Duchenne Muscular DystrophyNCT00312247
Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy, run by Shriners Hospitals for Children at 3 sites in United States.
Trial summary
The purpose of this research study is to understand the walking patterns, strength and function changes of boys with Duchenne muscular dystrophy on/off corticosteroids to determine the best timing and treatment options to maintain walking for as long as possible. Read more about this study
- Who is it for?
- Ages 4 to 21 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 85 participants
- Where
- 3 sitesUnited States
- Study dates
- Apr 2006to Mar 2015
- 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 4 to 21 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Boys with DMD who are ambulatory starting at the age of 4 until ambulation ceases
Who may be able to join
- Confirmed diagnosis of DMD
- Male.
- Four years of age or older.
- Ability to walk independently for five minutes to 10 minutes at self-selected speed.
- Ability to cognitively understand directions for testing procedures.
Who cannot join
- Female
- Nonambulatory
Trial locations and contacts
3 sites in 1 country.
United States3 sites
UCLA Department of Orthopaedic Surgery
Los Angeles, California, 90095
Shriners Hospitals for Children
Portland, Oregon, 97239
Shriners Hospitals for Children
Sacramento, California, 95817
About this study
The purpose of this research study is to understand the walking patterns, strength and function changes of boys with Duchenne muscular dystrophy on/off corticosteroids to determine the best timing and treatment options to maintain walking for as long as possible.
Read the detailed description
Duchenne muscular dystrophy (DMD) is an X-linked recessive disease of muscle characterized by a progressive loss of functional muscle mass, which is replaced with fibrofatty tissue. Historically, boys with DMD lose the ability to walk between the ages of 8-12 years, due to progressive weakness of the quadriceps coupled with the development of contractures at the hip, knee and ankle. This progressive loss in function necessitates individuals with DMD to spend less time walking and more time in wheelchairs, leading to the development of spinal deformities. Recently, corticosteroids have been shown to reduce the expected loss of muscle strength, extend the time that ambulation and standing are maintained, and minimize or eliminate spinal deformity in individuals with DMD; yet, the side effects of such treatment preclude use in some patients. To date, differences in gait patterns and other markers of disease progression between boys on corticosteroids and those not utilizing such treatment have not been objectively quantified. This lack of knowledge is a major obstacle to determining the most effective treatment for subsets of boys with DMD.
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Walking, Muscle Strength, Energy Cost, Quality of Life
- Medical subject headings
- Muscular Dystrophy, Duchenne
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 |
|---|---|---|
| Boys taking steroids | Boys who are taking prednisone or deflazacort | |
| Boys who are steroid naive | Boys who are not taking steroids for a variety of reasons |
What is being measured
The primary outcome is the main question the study is designed to answer.
Gait pattern
computerized assessment of walking
Measured: every six months (2x/year)
Secondary outcomes (4)
muscle strength
quantitative assessment of strength with a Biodex
Measured: every six months (2x/year)
energy cost of walking
assessment of how much energy it takes to walk, assessed with a Cosmed K4b2
Measured: every six months (2x/year)
gross motor functional skills
assessment of gross motor skills, ie getting up off the floor, ascending/descending stairs
Measured: every six months (2x/year)
Step activity Monitor-participation
measurement of the number of steps taken in the community/home environment during weekdays and weekends
Measured: one week every six months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Apr 2006 (estimated)
- Study startApr 2006
- Primary completionDec 2014
- Study completionMar 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Duchenne 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
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 85 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shriners Hospitals for Children
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Michael D. Sussman, MD, Shriners Hospitals for Children
- Investigators
- Michael D Sussman, MD, Principal investigator, Shriners Hospitals for Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00312247
- Sponsor's protocol ID
- SHC-DMD-79115
- Other IDs
- SHC-79115
- Status last verified
- May 2015
- First submitted
- 5 Apr 2006
- Last updated
- 19 May 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Sienko Thomas S, Buckon CE, Nicorici A, Bagley A, McDonald CM, Sussman MD. Classification of the gait patterns of boys with Duchenne muscular dystrophy and their relationship to function. J Child Neurol. 2010 Sep;25(9):1103-9. doi: 10.1177/0883073810371002. Epub 2010 Jun 29.
PubMed 20587736McDonald CM, McDonald DA, Bagley A, Sienko Thomas S, Buckon CE, Henricson E, Nicorici A, Sussman MD. Relationship between clinical outcome measures and parent proxy reports of health-related quality of life in ambulatory children with Duchenne muscular dystrophy. J Child Neurol. 2010 Sep;25(9):1130-44. doi: 10.1177/0883073810371509. Epub 2010 Jun 17.
PubMed 20558672
Study at a glance
From the registry record, last updated by the sponsor on 19 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT00312247
- Acronym / sponsor ID
- SHC-DMD-79115
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Walking, Muscle Strength, Energy Cost, Quality of Life
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 85 participants (actual)
- Lead sponsor
- Shriners Hospitals for ChildrenUniversity, hospital or other
- Study start
- Apr 2006
- Primary completion
- Dec 2014 (actual)
- Study completion
- Mar 2015 (actual)
- First posted
- 7 Apr 2006 (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.
The purpose of this research study is to understand the walking patterns, strength and function changes of boys with Duchenne muscular dystrophy on/off corticosteroids to determine the best timing and treatment options to maintain walking for as long as possible.
Detailed description
Duchenne muscular dystrophy (DMD) is an X-linked recessive disease of muscle characterized by a progressive loss of functional muscle mass, which is replaced with fibrofatty tissue. Historically, boys with DMD lose the ability to walk between the ages of 8-12 years, due to progressive weakness of the quadriceps coupled with the development of contractures at the hip, knee and ankle. This progressive loss in function necessitates individuals with DMD to spend less time walking and more time in wheelchairs, leading to the development of spinal deformities. Recently, corticosteroids have been shown to reduce the expected loss of muscle strength, extend the time that ambulation and standing are maintained, and minimize or eliminate spinal deformity in individuals with DMD; yet, the side effects of such treatment preclude use in some patients. To date, differences in gait patterns and other markers of disease progression between boys on corticosteroids and those not utilizing such treatment have not been objectively quantified. This lack of knowledge is a major obstacle to determining the most effective treatment for subsets of boys with DMD.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Boys taking steroids | – | –Boys who are taking prednisone or deflazacort |
| Boys who are steroid naive | – | –Boys who are not taking steroids for a variety of reasons |
Who may qualify
- Ages
- 4 to 21 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Boys with DMD who are ambulatory starting at the age of 4 until ambulation ceases
Key inclusion criteria (5 listed)
- Confirmed diagnosis of DMD
- Male.
- Four years of age or older.
- Ability to walk independently for five minutes to 10 minutes at self-selected speed.
- Ability to cognitively understand directions for testing procedures.
Key exclusion criteria (2 listed)
- Female
- Nonambulatory
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
Gait pattern
Time frame: every six months (2x/year)
computerized assessment of walking
4 secondary outcomes
muscle strength
Time frame: every six months (2x/year)
energy cost of walking
Time frame: every six months (2x/year)
gross motor functional skills
Time frame: every six months (2x/year)
Step activity Monitor-participation
Time frame: one week every six months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 May 2015.
- Results publication: PubMed 20587736Sienko Thomas S, Buckon CE, Nicorici A, Bagley A, McDonald CM, Sussman MD. Classification of the gait patterns of boys with Duchenne muscular dystrophy and their relationship to function. J Child Neurol. 2010 Sep;25(9):1103-9. doi: 10.1177/0883073810371002. Epub 2010 Jun 29.
- Results publication: PubMed 20558672McDonald CM, McDonald DA, Bagley A, Sienko Thomas S, Buckon CE, Henricson E, Nicorici A, Sussman MD. Relationship between clinical outcome measures and parent proxy reports of health-related quality of life in ambulatory children with Duchenne muscular dystrophy. J Child Neurol. 2010 Sep;25(9):1130-44. doi: 10.1177/0883073810371509. Epub 2010 Jun 17.
- Shriners Hospitals for ChildrenLink provided by the sponsor.
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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.
Michael D Sussman, MD principal investigator
Shriners Hospitals for Children
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy, run by Shriners Hospitals for Children at 3 sites 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 5 inclusion and 2 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: finished, as of the sponsor's last update on 19 May 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Gait pattern (time frame: every six months (2x/year)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00312247, which also lists 2 linked publications.
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 19 May 2015.