Edgewise Therapeutics 
(EDG-5506-210)

Study name: LYNX
A study to test safety, pharmacokinetics and biomarkers of muscle damage in boys with DMD ages 4-9.

An additional cohort, Cohort 2NS, to include participants (aged 4 to 7 years inclusive) not currently treated with corticosteroids, will enroll approximately 9 participants after Cohort 2 safety review and in parallel with the additional cohorts.

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  Current Status:
Active; Enrollment Closed

Edgewise Therapeutics
(EDG-5506-201)

Study name: CANYON
A study for men with Becker muscular dystrophy ages 18-55.

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 Current Status:
Active; Enrollment Closed

Edgewise Therapeutics
(EDG-5506-215)

Study name: FOX
A FOX study is a Phase 2, multicenter trial evaluating safety, pharmacokinetics, and biomarkers in children and adolescents (aged 6-17) with Duchenne muscular dystrophy previously treated with gene therapy. It includes a randomized, double-blind, placebo-controlled Part A, followed by an open-label Part B.

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 Current Status:
Active; Enrollment Closed

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.

Catalyst Pharmaceuticals, Inc. (DMD-001)

Registry Study to observe long-term safety of Vamorolone (AGAMREE®) in patients with Duchenne Muscular Dystrophy.

This is a multi-center, observational, prospective, longitudinal registry designed to collect data in male patients aged 2 years and older with DMD treated with AGAMREE®. 

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 Current Status:
Active; Recruiting

Hoffmann-La Roche Ltd (BN45398)

A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) (SHIELD DMD)

The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory participants with DMD age ≥ 8 to < 18 years old receiving corticosteroid therapy.

 Current Status:
Active; Enrolling

ITF Therapeutics, LLC

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy (PROVIDUS)

This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinostat or having started therapy within 6 months as part of routine clinical care in the US. The study has a planned maximum duration of 5 years for the first enrolled patients, including a 24-month enrollment period and a minimum of 2 years of follow-up.  

 Current Status:
In start up; Enrolling Soon

Investigator Initiation Studies: 

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.

Functional magnetic resonance imaging and neuropsychological analysis in dystrophinopathies (H00019631)

Description:

We are looking for 65 people who who are older than 10 years old and less than 21 years old who will take part here at UMass Chan Medical School. Of the 65, 30 will have DMD, 15 will have BMD, and 20 will have neither.

What we are hoping for:

The purpose of this research is to learn how different types of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy behave in the brain  

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.

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.