Duchenne Muscular DystrophyNCT01066455
Cardiac Outcome Measures in Children With Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy, run by Cooperative International Neuromuscular Research Group at 5 sites in United States.
Trial summary
The purpose of the research study is to evaluate different cardiac measures that are obtained by echocardiographic tests in patients with muscular dystrophy. Read more about this study
- Who is it for?
- Ages 8 to 18 years
- Conditions the study is for
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- How many people
- 48 participants
- Where
- 5 sitesUnited States
- Study dates
- Jan 2010to May 2012
- 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 8 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: 8 to 18 years old Confirmed diagnosis of Duchenne, Becker, or autosomal recessive limb-girdle muscular dystrophy (LGMD: 2C-2F and 2I)
Who may be able to join
- Participants must be between the ages of 8 and 18 years old
- Confirmed diagnosis of muscular dystrophy (DMD, BMD, or LGMD 2C-2F and 2I)
Who cannot join
- Investigator assessment of inability to comply with protocol
- History of a congenital cardiac defect or other cardiac disease unrelated to muscular dystrophy
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Texas Children's Hospital
Houston, Texas
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
University of California Davis
Sacramento, California
Washington University St. Louis
St Louis, Missouri
Children's National Medical Center
Washington D.C., District of Columbia
About this study
Official title: PITT1109: Cardiac Outcome Measures in Children With Muscular Dystrophy
The purpose of the research study is to evaluate different cardiac measures that are obtained by echocardiographic tests in patients with muscular dystrophy.
Read the detailed description
The research study will include 50 participants aged 8 to 18 years old (before 18th birthday) with Duchenne, Becker, or autosomal recessive limb-girdle (specifically: LGMD 2C-2F and 2I) muscular dystrophies.
Participants will be seen in one of five Cooperative International Neuromuscular Research Group (CINRG) centers located in the United States.
All study assessments will be completed in the same day and include reviewing past medical and surgical history, collecting vital signs, collecting cardiac measures through echocardiographic tests.
- Keywords
- Cardiac, Muscular Dystrophy, Prospective, Pediatric, Echocardiogram
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Limb-Girdle, Muscular Dystrophies
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2010 (estimated)
- Study startJan 2010
- Primary completionMay 2012
- Study completionMay 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Sponsor type
- Research network
- Collaborators
- National Institutes of Health (NIH)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01066455
- Sponsor's protocol ID
- PITT1109
- Status last verified
- Jan 2013
- First submitted
- 9 Feb 2010
- Last updated
- 11 Jan 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PITT1109: Cardiac Outcome Measures in Children With Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT01066455
- Acronym / sponsor ID
- PITT1109
- Keywords
- Cardiac, Muscular Dystrophy, Prospective, Pediatric, Echocardiogram
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Cooperative International Neuromuscular Research GroupResearch network
- Collaborators
- National Institutes of Health (NIH)
- Study start
- Jan 2010
- Primary completion
- May 2012 (actual)
- Study completion
- May 2012 (actual)
- First posted
- 10 Feb 2010 (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 the research study is to evaluate different cardiac measures that are obtained by echocardiographic tests in patients with muscular dystrophy.
Detailed description
The research study will include 50 participants aged 8 to 18 years old (before 18th birthday) with Duchenne, Becker, or autosomal recessive limb-girdle (specifically: LGMD 2C-2F and 2I) muscular dystrophies.
Participants will be seen in one of five Cooperative International Neuromuscular Research Group (CINRG) centers located in the United States.
All study assessments will be completed in the same day and include reviewing past medical and surgical history, collecting vital signs, collecting cardiac measures through echocardiographic tests.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 8 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- 8 to 18 years old Confirmed diagnosis of Duchenne, Becker, or autosomal recessive limb-girdle muscular dystrophy (LGMD: 2C-2F and 2I)
Key inclusion criteria (2 listed)
- Participants must be between the ages of 8 and 18 years old
- Confirmed diagnosis of muscular dystrophy (DMD, BMD, or LGMD 2C-2F and 2I)
Key exclusion criteria (2 listed)
- Investigator assessment of inability to comply with protocol
- History of a congenital cardiac defect or other cardiac disease unrelated to muscular dystrophy
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
Not reported in the source record.
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 11 Jan 2013.
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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.
The record lists no contact. Use the official record to reach the sponsor.
5 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 and Becker Muscular Dystrophy, run by Cooperative International Neuromuscular Research Group at 5 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 2 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 11 Jan 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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.
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 11 Jan 2013.