Duchenne Muscular DystrophyNCT01523964
DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls
A study testing Testing with EIM in people with Duchenne Muscular Dystrophy, run by Dart Therapeutics. LLC at 6 sites in United States.
Trial summary
Physicians seek a method to assess neuromuscular disease that is both non-invasive and quantifiable. Many patients do not tolerate standard current day assessment tools (such as needle electromyogram), and Electrical Impedance Myography (EIM) has the potential to serve as a non-invasive, quantifiable, diagnostic tool… Read more about this study
- Who is it for?
- Ages 3 to 12 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Testing with EIM
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 92 participants
- Where
- 6 sitesUnited States
- Study dates
- Feb 2012to Aug 2012
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 3 to 12 years
- Men only
- Healthy volunteers accepted
- Child
Who may be able to join
- DMD Subject Cohorts
- Male subjects
- Subjects with a chronologic age of 3 to 7 years inclusive for Cohort A, and 8 to 12 years inclusive for Cohort B
- Subjects with DMD diagnosed with mutational testing and/or absence of dystrophin on muscle biopsy
- Subjects with proximal pelvic girdle weakness (Gower's maneuver, difficulty with arising from floor and going up steps)
- Subjects who can walk 10 meters unassisted (ie, without braces, canes, or other aids)
Show all 14Show fewer
- Subjects who are taking systemic corticosteroids and/or any other medication which, in the judgment of the investigator, could impact muscle strength or physical activity levels, must be on a stable dose for at least 4 weeks prior to initiation of study measurements
- Subjects who provide assent, as stipulated by IRB requirements, and whose parent/guardian signs an informed consent form
- Subjects who are willing and able to cooperate and comply with all protocol requirements and procedures
- Healthy Control Cohort
- Healthy males with normal neuromuscular examination
- Subjects with a chronologic age of 3 to 7 years inclusive for Cohort C, and 8 to 12 years inclusive for Cohort D
- Subjects who provide assent, as stipulated by IRB requirements, and whose parent/guardian signs an informed consent form
- Subjects who are willing and able to cooperate and comply with all protocol requirements and procedures
Who cannot join
- Subjects with daytime ventilatory dependence (non-invasive or tracheostomy)
- Subjects enrolled in a DMD therapeutic clinical trial concomitantly or within the past 4 weeks
- Subjects with any physical or mental condition which may, in the investigator's opinion, render the subject unable to complete the tasks of the study appropriately
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Massauchusetts General Hospital
Boston, Massachusetts, 02114
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
The University of Texas Southwestern Medical Center-Dallas
Dallas, Texas, 75220
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University
St Louis, Missouri, 63110
About this study
Official title: A Phase I, Multi-Center, Controlled, Prospective Correlative Study of Electrical Impedance Myography in Males With Duchenne Muscular Dystrophy and In Healthy Males
Physicians seek a method to assess neuromuscular disease that is both non-invasive and quantifiable. Many patients do not tolerate standard current day assessment tools (such as needle electromyogram), and Electrical Impedance Myography (EIM) has the potential to serve as a non-invasive, quantifiable, diagnostic tool for neuromuscular disease. If successful, these devices will allow for improved ability to diagnose neuromuscular disease and to assess disease progression or remission, allowing for better care of individual patients as well as for use in clinical trials, where improved outcome measures for neuromuscular diseases is being sought.
Read the detailed description
Healthy subjects and subjects with DMD will be screened during Visit 1. Screening evaluations will establish eligibility and will include a medical history, abbreviated physical examination, vital signs (blood pressure, temperature, pulse, and respirations), and weight. Eligible subjects will provide assent, as stipulated by Institutional Review Board (IRB) requirements, and a parent/guardian will sign an informed consent, after which the subject will be considered enrolled.
Subjects will be enrolled into 4 study cohorts for a total of approximately 90 subjects. Allocation into these cohorts is planned due to considerations regarding the effect of age on outcomes.
Visit 2, Study Assessments, may take place on the day of Visit 1 screening evaluations. If the subject cannot complete study assessments on the same day, the subject may return and complete Visit 2 evaluations within approximately 24 hours of the end of Visit 1.
Visit 2 assessments will include morphological testing of muscle length and girth, skin fat measurement, Clinical Assessments (6-Minute Walk Test [6MWT], North Star Ambulatory Assessment, timed function tests, dynamometry), and 3 phases of Device Performance testing. Phase 1 Device Performance testing will be done by each of 2 trained raters (Rater A and Rater B) from the research site, designated to perform the Device Performance testing on all subjects at their respective site. At least 10 minutes after completion of the first assessments, Phase 2 Device Performance testing will be repeated by Rater A. Upon completion of the Phase 2 Device Performance testing, the subjects will proceed with Clinical Assessments performed by a physical therapist. Following Clinical Assessments, Phase 3 Device Performance testing will be performed by Rater B.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Testing with EIM
- Keywords
- DMD, EIM, Duchenne Muscular Dystrophy, electrical impedance myography
- 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.
Testing with EIMOther
| Study group | Role | What they receive |
|---|---|---|
| DMD subject ages 3-7 inclusive | Other | Young DMD Testing with EIM Receives: Other: Testing with EIM |
| DMD subject ages 8-12 inclusive | Other | Older DMD Testing with EIM Receives: Other: Testing with EIM |
| Healthy Control ages 3-7 inclusive | Other | Young Healthy Testing with EIM Receives: Other: Testing with EIM |
| Healthy Control ages 8-12 inclusive | Other | Older Healthy Testing with EIM Receives: Other: Testing with EIM |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects With an Adverse Event.
Adverse events will be assessed during the time the subject is enrolled in the trial.
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Feb 2012 (estimated)
- Study startFeb 2012
- Primary completionAug 2012
- Study completionAug 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Interventional
- Phase
- Phase N/A
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 92 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dart Therapeutics. LLC
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01523964
- Sponsor's protocol ID
- CMD-001
- Status last verified
- Oct 2013
- First submitted
- 30 Jan 2012
- Last updated
- 9 Dec 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Multi-Center, Controlled, Prospective Correlative Study of Electrical Impedance Myography in Males With Duchenne Muscular Dystrophy and In Healthy Males
- ClinicalTrials.gov ID
- NCT01523964
- Acronym / sponsor ID
- CMD-001
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, EIM, Duchenne Muscular Dystrophy, electrical impedance myography
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 92 participants (actual)
- Lead sponsor
- Dart Therapeutics. LLCUniversity, hospital or other
- Study start
- Feb 2012
- Primary completion
- Aug 2012 (actual)
- Study completion
- Aug 2012 (actual)
- First posted
- 1 Feb 2012 (estimated)
- Results
- Posted 9 Dec 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Physicians seek a method to assess neuromuscular disease that is both non-invasive and quantifiable. Many patients do not tolerate standard current day assessment tools (such as needle electromyogram), and Electrical Impedance Myography (EIM) has the potential to serve as a non-invasive, quantifiable, diagnostic tool for neuromuscular disease. If successful, these devices will allow for improved ability to diagnose neuromuscular disease and to assess disease progression or remission, allowing for better care of individual patients as well as for use in clinical trials, where improved outcome measures for neuromuscular diseases is being sought.
Detailed description
Healthy subjects and subjects with DMD will be screened during Visit 1. Screening evaluations will establish eligibility and will include a medical history, abbreviated physical examination, vital signs (blood pressure, temperature, pulse, and respirations), and weight. Eligible subjects will provide assent, as stipulated by Institutional Review Board (IRB) requirements, and a parent/guardian will sign an informed consent, after which the subject will be considered enrolled.
Subjects will be enrolled into 4 study cohorts for a total of approximately 90 subjects. Allocation into these cohorts is planned due to considerations regarding the effect of age on outcomes.
Visit 2, Study Assessments, may take place on the day of Visit 1 screening evaluations. If the subject cannot complete study assessments on the same day, the subject may return and complete Visit 2 evaluations within approximately 24 hours of the end of Visit 1.
Visit 2 assessments will include morphological testing of muscle length and girth, skin fat measurement, Clinical Assessments (6-Minute Walk Test [6MWT], North Star Ambulatory Assessment, timed function tests, dynamometry), and 3 phases of Device Performance testing. Phase 1 Device Performance testing will be done by each of 2 trained raters (Rater A and Rater B) from the research site, designated to perform the Device Performance testing on all subjects at their respective site. At least 10 minutes after completion of the first assessments, Phase 2 Device Performance testing will be repeated by Rater A. Upon completion of the Phase 2 Device Performance testing, the subjects will proceed with Clinical Assessments performed by a physical therapist. Following Clinical Assessments, Phase 3 Device Performance testing will be performed by Rater B.
Study design
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| DMD subject ages 3-7 inclusive | Other | Other: Testing with EIMYoung DMD Testing with EIM |
| DMD subject ages 8-12 inclusive | Other | Other: Testing with EIMOlder DMD Testing with EIM |
| Healthy Control ages 3-7 inclusive | Other | Other: Testing with EIMYoung Healthy Testing with EIM |
| Healthy Control ages 8-12 inclusive | Other | Other: Testing with EIMOlder Healthy Testing with EIM |
Who may qualify
- Ages
- 3 to 12 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (14 listed)
- DMD Subject Cohorts
- Male subjects
- Subjects with a chronologic age of 3 to 7 years inclusive for Cohort A, and 8 to 12 years inclusive for Cohort B
- Subjects with DMD diagnosed with mutational testing and/or absence of dystrophin on muscle biopsy
- Subjects with proximal pelvic girdle weakness (Gower's maneuver, difficulty with arising from floor and going up steps)
Key exclusion criteria (3 listed)
- Subjects with daytime ventilatory dependence (non-invasive or tracheostomy)
- Subjects enrolled in a DMD therapeutic clinical trial concomitantly or within the past 4 weeks
- Subjects with any physical or mental condition which may, in the investigator's opinion, render the subject unable to complete the tasks of the study appropriately
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Testing with EIM | Other | DMD subject ages 3-7 inclusive, DMD subject ages 8-12 inclusive, Healthy Control ages 3-7 inclusive, Healthy Control ages 8-12 inclusive |
Primary outcome
Number of Subjects With an Adverse Event.
Time frame: 1 day
Adverse events will be assessed during the time the subject is enrolled in the trial.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Dec 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Testing with EIM in people with Duchenne Muscular Dystrophy, run by Dart Therapeutics. LLC at 6 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 14 inclusion and 3 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 9 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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?
Yes. Results are posted in the registry (first posted 9 Dec 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Subjects With an Adverse Event. (time frame: 1 day). 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 9 Dec 2013.