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A Study to Test Lung Stretch Therapy (Hyperinsufflation) to Slow the Rate of Decline in Children With Collagen VI Muscular Dystrophy

This study is currently recruiting participants.
Verified December 2013 by Children's Hospital Medical Center, Cincinnati
Sponsor:
Collaborator:
Cure CMD
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:
NCT01836627
First received: April 12, 2013
Last updated: December 11, 2013
Last verified: December 2013

April 12, 2013
December 11, 2013
April 2013
April 2016   (final data collection date for primary outcome measure)
Difference in rate of decline of lung vital capacity between the two groups [ Time Frame: Baseline and app. weeks 17, 34, and 52 ] [ Designated as safety issue: No ]
vital capacity (VC)= the volume of gas that can be expelled from the lungs from a position of full inspiration, with no limit to duration of inspiration; equal to inspiratory capacity plus expiratory reserve volume
To determine difference in rate of decline of lung vital capacity between the two groups [ Time Frame: Baseline and app. weeks 17, 34, and 52 ] [ Designated as safety issue: No ]
vital capacity (VC)= the volume of gas that can be expelled from the lungs from a position of full inspiration, with no limit to duration of inspiration; equal to inspiratory capacity plus expiratory reserve volume
Complete list of historical versions of study NCT01836627 on ClinicalTrials.gov Archive Site
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A Study to Test Lung Stretch Therapy (Hyperinsufflation) to Slow the Rate of Decline in Children With Collagen VI Muscular Dystrophy
Congenital Muscular Dystrophy (CMD) and the Feasibility of Hyperinsufflation Therapy to Slow Rate of Decline in Lung Volume

This research study includes children ages 5 to 20 years old with Collagen Type 6 Congenital Muscular Dystrophy. The goal of this study is to measure the effect of breathing exercise to stretch the chest in slowing the loss of breathing function. The breathing stretches are done with a machine called Cough Assist®.

The study is being done at Cincinnati Children's Hospital Medical Center and Children's Hospital of Philadelphia. The study involves traveling to one of these 2 centers for 4 visits over 13 months. The study also includes 3 sets of phone visits called Daily Phone Diaries.

Participants will be "randomized" into one of 2 study groups in a 1:1 ratio. The treatment group will use the Cough Assist® machine twice a day for 15 minutes. The control group will continue with their current daily care. The Cough Assist® is a machine that blows air into the lungs (insufflation) and helps pull air out of the lungs. The investigators will be blowing enough air into the lungs to cause a stretch to the chest. This is called hyperinsufflation.

Study visits will last about 5 to 6 hours and will include medical and quality of life questionnaires and pulmonary function tests to determine lung function and the individualized settings to be prescribed for the Cough Assist®.

Congenital muscular dystrophy (CMD) secondary to collagen VI deficiency is a distinct genetic neuromuscular disorder presenting from birth. Respiratory failure is the principal cause of morbidity and mortality from the disease. As CMD has no existing treatment, the majority of affected children develop respiratory failure and require non-invasive ventilation between the first and second decades of life. There is evidence that in muscular dystrophy the progressive decrease in lung volume is disproportionally greater than anticipated for the degree of respiratory muscle weakness. Chest wall stiffness and decreased lung elasticity which collectively diminish compliance of the respiratory system are believed to be important contributors to the disproportionate decrease in lung volume. The investigators' research aims at testing the hypothesis that in subjects with CMD Collagen VI, daily passive stretch of the chest wall through lung hyperinsufflation therapy could slow down the annual rate of decline in lung volume, delay the onset of respiratory failure and improve quality of life.

This is a randomized, controlled, interventional study. The intervention consists of an individualized hyperinsufflation titration protocol. The duration of the active protocol will last 12 months. Because CMD presents a limited subject pool, this study is a partnership with Cincinnati Children's Hospital of Cincinnati (CCHMC) and Children's Hospital of Philadelphia (CHOP) with approximately half of the studies being done at each site. Potential subjects will be identified through Cure CMD international registry and disease specific websites to meet the needed number of subjects.

At Visit 1, subjects will have their baseline assessments, QOL (quality of Life) questionnaires, and pressure titration and hyperinsufflation protocol to determine Peak Insufflation Pressure (PIP). Subjects will be given their randomization assignment into either the Treatment or Control arm. Both groups will have pressure titration and hyperinsufflation protocols for Peak Insufflation Pressure (PIP) at each study visit.

For the Treatment Group, Visit 1 will be followed by a variable run-in period of up to one month without study intervention to allow for central reading of the pressure titration and hyperinsufflation results and issuing of prescription for settings on the Cough Assist® device. Once the prescription is acquired, the 12 month interventional period will begin with 15 minute, twice daily hyperinsufflation treatments. All visits will be identical for both treatment and control group and will include all the activities from Visit 1 except for randomization.

There will be 3 scheduled phone visits for the Daily Phone Diary (DPD) encounters. The DPD is a phone-based diary that tracks patients and / or caregivers through their activities over the past 24 hours using a cued recall procedure. A set of two DPDs (one weekday and one weekend day) is conducted by phone at each of the 3 assessment points.

The investigators anticipate a significant number of subjects will live greater than 100 miles from the research centers. Travel arrangements will be made by family and paid for by the study through additional funding provided by Cure CMD.

Interventional
Not Provided
Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Investigator)
Primary Purpose: Treatment
  • Congenital Muscular Dystrophy
  • Collagen VI Deficiency
Other: Hyperinsufflation therapy
15 minutes twice a day of hyperinsufflation with Cough Assist® device
  • Experimental: Treatment Hyperinsufflation Therapy
    Treatment group will have 15 minute hyperinsufflation treatments twice a day for one year.
    Intervention: Other: Hyperinsufflation therapy
  • No Intervention: Control
    The control group will continue with their current daily care
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
34
April 2016
April 2016   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • 5 years through 20.9 years of age with vital capacity ≥30 and ≤80% predicted within the past 12 months with vital capacity based on the highest value in past 12 months
  • either gender and we will try to recruit equal numbers of male and female,
  • confirmed collagen VI CMD by gene mutation or muscle / skin biopsy

Exclusion Criteria:

  • a major medical condition such as diabetes, renal failure, hepatic failure, cancer, or other known systemic disease or any neuromuscular disorder other than the CMD group
  • inability to perform reliable Pulmonary Function Test (PFT)
  • tracheostomy
  • use of daytime ventilatory support. Patients on positive pressure during sleep or who require cough augmentation will not be excluded.
Both
5 Years to 20 Years
No
Contact: Kathleen Fields, BA, RC 513-636-0736 Kathleen.Fields@cchmc.org
Contact: Anne Rutkowski, MD 307-363-2630 anne.rutkowski@curecmd.org
United States
 
NCT01836627
CIN001CMDwHyperinsufflation
Yes
Children's Hospital Medical Center, Cincinnati
Children's Hospital Medical Center, Cincinnati
Cure CMD
Principal Investigator: Raouf S Amin, MD Cincinnati Childrens Hospital Medical Center
Children's Hospital Medical Center, Cincinnati
December 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP