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

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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

December 5, 2011
December 14, 2011
March 24, 2016
April 2012
September 2015   (Final data collection date for primary outcome measure)
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.
Not Provided
Complete list of historical versions of study NCT01491555 on ClinicalTrials.gov Archive Site
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.
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Not Provided
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Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy
Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy

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.

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.
Observational
Observational Model: Case Control
Time Perspective: Prospective
Not Provided
Not Provided
Non-Probability Sample
This study will involve boys with DMD and healthy male controls.
Duchenne Muscular Dystrophy
Not Provided
  • DMD patients
    35 boys ages 2 through 30 with DMD
  • Control Group
    35 healthy boys ages 2 through 30
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
73
70
September 2015
September 2015   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: Male
2 Years to 30 Years   (Child, Adult)
Yes
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT01491555
IRB-P00001218
No
Not Provided
Plan to Share IPD: Undecided
Basil Darras, Boston Children’s Hospital
Boston Children’s Hospital
Beth Israel Deaconess Medical Center
Principal Investigator: Basil Darras, MD Boston Children’s Hospital
Principal Investigator: Seward Rutkove, MD Beth Israel Deaconess Medical Center
Boston Children’s Hospital
March 2016