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Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy (QED)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01491555
Recruitment Status : Completed
First Posted : December 14, 2011
Last Update Posted : March 24, 2016
Sponsor:
Collaborator:
Beth Israel Deaconess Medical Center
Information provided by (Responsible Party):
Basil Darras, Boston Children’s Hospital

Tracking Information
First Submitted Date December 5, 2011
First Posted Date December 14, 2011
Last Update Posted Date March 24, 2016
Study Start Date April 2012
Actual Primary Completion Date September 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: March 23, 2016)
The rate of decline of DMD patients versus normal subjects as assessed by EIM and quantitative ultrasound [ Time Frame: up to 45 months ]
With the successful completion of this aim, the investigators will establish that alterations in both EIM and QUS provide meaningful surrogate measures of disease progression in DMD.
Original Primary Outcome Measures Not Provided
Change History
Current Secondary Outcome Measures
 (submitted: March 23, 2016)
The rate of decline of DMD patients versus normal subjects as assessed by handheld dynamometry, 6-minute walk, and other functional tests. [ Time Frame: up to 45 months ]
With the successful completion of this aim, the investigators will establish that alterations in functional assessments may provide additional meaningful surrogate measures of disease progression in DMD.
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy
Official Title Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy
Brief Summary

Researchers at Children's Hospital Boston Neurology Department invite children to participate in a new research study. Researchers are looking for boys ages 2 - 30 with Duchenne Muscular Dystrophy (DMD) and healthy boys ages 2 - 30 (without any nerve or muscle concerns) to serve as controls. The study is evaluating a new technique that will test nerve and muscle function. The testing is all pain free.

Children participating in the study will come in for 10 visits over two years. Visits will take place every month at first, then less often for the remaining visits. The tests for the study itself take approximately 2hours. If participants are interested or would like to learn more about the study, please call Lavanya Madabusi at 617-919-3554 or Lavanya.Madabusi@childrens.harvard.edu. All inquiries will be kept strictly confidential.

Detailed Description Characterized by progressive disability leading to death, Duchenne muscular dystrophy (DMD) remains one of the most common and devastating neuromuscular disorders of childhood. Although a variety of promising new treatment strategies are in development, outcome measures for clinical trials remain limited for the most part to a set of functional measures, such as the six-minute walk test. While clearly useful, such measures are impacted by unrelated factors, such as mood and effort, and have limited repeatability. To address this and other limitations, magnetic resonance imaging (MRI) is now being investigated as a surrogate measure. However, more easily applied, cost-effective, office-based surrogate measures that provide high repeatability and sensitivity while still correlating strongly to disease status would find wider use in Phase II and possibly in Phase III clinical trials in DMD. Quantitative ultrasound (QUS) and electrical impedance myography (EIM) are two techniques that could serve in this role. In QUS, muscle pathology (fibrosis and fatty infiltration) in DMD results in an increase in energy reflected back (backscatter) to the ultrasound. The amount of backscatter can be measured directly by analyzing the raw frequency-based acoustic data or indirectly by controlled processing of the gray-scale image. EIM, in contrast, relies upon the application of localized electrical current and measurement of the resulting surface voltages, but is similarly impacted by the fibrotic changes that develop as muscle disease progresses. Here, the investigators propose to evaluate and compare both methodologies simultaneously in a group of DMD patients and normal subjects in order to assess their ability to identify clinically meaningful alterations in muscle health over short intervals of time. As a final exploratory analysis, the investigators will also study the possibility of combining the two modalities. The results of this work will have broad application as they could be applied to a variety of neuromuscular conditions, including other muscular dystrophies. Thus, the hypothesis of this proposal is that both QUS and EIM can serve as convenient, non-invasive, clinically meaningful surrogate markers of disease progression in DMD that surpass the functional measures currently in use.
Study Type Observational
Study Design Observational Model: Case Control
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population This study will involve boys with DMD and healthy male controls.
Condition Duchenne Muscular Dystrophy
Intervention Not Provided
Study Groups/Cohorts
  • DMD patients
    35 boys ages 2 through 30 with DMD
  • Control Group
    35 healthy boys ages 2 through 30
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: March 23, 2016)
73
Original Estimated Enrollment
 (submitted: December 13, 2011)
70
Actual Study Completion Date September 2015
Actual Primary Completion Date September 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion criteria (DMD):

  1. Genetically or histologically established diagnosis of DMD
  2. Male, age 2 - 30

Inclusion criteria (Control):

1. Male, age 2 - 30

Exclusion criteria (DMD):

  1. Presence of implanted pacemaker or other electrical device
  2. Presence of a superimposed neuromuscular or other medical condition that substantially impacts the individual's health

Exclusion criteria (control):

  1. Presence or past history of a neuromuscular disorder or other disease that substantially impacts health
  2. Presence of implanted pacemaker or other electrical device.
Sex/Gender
Sexes Eligible for Study: Male
Ages 2 Years to 30 Years   (Child, Adult)
Accepts Healthy Volunteers Yes
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT01491555
Other Study ID Numbers IRB-P00001218
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement
Plan to Share IPD: Undecided
Responsible Party Basil Darras, Boston Children’s Hospital
Study Sponsor Boston Children’s Hospital
Collaborators Beth Israel Deaconess Medical Center
Investigators
Principal Investigator: Basil Darras, MD Boston Children’s Hospital
Principal Investigator: Seward Rutkove, MD Beth Israel Deaconess Medical Center
PRS Account Boston Children’s Hospital
Verification Date March 2016