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Trial record 1 of 1 for:    Stacking Exercises Aid the Decline in FVC and Sick Time
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Stacking Exercises Aid the Decline in FVC and Sick Time (STEADFAST)

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Jesse's Journey-The Foundation for Gene and Cell Therapy
Information provided by (Responsible Party):
Sherri Katz, Children's Hospital of Eastern Ontario
ClinicalTrials.gov Identifier:
NCT01999075
First received: November 14, 2013
Last updated: January 16, 2017
Last verified: January 2017
  Purpose
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.

Condition Intervention Phase
Duchenne Muscular Dystrophy Device: Lung Volume Recruitment (LVR) Other: Conventional Treatment Phase 4

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single Blind (Investigator)
Primary Purpose: Treatment
Official Title: Stacking Exercises Attenuate the Decline in Forced Vital Capacity and Sick Time (STEADFAST)

Resource links provided by NLM:


Further study details as provided by Sherri Katz, Children's Hospital of Eastern Ontario:

Primary Outcome Measures:
  • Relative decline in FVC (%-predicted) over 2 years, measured according to American Thoracic Society (ATS) standards, using the Stanojevic normative equations. [ Time Frame: 2 years ]
    Relative decline 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 decline is an appropriate clinical laboratory measure and valid surrogate endpoint to use for this trial.


Secondary Outcome Measures:
  • Time to FVC decline of 10% of predicted. [ Time Frame: 2 years ]
  • Total number and duration of outpatient oral antibiotic courses, hospital and ICU admissions for respiratory exacerbations over 2 years [ Time Frame: 2 years ]
  • Health-related quality of life over 2 years [ Time Frame: 2 years ]
    Measured biannually with PedsQL 4.0, Pediatric Quality of Life Inventory

  • Change in unassisted peak cough flow (PCF), maximal insufflation capacity (MIC), maximum inspiratory and expiratory pressures (MIP, MEP), as well as MIC and PCF with LVR, over 2 years [ Time Frame: 2 years ]

Other Outcome Measures:
  • Maximal and average pressure achieved with LVR (cmH2O) [ Time Frame: 2 years ]
  • Respiratory symptoms [ Time Frame: 2 years ]
    Respiratory symptoms, as assessed every 3 months by phone and personnel interview at clinic visits (Appendix 10_A self-report usage diary (Appendix 12)will be given to the participant to record daily activities to help with recall at the telephone follow ups

  • Satisfaction with LVR [ Time Frame: 2 years ]
    Satisfaction with LVR, as assessed every 3 months by phone


Estimated Enrollment: 110
Study Start Date: March 2013
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: August 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Conventional Treatment
Conventional Treatment
Other: Conventional Treatment
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
Active Comparator: Lung Volume Recruitment
Conventional treatment plus the use of Lung Volume Recruitment (LVR) twice per day
Device: Lung Volume Recruitment (LVR)
LVR will be used twice per day
Other: Conventional Treatment
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

Detailed Description:

Background: Respiratory complications are the primary cause of morbidity and mortality associated with childhood Duchenne Muscular Dystrophy (DMD). Involvement of the respiratory muscles leads to progressive hypoventilation and/or recurrent atelectasis and pneumonia secondary to decreased cough efficacy. Lung volume recruitment (LVR) is a means of stacking breaths to achieve maximal lung inflation (MIC), prevent micro-atelectasis, and improve cough efficacy. Although it has been recommended by some experts as the "standard of care" for individuals with neuromuscular disease, the strategy has not been widely implemented in DMD given the lack of clinical trials to date to support its efficacy as well as the additional burden of care required in a population already requiring multiple interventions.

Primary Objective: To determine whether LVR, in addition to conventional treatment, is successful in reducing decline from baseline in forced vital capacity (FVC) over 2 years (percent predicted, measured according to American Thoracic Society standards), compared to conventional treatment alone in children with DMD.

Secondary Objectives: To determine differences between children treated with LVR in addition to conventional treatment, compared to those treated with conventional treatment alone, in: (1) the number of courses of antibiotics, hospitalizations and intensive care admissions for respiratory exacerbations, (2) health-related quality of life, and (3) peak cough flow and other pulmonary function tests.

Methods: We propose a 3-year multi-centre randomized controlled trial involving fifteen tertiary care pediatric hospitals across Canada. The study population consists of boys aged 6-16 years with DMD and FVC ≥ 30% of predicted. A sample size of 110 participants will be enrolled. This has been informed by chart review and survey of participating centres to be feasible, and will be re-assessed with an ongoing internal pilot study. Intervention: Participants will be allocated with a minimization procedure to receive conventional treatment (non-invasive ventilation, nutritional supplementation, physiotherapy and/or antibiotics, as decided by the treating physician) or conventional treatment plus twice daily LVR exercises performed with an inexpensive, portable self-inflating resuscitation bag containing a one-way valve and a mouthpiece. Data Analysis: The primary outcome (change in percent predicted FVC over 2 years) will be compared between the two study groups using an analysis of co-variance (ANCOVA) that takes into account baseline FVC and minimization factors.

Importance: Decline in pulmonary function among children with DMD negatively affects quality of life and predicts mortality. The relatively simple strategy of LVR has the potential to optimize pulmonary function and reduce respiratory exacerbations, thereby improving quality of life for individuals with DMD. This study is novel in that it is the first randomized controlled trial of LVR. A major strength is that the results will give support or refute recommendations regarding inclusion of LVR in the standard of care for individuals with DMD worldwide.

  Eligibility

Ages Eligible for Study:   6 Years to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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% )
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01999075

Locations
Canada, Alberta
Alberta Children's Hospital
Calgary, Alberta, Canada, T3B 6A8
Stollery Children's Hospital
Edmonton, Alberta, Canada, TGG 2J3
Canada, British Columbia
BC Children's Hospital
Vancouver, British Columbia, Canada, V6H 3V4
Canada, Ontario
McMaster University
Hamilton, Ontario, Canada, L8S 4K1
London Health Sciences
London, Ontario, Canada, N6A 4G5
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Holland Bloorview Kids Rehabilitation Hospital
Toronto, Ontario, Canada, M4G 1R8
SickKids Hospital
Toronto, Ontario, Canada, M5G 1X8
Canada, Quebec
Hôpital Ste. Justine
Montreal, Quebec, Canada, H3T 1C5
Sponsors and Collaborators
Children's Hospital of Eastern Ontario
Jesse's Journey-The Foundation for Gene and Cell Therapy
Investigators
Principal Investigator: Sherri Katz, MD Children's Hospital of Eastern Ontario
Study Director: Ian MacLusky, MD Children's Hospital of Eastern Ontario
Study Director: Nicholas Barrowman, PhD Children's Hospital of Eastern Ontario
  More Information

Responsible Party: Sherri Katz, Dr. Sherri Katz, Children's Hospital of Eastern Ontario
ClinicalTrials.gov Identifier: NCT01999075     History of Changes
Other Study ID Numbers: 12/26E
Study First Received: November 14, 2013
Last Updated: January 16, 2017
Individual Participant Data  
Plan to Share IPD: No

Keywords provided by Sherri Katz, Children's Hospital of Eastern Ontario:
Duchenne Muscular Dystrophy
Pulmonary complications
Lung volume recruitment,
Breath-stacking,
Cough efficacy,
Maximal insufflation capacity

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on June 23, 2017