Observational Study for Becker Muscular Dystrophy

(GRASP-01-002 Trial)

Not currently recruiting at 14 trial locations

Overseen By Jennifer Raymond

Age: Any Age

Sex: Male

Trial Phase: Academic

Sponsor: Virginia Commonwealth University

Must not be taking: Chronic corticosteroids

Disqualifiers: Non-ambulatory, Ventilatory support, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to better understand Becker muscular dystrophy (BMD), a condition that causes muscle weakness. Researchers will conduct an observational study, monitoring participants over time without providing treatment. The study seeks participants aged 6 and older who have BMD and can walk independently. The goal is to gather data that could aid in developing future treatments. Individuals diagnosed with BMD who can walk 10 meters without assistance might be suitable for this trial. As an unphased study, this trial offers a unique opportunity to contribute to research that could lead to new treatment options for BMD.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop taking your current medications. However, it mentions that the use of chronic corticosteroids is limited to a certain percentage of participants, so you might need to discuss this with the trial team.

Why are researchers excited about this trial?

Researchers are excited about this trial because it focuses on defining important endpoints in Becker Muscular Dystrophy (BMD), which could revolutionize how we measure the effectiveness of future treatments. Unlike standard treatments for BMD, which might include corticosteroids or physical therapy aimed at managing symptoms, this observational study aims to better understand the progression of the disease itself. By identifying precise endpoints, the trial could pave the way for more targeted and effective therapies, ultimately improving the quality of life for those with BMD.

Who Is on the Research Team?

Nicholas E. Johnson, MD

Principal Investigator

Virginia Commonwealth University

Are You a Good Fit for This Trial?

Inclusion Criteria

For ages 8-16

Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD)

Genetic confirmation of an in-frame dystrophin mutation

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observational Study

Participants undergo various assessments to define endpoints in Becker Muscular Dystrophy, including pulmonary function, muscle quality, and motor performance.

24 months

Regular visits throughout the study period

Follow-up

Participants are monitored for safety and effectiveness after the observational study period

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Observational Study

Who Is Running the Clinical Trial?

Virginia Commonwealth University

Lead Sponsor

Trials

732

Recruited

22,900,000+

Edgewise Therapeutics, Inc.

Industry Sponsor

Trials

13

Recruited

1,000+