Duchenne Muscular DystrophyNCT01125709
Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS)
An observational study of Duchenne Muscular Dystrophy, run by Cooperative International Neuromuscular Research Group at 5 sites in 3 countries.
Trial summary
The aim of the proposed research is to compare two commonly used pediatric strength testing measures: handheld myometry (HHM) and CINRG Quantitative Measurement System (CQMS), with the goal of identifying a sensitive and valid tool for measuring muscle strength in children with DMD. Read more about this study
- Who is it for?
- Ages 6 to 18 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 30 participants
- Where
- 5 sitesin 3 countries
- Study dates
- Jan 2010to Aug 2010
- 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 18 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Participants will be selected from participating CINRG institution's neuromuscular clinics.
Who may be able to join
None listed.
Who cannot join
- Participants must confirm:
- No Surgical procedures were performed ≤ 8 weeks before study procedures.
- No musculoskeletal injuries were experienced ≤ 8 weeks before study procedures.
- Investigator assessment that patient or parent/legal guardian are not willing or able to comply with study procedures.
Trial locations and contacts
5 sites in 3 countries.
5 sites in 3 countries
United States3 sites
Carolinas Medical Center
Charlotte, North Carolina, 28207
Washington University - St. Louis
St Louis, Missouri, 63110
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: Comparative Study of Clinical Endpoint in DMD: HHM vs. CQMS
The aim of the proposed research is to compare two commonly used pediatric strength testing measures: handheld myometry (HHM) and CINRG Quantitative Measurement System (CQMS), with the goal of identifying a sensitive and valid tool for measuring muscle strength in children with DMD. The data obtained from this study will be used to make recommendations for strength measurement endpoints in prospective muscular dystrophy trials and provide more reliable and accurate recommendations in the clinic for strength assessment. This study will be performed at six participating sites in the Cooperative International Neuromuscular Research Group (CINRG).
Read the detailed description
We propose to compare the reliability of CQMS vs. HHM in the DMD population. The importance of this study is to be able to compare the results of clinical trials done by different networks using distinct strength endpoints. If results indicate a distinct difference in reliability of one tool over the other, a standardized tool could be established for research groups throughout the world to interpret strength in the context of clinical trials. If the results indicate minor differences then it would be possible to interpret and compare/contrast strength measurements used in different studies.
Understanding the relationship between the HHM vs CQMS will help us examine other surrogate measures capable of predicting functionality that are based on strength measurements.
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Neuromuscular disease, Duchenne, Muscular Dystrophy, DMD, Strength assessment, CINRG Quantitative Measurement System, CQMS, Hand Held Myometry, HHM
- Medical subject headings
- Muscular Dystrophy, Duchenne, Neuromuscular Diseases, Muscular Dystrophies
What is being measured
The primary outcome is the main question the study is designed to answer.
Compare the inter and intra rater reliability of HHM and CQMS by measuring Elbow and Knee Flexor/Extensor Strength in children ages 6-18 diagnosed with DMD tested by experienced clinical evaluators in both HHM and CQMS.
Muscle groups will be tested in a standardized order 1. Knee extension 2. Knee flexion 3. Elbow Flexion 4. Elbow extension with all tests sequencing following a right to left pattern. This will reduce assessment bias and the impact of muscle fatigue per muscle group. Study participants are randomized to two different sequences of four assessments, one sequence performed on one testing day (Visit 1) and another on a different testing day (Visit 2).
Measured: two-day visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2010 (estimated)
- Study startJan 2010
- Primary completionJun 2010
- Study completionAug 2010
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 crossover
- Time perspective
- Prospective
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Sponsor type
- Research network
- Collaborators
- Muscular Dystrophy Association, Children's National Research Institute, Washington University School of Medicine, Royal Children's Hospital, Sydney Children's Hospitals Network, Fondazione Serena Onlus - Centro Clinico NeMO Milano, Carolinas Medical Center
- Investigators
- Tina T Duong, MPT, Principal investigator, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01125709
- Sponsor's protocol ID
- CNMC0609
- Status last verified
- Jan 2013
- First submitted
- 17 May 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
- Comparative Study of Clinical Endpoint in DMD: HHM vs. CQMS
- ClinicalTrials.gov ID
- NCT01125709
- Acronym / sponsor ID
- CNMC0609
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Neuromuscular disease, Duchenne, Muscular Dystrophy, DMD, Strength assessment, CINRG Quantitative Measurement System, CQMS, Hand Held Myometry
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Cooperative International Neuromuscular Research GroupResearch network
- Collaborators
- Muscular Dystrophy Association, Children's National Research Institute, Washington University School of Medicine, Royal Children's Hospital, Sydney Children's Hospitals Network, Fondazione Serena Onlus - Centro Clinico NeMO Milano, Carolinas Medical Center
- Study start
- Jan 2010
- Primary completion
- Jun 2010 (actual)
- Study completion
- Aug 2010 (actual)
- First posted
- 18 May 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 aim of the proposed research is to compare two commonly used pediatric strength testing measures: handheld myometry (HHM) and CINRG Quantitative Measurement System (CQMS), with the goal of identifying a sensitive and valid tool for measuring muscle strength in children with DMD. The data obtained from this study will be used to make recommendations for strength measurement endpoints in prospective muscular dystrophy trials and provide more reliable and accurate recommendations in the clinic for strength assessment. This study will be performed at six participating sites in the Cooperative International Neuromuscular Research Group (CINRG).
Detailed description
We propose to compare the reliability of CQMS vs. HHM in the DMD population. The importance of this study is to be able to compare the results of clinical trials done by different networks using distinct strength endpoints. If results indicate a distinct difference in reliability of one tool over the other, a standardized tool could be established for research groups throughout the world to interpret strength in the context of clinical trials. If the results indicate minor differences then it would be possible to interpret and compare/contrast strength measurements used in different studies.
Understanding the relationship between the HHM vs CQMS will help us examine other surrogate measures capable of predicting functionality that are based on strength measurements.
Study design
- Observational model
- Case crossover
- Time perspective
- Prospective
Who may qualify
- Ages
- 6 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Participants will be selected from participating CINRG institution's neuromuscular clinics.
Key inclusion criteria (0 listed)
Key exclusion criteria (4 listed)
- No Surgical procedures were performed ≤ 8 weeks before study procedures.
- No musculoskeletal injuries were experienced ≤ 8 weeks before study procedures.
- Investigator assessment that patient or parent/legal guardian are not willing or able to comply with study procedures.
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
Compare the inter and intra rater reliability of HHM and CQMS by measuring Elbow and Knee Flexor/Extensor Strength in children ages 6-18 diagnosed with DMD tested by experienced clinical evaluators in both HHM and CQMS.
Time frame: two-day visit
Muscle groups will be tested in a standardized order 1. Knee extension 2. Knee flexion 3. Elbow Flexion 4. Elbow extension with all tests sequencing following a right to left pattern. This will reduce assessment bias and the impact of muscle fatigue per muscle group. Study participants are randomized to two different sequences of four assessments, one sequence performed on one testing day (Visit 1) and another on a different testing day (Visit 2).
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.
- CINRG website where all current and completed protocols are listed.Link 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.
Tina T Duong, MPT principal investigator
Children's National Research Institute
5 sites in 3 countries: 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 Cooperative International Neuromuscular Research Group at 5 sites in 3 countries.
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 0 inclusion and 4 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, Australia, Italy. 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?
Compare the inter and intra rater reliability of HHM and CQMS by measuring Elbow and Knee Flexor/Extensor Strength in children ages 6-18 diagnosed with DMD tested by experienced clinical evaluators in both HHM and CQMS. (time frame: two-day visit). 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 11 Jan 2013.