Duchenne Muscular DystrophyDMDNCT01098708
This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy, run by Washington University School of Medicine at 1 site in United States.
Trial summary
Many therapeutic trials in DMD exclude non-ambulatory boys and men. Rate of progression in these non-ambulatory patients has been studied but consensus has not been reached for what measures are most reliable and reproducible. Read more about this study
- Who is it for?
- Ages 7 to 22 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2010to Nov 2013
- 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 7 to 22 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Non ambulatory boys and men with Duchenne muscular dystrophy
Who may be able to join
- For Non-ambulatory study, boys must be unable to walk without assistive devices for greater than or equal to one year.
- Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years through age 22 years.
- Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with biopsy/genetic confirmation in a primary relative.
Who cannot join
- 1. For Non-ambulatory study, the inability to understand and cooperate with the testing would exclude a subject. -
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Clinical Outcomes Validation in Non Ambulatory and Young Boys/Men With Duchenne Muscular Dystrophy (DMD)
Many therapeutic trials in DMD exclude non-ambulatory boys and men. Rate of progression in these non-ambulatory patients has been studied but consensus has not been reached for what measures are most reliable and reproducible. Furthermore, any treatment trial would be expected to demonstrate improved function and improvement in quality of life. Therefore, function, strength, and quality of life must be understood and standardized. While the goal of this proposal is to standardize clinical outcomes for therapeutic trials, careful understanding of the progression of DMD in non ambulatory boys may also lead to better medical treatment.
Read the detailed description
For Non-ambulatory study, boys must be unable to walk without assistive devices for greater than or equal to one year.
Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years through age 22 years.
Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with biopsy/genetic confirmation in a primary relative.
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, non ambulatory
- Medical subject headings
- Muscular Dystrophy, Duchenne
What is being measured
The primary outcome is the main question the study is designed to answer.
Dystrophin gene mutations that predispose to early onset cardiomyopathy
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2010 (estimated)
- Study startMar 2010
- Primary completionNov 2013
- Study completionNov 2013
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.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- 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
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Nationwide Children's Hospital, Boston Children's Hospital, University of Minnesota, University of California, Davis
- Responsible party
- Principal investigator, Anne Connolly, Washington University School of Medicine
- Investigators
- Anne Connolly, M.D., Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01098708
- Sponsor's protocol ID
- 10-0067
- Acronym
- DMD
- Status last verified
- Jan 2017
- First submitted
- 1 Apr 2010
- Last updated
- 18 Jan 2017
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Outcomes Validation in Non Ambulatory and Young Boys/Men With Duchenne Muscular Dystrophy (DMD)
- ClinicalTrials.gov ID
- NCT01098708
- Acronym / sponsor ID
- DMD / 10-0067
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, non ambulatory
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Collaborators
- Nationwide Children's Hospital, Boston Children's Hospital, University of Minnesota, University of California, Davis
- Study start
- Mar 2010
- Primary completion
- Nov 2013 (actual)
- Study completion
- Nov 2013 (actual)
- First posted
- 5 Apr 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.
Many therapeutic trials in DMD exclude non-ambulatory boys and men. Rate of progression in these non-ambulatory patients has been studied but consensus has not been reached for what measures are most reliable and reproducible. Furthermore, any treatment trial would be expected to demonstrate improved function and improvement in quality of life. Therefore, function, strength, and quality of life must be understood and standardized. While the goal of this proposal is to standardize clinical outcomes for therapeutic trials, careful understanding of the progression of DMD in non ambulatory boys may also lead to better medical treatment.
Detailed description
For Non-ambulatory study, boys must be unable to walk without assistive devices for greater than or equal to one year.
Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years through age 22 years.
Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with biopsy/genetic confirmation in a primary relative.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 7 to 22 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Non ambulatory boys and men with Duchenne muscular dystrophy
Key inclusion criteria (3 listed)
- For Non-ambulatory study, boys must be unable to walk without assistive devices for greater than or equal to one year.
- Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years through age 22 years.
- Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with biopsy/genetic confirmation in a primary relative.
Key exclusion criteria (1 listed)
- 1. For Non-ambulatory study, the inability to understand and cooperate with the testing would exclude a subject. -
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
Dystrophin gene mutations that predispose to early onset cardiomyopathy
Time frame: 3 years
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 18 Jan 2017.
- Individual participant dataSharing planned: No
Related trials for Duchenne Muscular Dystrophy
Active, not recruitingPhase 1
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
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Recruiting
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
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An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
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RecruitingObservational study
Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy?
Duchenne Muscular Dystrophy (DMD), Chronic Pain
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Bone Health in Adults With Duchenne Muscular Dystrophy: A Survey on Osteoporosis, Fractures and Treatment.
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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.
Anne Connolly, M.D. principal investigator
Washington University School of Medicine
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 Muscular Dystrophy, run by Washington University School of Medicine 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 3 inclusion and 1 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 18 Jan 2017. 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 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?
Dystrophin gene mutations that predispose to early onset cardiomyopathy (time frame: 3 years). 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 18 Jan 2017; AzTrials last refreshed its copy on 29 Oct 2026.