Clinical Trials
The Duchenne Program at UMass Chan is committed to providing the best possible care today while helping to develop the treatments of tomorrow.
Our ultimate goal is a world without Duchenne muscular dystrophy. It takes teamwork, dedication, and careful science to get there, and we are committed to being a part of the solution. To help advance new potential treatments, we’re participating in a variety of clinical trials.
Check this page regularly, as we are building our clinical trials program and will be adding more trials in the near future. If you are interested in any of the clinical trials below, please email dmdresearchprogram@umassmed.edu.
Active clinical trials at our site
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Edgewise Therapeutics
(EDG-5506-201)
Study name: LYNX
A study to test safety, pharmacokinetics and biomarkers of muscle damage in boys with DMD ages 4-9.
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To learn more details about this trial
Current Status:
Active; Recruiting
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Edgewise Therapeutics
(EDG-5506-201)
Study name: CANYON
A study for men with Becker muscular dystrophy ages 18-55.
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To learn more details about this trial
Current Status:
Active; Recruiting
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Dyne Therapeutics
(Dyne-251-DMD-201)
Study name: Deliver
A study to evaluate safety, tolerability, and dystrophin levels in males with DMD ages 4 to 16 who have mutations amenable to exon 51 skipping therapy. The study will also measure muscle function, exon skipping, and pharmacokinetics.
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To learn more details about this trial
Current Status:
Active; Recruiting |
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Sarepta Therapeutics Vesleteplirsen (SRP-5051)
Study name: Momentum
A study to evaluate the safety and tolerability of a second-generation exon skipping treatment
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To learn more details about this trial
Current Status:
In process; Not recruiting
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Investigator Initiation Studies:
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Registry - Description
This study is intended to collect data from medical records on patients who have Duchenne Muscular Dystrophy
Overview:
This is a data collection study that will allow researchers to gather information to better understand Duchenne Muscular Dystrophy . Examples of data collected form medical records include descriptions of muscle function, lab results, dexascans, and outcomes in response to treatment.
What we're hoping for:
We are hoping that the information gathered by this study may be used to help improve identification and treatment of children with dystrophin disorders.
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Biorepository
Description:
Seeking boys and men with Duchenne muscular dystrophy (DMD) or Becker Muscular Dystrophy (BMD) to provide samples including blood, plasma, serum, urine, as well as the remainders of tissue samples collected as part of their usual clinical care for a biorepository.
Overview:
We are collecting biological samples including an annual blood plasma and serum sample and a urine sample from volunteers with Duchenne muscular dystrophy (DMD) and Becker Muscular Dystrophy (BMD) to support future scientific investigation into these rare disorders.
What we're hoping for:
We hope to collect biological samples from boys and men with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) to support future scientific investigation.
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f-MRI
Seeking male participants aged 10-21 years of age who have been diagnosed with Duchenne Muscular Dystrophy (DMD) for participation in an imaging study.
Overview:
The purpose of this research is to learn how different mutations of Duchenne Muscular Dystrophy mutations present in a neuropsycholgical view.
What we're hoping for:
The purpose of this research is to learn how different mutations of Duchenne Muscular Dystrophy mutations are present in a neuropsychological view.
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