Using MRI and biomarkers to monitor muscular dystrophy progression

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This research aims to evaluate the effectiveness of magnetic resonance imaging (MRI) and spectroscopy (MRS) in tracking the progression of Duchenne and Becker muscular dystrophies. The study will compare muscle conditions in boys and men with Duchenne muscular dystrophy (DMD) to healthy individuals over a period of 5-10 years, assessing muscle damage and fat content in relation to daily activity performance. Additionally, it will explore the effects of corticosteroid treatment on muscle measurements and map the progression of Becker muscular dystrophy in adults. The goal is to establish non-invasive biomarkers that can serve as reliable outcome measures for future clinical trials.

Why it matters

Who can join: the full rules

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

Where it's running

• University of Florida
  Gainesville, Florida, United States Recruiting
• Oregon Health and Science University
  Portland, Oregon, United States Recruiting
• Children's Hospital of Philadelphia
  Philadelphia, Pennsylvania, United States Active not recruiting

Study contacts

• Principal investigator: William Rooney, PhD — Oregan Health and Science University
• Study coordinator: Krista Vandenborne, PhD
• Email: kvandenb@phhp.ufl.edu
• Phone: 352-273-6100

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Developing new biomarkers for muscular dystrophies

  This study is trying to find easier and less painful ways to measure how serious muscular dystrophies are by looking at urine and blood samples and using simple tests, to help improve diagnosis and treatment for people with these conditions.

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• An online registry for Duchenne and Becker muscular dystrophy patients

  This study is creating an online registry to help people with Duchenne and Becker muscular dystrophy connect with clinical trials and find helpful information about their condition.

  Recruiting The Duchenne Registry · Washington D.C., District of Columbia · NCT02069756

• VM100: a nutritional supplement to see if it improves quality of life for boys with Duchenne

  This Phase 2 study tests VM100, a nutritional drink, in boys aged 8 and up with Duchenne muscular dystrophy to see if it can improve quality of life and reduce fatigue.

  Recruiting Phase 2 University of Florida · Gainesville, Florida · NCT07766980

• KER-065 for Duchenne Muscular Dystrophy: safety and potential benefit in boys and men

  This study tests whether KER-065 is safe and can help with symptoms in males with Duchenne muscular dystrophy, including both ambulatory and nonambulatory participants who are on standard care.

  Recruiting at 2 sites Phase 2 Keros Therapeutics, Inc. · Tbilisi and 3 other locations · NCT07704099

• Modeling heart failure in Duchenne and Becker muscular dystrophy to find surrogate heart measures

  This study will look at heart disease in people with Duchenne/Becker muscular dystrophy and carriers to see if MRI-based heart measures can predict death or major heart problems.

  Recruiting at 9 sites Vanderbilt University Medical Center · Sacramento, California and 8 other locations · NCT07674758

• GNR-097 gene therapy for Duchenne muscular dystrophy in boys

  This study tests a gene therapy (GNR-097) to see if it is safe, tolerable, and potentially beneficial for ambulatory boys with Duchenne muscular dystrophy.

  Recruiting at 6 sites Phase 1/2 Ao Generium · Minsk and 5 other locations · NCT07673809

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.