A Study to Test Lung Stretch Therapy (Hyperinsufflation) in Children With Collagen VI Muscular Dystrophy

Completed · Not applicable

Conditions studied: Congenital Muscular Dystrophy

In brief

This research study includes children ages 5 to 20 years old with Collagen Type 6 Congenital Muscular Dystrophy or Laminin α2-related muscular dystrophy (LAMA2-MD). 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®.

Key facts

Study ID: NCT01836627
Run by: Children's Hospital Medical Center, Cincinnati
People needed: 34
Starts: 2013-04-01
Expected to finish: 2016-05-01
Last updated by the study team: 2016-07-26

Who can join

Age: 5 and older, up to 20. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

5 years through 20.9 years of age
for non-ambulatory, subjects, vital capacity ≥30 and ≤ 80% predicted within the past 18 months,
either gender and we will try to recruit equal numbers of male and female, with vital capacity based on the highest value in past 12 months
confirmed collagen VI CMD by gene mutation or muscle / skin biopsy OR
confirmed LAMA2-MD by clinical history and muscle / skin biopsy or by gene mutation that are non-ambulatory and not ventilator dependent.

You may not qualify if…

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
PFT values for vital capacity >80 or < 30 %
Patients on positive pressure during sleep or who require cough augmentation will not be excluded.

Where it is running

Cincinnati Childrens Hospital Medical Center — Cincinnati, Ohio, United States
Childrens Hospital of Philadelphia — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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