Duchenne's Muscular DystrophyNCT00207857
Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne's Muscular Dystrophy
An observational study of Duchenne's Muscular Dystrophy and Scoliosis, run by Children's Healthcare of Atlanta at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 6 to 21 years, men only
- Conditions the study is for
- Duchenne's Muscular Dystrophy, Scoliosis
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2004to Aug 2005
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 21 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Healthcare of Atlanta @ Scottish Rite
Atlanta, Georgia, 30342
About this study
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.
Read the 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.
- Conditions
- Duchenne's Muscular Dystrophy, Scoliosis
- Keywords
- Duchenne's Muscular Dystrophy, Scoliosis
- Medical subject headings
- Muscular Dystrophy, Duchenne, Scoliosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| With and Without PFTs | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
PFT training
Measured: 2 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2005 (estimated)
- Study startMar 2004
- Primary completionAug 2005
- Study completionAug 2005 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne's Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Healthcare of Atlanta
- Sponsor type
- University, hospital or other
- Investigators
- Tim Schrader, MD, Principal investigator, Children's Orthopaedics of Atlanta
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00207857
- Sponsor's protocol ID
- Duchenne's Muscular Dystrophy
- Status last verified
- Feb 2015
- First submitted
- 13 Sep 2005
- Last updated
- 4 Feb 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Feb 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne's Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT00207857
- Acronym / sponsor ID
- Duchenne's Muscular Dystrophy
- Conditions
- Duchenne's Muscular Dystrophy, Scoliosis
- Keywords
- Duchenne's Muscular Dystrophy, Scoliosis
- Study type
- Observational
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Children's Healthcare of AtlantaUniversity, hospital or other
- Study start
- Mar 2004
- Primary completion
- Aug 2005 (actual)
- Study completion
- Aug 2005 (estimated)
- First posted
- 21 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| With and Without PFTs | – | – |
Who may qualify
- Ages
- 6 to 21 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
PFT training
Time frame: 2 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Feb 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Tim Schrader, MD principal investigator
Children's Orthopaedics of Atlanta
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne's Muscular Dystrophy and Scoliosis, run by Children's Healthcare of Atlanta at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 4 Feb 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PFT training (time frame: 2 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Feb 2015.