Recruiting

Muscle Imaging

Sponsor:

University of Florida

Code:

NCT01484678

Conditions

Duchenne Muscular Dystrophy

Becker Muscular Dystrophy

Eligibility Criteria

Sex: Male

Age: 5 - 62

Healthy Volunteers: Accepted

Study Details

Brief summary:

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.

Conditions

Duchenne Muscular Dystrophy

Becker Muscular Dystrophy

Study ID

NCT01484678

Start date

Sep 1, 2020

Status verified date

Oct, 2025

Completion date

Aug 31, 2026

Anticipated

Primary completion date

Aug 31, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 5 - 62

Healthy Volunteers: Accepted

Inclusion Criteria for boys with DMD:

1\. Ambulatory and non-ambulatory males (ages 5-30 at baseline testing) previously diagnosed with DMD based on:

  • clinical features with onset of symptoms before age five
  • elevated serum creatine kinase level or
  • absence of dystrophin expression, as determined by immunostain or western blot (<2%) and/or DNA confirmation of a dystrophin mutation \*Subjects will not be excluded based on corticosteroid treatment or other clinical trials

Inclusion Criteria for adults with Becker MD:

1. Ambulatory males (ages 18-62) without disease or injury to the lower extremities
2. Specific recruitment of a subset of individuals with deletion mutations in the dystrophin gene involving either exon 51 or exon 45.

Inclusion Criteria for age matched controls for Becker MD subjects:

1\. Ambulatory males (ages 18-62) without disease or injury to the lower and/or upper extremities will be eligible to participate in this study

Exclusion Criteria:

1. Males with a contraindication to an MR examination
2. Males with unstable medical problems
3. Males who are not able to cooperate during testing
4. Males with a secondary condition that may impact muscle metabolism, muscle function or functional ability (i.e. cerebral palsy, endocrine disorders, mitochondrial disease)
5. Daytime ventilation
6. Implantable Cardioverter Defibrillator- (ICD) or pace maker
7. Healthy boys/men who participate in competitive sports specific training in excess of 8 hours per week

Study Design

Enrollment

550 participants

Anticipated

Interventions and Outcome Measures

Arms

Age Matched Controls

Age matched non-affected (non-DMD) boys \* This arm is full

Age matched non-affected men, matched for men with Becker MD \*Recruiting

Boys/Men with DMD

This group will include ambulatory and non-ambulatory boys/men with Duchenne Muscular Dystrophy ranging from 5-30 years old. \*Recruiting

Adults with Becker MD

This group will include ambulatory and non-ambulatory men with Becker Muscular Dystrophy ranging from 18-62 years old. \* Recruiting

Primary outcome measure

  • Change from baseline in intramuscular lipid up to 3-10 years [ Time Frame: Change in baseline up to 3-10 years ]
  • Change from baseline in muscle T2 up to 3 months in DMD [ Time Frame: Change in baseline up to 3 months ]
  • Correlation between MR measures of intramuscular lipid, functional endpoints and histological markers. [ Time Frame: Through study completion, an average of 1 year ]

Central Contacts and Locations

Central contacts

Krista Vandenborne, PhD

352-273-6100kvandenb@phhp.ufl.edu

Kelly Rock, PhD

352-294-5798k.rock@phhp.ufl.edu

Locations

University of Florida

Recruiting

Gainesville, Florida, United States, 32610

Contacts

Krista Vandenborne, PhD

352-273-6100kvandenb@phhp.ufl.edu

Kelly Rock, PhD

352-294-5798k.rock@phhp.ufl.edu

Principal Investigator:

Krista Vandenborne, PhD

Oregon Health and Science University

Recruiting

Portland, Oregon, United States, 97239

Contacts

William Rooney, PhD

503-418-1532rooneyw@ohsu.edu

Olivia Huerta

971-347-6084huertao@ohsu.edu

Principal Investigator:

Bill Rooney, PhD

More Information

Sponsor

University of Florida

Last update posted

Oct 15, 2025

Last verified

Oct, 2025

Keywords

  • Duchenne Muscular Dystrophy
  • Becker Muscular Dystrophy
  • Magnetic Resonance Spectroscopy
  • Magnetic Resonance Imaging
  • Muscle

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of Florida on 2025-10-15.