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NCT01484678

Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

Recruiting now Last updated 15 October 2025
What this trial tests

trial in Duchenne Muscular Dystrophy in 550 participants. Currently enrolling.

Timeline
1 September 2020
Primary endpoint
31 August 2026
31 August 2026

Quick facts

Lead sponsorUniversity of Florida
StatusRecruiting now
Study typeOBSERVATIONAL
Enrollment550
Start date1 September 2020
Primary completion31 August 2026
Estimated completion31 August 2026
Sites3 locations across United States

Conditions studied

Sponsor

University of Florida

Who can join

Adults 5 to 62, male only, with Duchenne Muscular Dystrophy or Becker Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The purpose of this research study is to determine the potential of magnetic resonance imaging, spectroscopy, and whole body imaging to monitor disease progression and to serve as an objective outcome measure for clinical trials in Muscular Dystrophy (MD). The investigators will compare the muscles of ambulatory or non-ambulatory boys/men with DMD with muscles of healthy individuals of the same age and monitor disease progression in those with DMD over a 5-10 year period. The amount of muscle damage and fat that the investigators measure will also be related to performance in daily activities, such as walking and the loss of muscle strength. In a small group of subjects the investigators will also assess the effect of corticosteroid drugs on the muscle measurements. Additionally, the investigators will map the progression of Becker MD following adults with this rare disease. The primary objective is to conduct a multi-centered study to validate the potential of non-invasive magnetic resonance imaging and magnetic resonance spectroscopy to monitor disease progression and to serve as a noninvasive surrogate outcome measure for clinical trials in DMD and BMD. The secondary objective is to characterize the progressive involvement of the lower extremity, upper extremity, trunk/respiratory muscles in boys/men with DMD and BMD guiding clinical trials.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort.
    Willcocks RJ, Rooney WD, Triplett WT, Forbes SC, et al · · 2016 · cited 140× · PMID 26891991 · DOI 10.1002/ana.24599
  2. Examination of effects of corticosteroids on skeletal muscles of boys with DMD using MRI and MRS.
    Arpan I, Willcocks RJ, Forbes SC, Finkel RS, et al · · 2014 · cited 122× · PMID 25098537 · DOI 10.1212/wnl.0000000000000775
  3. MR biomarkers predict clinical function in Duchenne muscular dystrophy.
    Barnard AM, Willcocks RJ, Triplett WT, Forbes SC, et al · · 2020 · cited 74× · PMID 32024675 · DOI 10.1212/wnl.0000000000009012
  4. Modeling disease trajectory in Duchenne muscular dystrophy.
    Rooney WD, Berlow YA, Triplett WT, Forbes SC, et al · · 2020 · cited 64× · PMID 32184340 · DOI 10.1212/wnl.0000000000009244
  5. Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history.
    Arora H, Willcocks RJ, Lott DJ, Harrington AT, et al · · 2018 · cited 58× · PMID 29742798 · DOI 10.1002/mus.26161
  6. Skeletal muscle magnetic resonance biomarkers correlate with function and sentinel events in Duchenne muscular dystrophy.
    Barnard AM, Willcocks RJ, Finanger EL, Daniels MJ, et al · · 2018 · cited 54× · PMID 29554116 · DOI 10.1371/journal.pone.0194283
  7. Suitability of external controls for drug evaluation in Duchenne muscular dystrophy.
    Goemans N, Signorovitch J, Sajeev G, Yao Z, et al · · 2020 · cited 29× · PMID 32611643 · DOI 10.1212/wnl.0000000000010170
  8. Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy.
    Willcocks RJ, Forbes SC, Walter GA, Sweeney L, et al · · 2021 · cited 16× · PMID 33394000 · DOI 10.1001/jamanetworkopen.2020.31851

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Other recruiting trials for Duchenne Muscular Dystrophy

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