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

NCT01836627 | Completed DMC

• 2 other identifiers: CIN001CMDwHyperinsufflationR34HL113390
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 2

Brief Summary

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®.

Conditions

Congenital Muscular Dystrophy

Keywords

Congenital Muscular Dystrophy; Collagen VI Deficiency; Hyperinsufflation; Cough Assist; LAMA2- Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• Difference in rate of decline of lung vital capacity between the two groups

  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

  Baseline and app. weeks 17, 34, and 52

Study Arms (2)

Treatment Hyperinsufflation Therapy

EXPERIMENTAL

Treatment group will have 15 minute hyperinsufflation treatments twice a day for one year.

Other: Hyperinsufflation therapy

Control

NO INTERVENTION

The control group will continue with their current daily care

Interventions

Hyperinsufflation therapy OTHER

15 minutes twice a day of hyperinsufflation with Cough Assist® device

Treatment Hyperinsufflation Therapy

Eligibility Criteria

• Age: 5 Years - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• Cure CMD: collaborator
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (2)

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Study Officials

• Raouf S Amin, MD

  Cincinnati Childrens Hospital Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 12, 2013
• First Posted: April 22, 2013
• Study Start: April 1, 2013
• Primary Completion: April 1, 2016
• Study Completion: May 1, 2016
• Last Updated: July 26, 2016

Record last verified: 2016-07

Locations

US (2)