ImagingDMD - Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

Frequently Asked Questions (FAQs) about this study:

 

What stage is this research?

  •  This is an active trial in Cycle 2 and enrolling. 

 

What is the goal or purpose of this study?

  • This study focuses on developing Magnetic Resonance Imaging (MRI) as a tool to monitor disease progression in Duchenne and to serve as an outcome measure for clinical trials. The aim of the study is to determine whether noninvasive MRI outcome measures can replace muscle biopsies in evaluating the effectiveness of new treatments in future clinical trials.
  • In Cycle I of this study we demonstrated that MRI studies of the legs in ambulatory boys are very sensitive to disease progression, can be reliably implemented across sites, are predictive of loss of ambulation, and correlate with function. This is extremely important as it provides strong evidence that MRI can be valuable as a biomarker in clinical trials.
  • In Cycle II of this study we will perform MRI studies of the arms and study the effect of loss of ambulation on the MRI biomarkers. The reason for studying the arms and MRI biomarkers in boys that lose the ability to walk is to set the stage for the inclusion of non-ambulatory boys in future clinical trials.

 

Who is funding this study?

  • This study is funded by the NIH – NIAMS/NINDS.

 

Who is eligible to participate in this study?

  • Cycle I: Enrollment is complete.
  • Cycle II: Participants in this study will be males with Duchenne, ages 5-18 years, ambulatory and non-ambulatory upon entering the study.

 

What are participants doing in this study?

  • Phase II: MRI and Magnetic Resonance Spectroscopy (MRS) measurements are performed on the participants' arm as well as leg muscles, muscle strength, and functional tests such as performance of the upper limb, timed tests, and walking (if ambulatory) are also performed.  Additionally, blood, urine, and saliva samples will be taken from the participants and stored in established tissue banks.

 

Where does this study take place?

  • This study is taking place in 3 different cities in the US: Gainesville, FL at the University of Florida; Philadelphia, PA at Children’s Hospital of Philadelphia (CHOP); and Portland, OR at the Oregon Health and Science University (OHSU) and Shiners Hospital for Children-Portland.

 When will the study be completed?

  • The estimated study completion date is May, 2020

 

Are there any preliminary results available?

 

Where can I learn more about this study?

  • You can learn more about this study at www.ImagingDMD.org and www.ClinicalTrials.gov (NCT01484678) or contact Jennifer Claudia, This email address is being protected from spambots. You need JavaScript enabled to view it., 352-273-6453.
  • Please check www.DuchenneConnect.org for updates to this FAQ sheet.

 

(Updated 4/27/2017)