment,; Breath-stacking,; Cough efficacy,; Maximal insufflation capacity

Outcome Measures

Primary Outcomes (1)

• Change in FVC (%-Predicted) From Baseline to 2 Years.

  Change in FVC (%-predicted) was chosen as the primary outcome as it is a strong predictor of subsequent respiratory failure and mortality. Although survival is not a realistic endpoint for this trial, given expected mortality is less than 5% for the pediatric age group, FVC change is an appropriate clinical laboratory measure and valid surrogate endpoint to use for this trial.

  2 years

Secondary Outcomes (7)

• FVC Decline of 10% of Predicted

  2 years

• Number of Participants Prescribed Outpatient Oral Antibiotic Courses Between Baseline and 2 Years

  2 years

• Health-related Quality of Life From Baseline to 2 Years

  2 years

• Change in Difference Between Assisted and Unassisted Peak Cough Flow (PCF) From Baseline to 2 Years

  2 years

• Change in Maximal Insufflation Capacity (MIC)-Vital Capacity (VC) From Baseline to 2 Years

  2 years

Other Outcomes (2)

• Maximal and Average Pressure Achieved With LVR (cmH2O)

  2 years

• Respiratory Symptoms

  2 years

Study Arms (2)

Conventional Treatment

PLACEBO COMPARATOR

Conventional Treatment

Other: Conventional Treatment

Lung Volume Recruitment

ACTIVE COMPARATOR

Conventional treatment plus the use of Lung Volume Recruitment (LVR) twice per day

Device: Lung Volume Recruitment (LVR); Other: Conventional Treatment

Interventions

Lung Volume Recruitment (LVR) DEVICE

LVR will be used twice per day

Lung Volume Recruitment

Conventional Treatment OTHER

This may include: a. Physiotherapy, consisting of percussion, active cycle of breathing and/or postural drainage; b. Nutritional support, consisting of oral or tube-fed dietary supplements; c. Antibiotics (oral or intravenous), if there is evidence of respiratory infection; d. Non-invasive positive pressure ventilation, if there is evidence of nocturnal hypoventilation or sleep-disordered breathing; e. Systemic steroids

Conventional Treatment; Lung Volume Recruitment

Eligibility Criteria

• Age: 6 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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% )

Sponsors & Collaborators

• Children's Hospital of Eastern Ontario: lead
• Jesse's Journey: collaborator

Study Sites (9)

Alberta Children's Hospital

Calgary, Alberta, T3B 6A8, Canada

Location

Stollery Children's Hospital

Edmonton, Alberta, TGG 2J3, Canada

Location

BC Children's Hospital

Vancouver, British Columbia, V6H 3V4, Canada

Location

McMaster University

Hamilton, Ontario, L8S 4K1, Canada

Location

London Health Sciences

London, Ontario, N6A 4G5, Canada

Location

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada

Location

Holland Bloorview Kids Rehabilitation Hospital

Toronto, Ontario, M4G 1R8, Canada

Location

SickKids Hospital

Toronto, Ontario, M5G 1X8, Canada

Location

Hôpital Ste. Justine

Montreal, Quebec, H3T 1C5, Canada

Location

Related Publications (2)

• Katz SL, Mah JK, McMillan HJ, Campbell C, Bijelic V, Barrowman N, Momoli F, Blinder H, Aaron SD, McAdam LC, Nguyen TTD, Tarnopolsky M, Wensley DF, Zielinski D, Rose L, Sheers N, Berlowitz DJ, Wolfe L, McKim D. Routine lung volume recruitment in boys with Duchenne muscular dystrophy: a randomised clinical trial. Thorax. 2022 Aug;77(8):805-811. doi: 10.1136/thoraxjnl-2021-218196. Epub 2022 Mar 2.

  PMID: 35236763 DERIVED

• Morrow B, Argent A, Zampoli M, Human A, Corten L, Toussaint M. Cough augmentation techniques for people with chronic neuromuscular disorders. Cochrane Database Syst Rev. 2021 Apr 22;4(4):CD013170. doi: 10.1002/14651858.CD013170.pub2.

  PMID: 33887060 DERIVED

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Dr. Sherri Katz (Principal Investigator)
• Organization: CHEO Research Institute

skatz@cheo.on.ca

613-737-7600

Study Officials

• Sherri Katz, MD

  Children's Hospital of Eastern Ontario

  PRINCIPAL INVESTIGATOR

• Ian MacLusky, MD

  Children's Hospital of Eastern Ontario

  STUDY DIRECTOR

• Nicholas Barrowman, PhD

  Children's Hospital of Eastern Ontario

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Dr. Sherri Katz

Study Record Dates

• First Submitted: November 14, 2013
• First Posted: December 3, 2013
• Study Start: March 1, 2013
• Primary Completion: November 22, 2018
• Study Completion: November 22, 2018
• Last Updated: January 9, 2025
• Results First Posted: January 9, 2025

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

CA (9)