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Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne's Muscular Dystrophy
This study is currently recruiting participants.
Verified by Children's Healthcare of Atlanta, September 2006
Sponsored by: Children's Healthcare of Atlanta
Information provided by: Children's Healthcare of Atlanta
ClinicalTrials.gov Identifier: NCT00207857
  Purpose

Nearly all patients with Duchenne’s Muscular Dystrophy (DMD) have scoliosis. Posterior instrumented spinal fusion, which is a surgery to correct scoliosis, has been shown to improve quality of life and satisfaction of both parents and families. The progressive muscular weakness leads to the development of scoliosis soon after the child has become unable to walk. The muscular weakness and scoliosis also affect the pulmonary function of these children. Pulmonary Function Tests (PFT) have been used to determine “pulmonary fitness” prior to surgery as a way to determine how well or if the child will tolerate surgery. Children with poor results on the PFT are determined to be too fragile to tolerate such a large operation. The physicians conducting this study feel that the PFT may be inaccurate and that this may not be the best single test to determine “pulmonary fitness”. The physicians conducting the study think things like the time of day the study is done, how tired you are when you complete the test, and how well you understand the test may affect the results of the test.


Condition
Duchenne's Muscular Dystrophy
Scoliosis

Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy L1 syndrome
MedlinePlus related topics: Muscular Dystrophy Scoliosis
U.S. FDA Resources
Study Type: Observational
Study Design: Natural History, Longitudinal, Defined Population, Prospective Study
Official Title: Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne's Muscular Dystrophy

Further study details as provided by Children's Healthcare of Atlanta:

Estimated Enrollment: 100
Study Start Date: March 2004
Estimated Study Completion Date: August 2005
Detailed Description:

All patients with DMD have routine PFT’s every six months as standard of care. You will be randomized in to one of two groups. The group assignment will be chosen by chance, similar to flipping a coin. If you are assigned to group one you will first have your regularly scheduled PFT in the morning. You will be asked to complete a second PFT that afternoon as least 4 hours after the first PFT. You will then be asked to come back to the clinic in two weeks and repeat these PFT’s, once in the morning and once in the afternoon at least 4 hours apart. Each PFT will take approximately 10 to 15 minutes. If you are assigned to group two, you will have your regularly scheduled PFT in the morning and then a second test in the afternoon at least four hours after the first test. After the second PFT you will receive training from a Respiratory Therapist. You will receive teaching handouts to take home and an incentive spirometer and peak flow meter to practice with. You will be asked to practice these tests 2 times a day and record your results. You will then be asked to come back to the clinic in two weeks to repeat 2 PFT’s, once in the morning and again at least 4 hours later. All participants will be asked how they feel at the time of testing.

  Eligibility

Ages Eligible for Study:   6 Years to 21 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male
  • Duchenne’s Muscular Dystrophy with diagnosed confirmed by a neurologist
  • No longer able to ambulate for any meaningful amount of time
  • No previous spinal operation

Exclusion Criteria:

  • Any other type of muscular dystrophy or spinal muscular atrophy
  • Tracheotomy
  • Contractures of the upper extremities that would preclude using arm span as an estimation for height
  • Previous spinal surgery
  • Asthma, recurrent pneumonia, or other chronic lung disease
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00207857

Contacts
Contact: Elena Morales, BS elena.morales@choa.org

Locations
United States, Georgia
Children's Healthcare of Atlanta @ Scottish Rite Recruiting
Atlanta, Georgia, United States, 30342
Contact: Elena Morales, BS         elena.morales@choa.org    
Principal Investigator: Tim Schrader, MD            
Sponsors and Collaborators
Children's Healthcare of Atlanta
Investigators
Principal Investigator: Tim Schrader, MD Children's Orthopaedics of Atlanta
  More Information

Study ID Numbers: Duchenne's Muscular Dystrophy
Study First Received: September 13, 2005
Last Updated: September 6, 2006
ClinicalTrials.gov Identifier: NCT00207857  
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Healthcare of Atlanta:
Duchenne's Muscular Dystrophy
Scoliosis

Study placed in the following topic categories:
Spinal Diseases
Scoliosis
Muscular dystrophy, Duchenne and Becker type
Bone Diseases
Muscular Dystrophies
Muscular Diseases
Becker's muscular dystrophy
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Muscular Dystrophy, Duchenne
Genetic Diseases, X-Linked
Duchenne muscular dystrophy
Atrophy
Muscular dystrophy

Additional relevant MeSH terms:
Spinal Curvatures
Nervous System Diseases

ClinicalTrials.gov processed this record on January 16, 2009