Status:
COMPLETED
A Study to Test Lung Stretch Therapy (Hyperinsufflation) in Children With Collagen VI Muscular Dystrophy
Lead Sponsor:
Children's Hospital Medical Center, Cincinnati
Collaborating Sponsors:
Cure CMD
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Congenital Muscular Dystrophy
Eligibility:
All Genders
5-20 years
Phase:
NA
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 measur...
Detailed Description
Congenital muscular dystrophy (CMD) secondary to collagen VI deficiency and Laminin α2-related muscular dystrophy (LAMA2-MD) are distinct genetic neuromuscular disorders presenting from birth. Respira...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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.
Key Trial Info
Start Date :
April 1 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2016
Estimated Enrollment :
34 Patients enrolled
Trial Details
Trial ID
NCT01836627
Start Date
April 1 2013
End Date
May 1 2016
Last Update
July 26 2016
Active Locations (2)
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1
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, United States, 45229
2
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104