175 Participants Needed

EDG-5506 for Becker Muscular Dystrophy

Recruiting at 54 trial locations
ET
Overseen ByEdgewise Therapeutics
Age: < 65
Sex: Male
Trial Phase: Phase 2
Sponsor: Edgewise Therapeutics, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is testing a new drug called sevasemten to see if it can help people with Becker muscular dystrophy, a condition that weakens muscles. The study aims to find out if the drug is safe and effective in improving muscle function and reducing symptoms.

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

The trial protocol does not specify if you must stop taking your current medications. However, you cannot have taken oral corticosteroids for Becker muscular dystrophy in the past 6 months or any investigational drug within 30 days or 5 half-lives before the screening.

Will I have to stop taking my current medications?

The trial requires that you have not taken oral corticosteroids for Becker muscular dystrophy in the past 6 months. If you are on other medications, the protocol does not specify whether you need to stop them, so it's best to discuss this with the study team.

What safety data is available for EDG-5506 in Becker Muscular Dystrophy?

The provided research does not contain specific safety data for EDG-5506 in Becker Muscular Dystrophy. The studies mentioned focus on other treatments and conditions, such as Drisapersen, DT-DEC01, Edasalonexent, and Vamorolone, primarily in Duchenne Muscular Dystrophy or animal models. Therefore, no relevant safety data for EDG-5506 is available in the given research.12345

Is the drug EDG-5506, Placebo a promising treatment for Becker Muscular Dystrophy?

The information provided does not mention EDG-5506 or Placebo as promising treatments for Becker Muscular Dystrophy. The articles focus on other aspects of the disease and different treatments, like vamorolone, which shows potential in increasing dystrophin protein and improving muscle function in a mouse model. Therefore, there is no evidence here to suggest that EDG-5506, Placebo is promising for Becker Muscular Dystrophy.46789

How is the drug EDG-5506 different from other treatments for Becker muscular dystrophy?

EDG-5506 is unique because it is specifically being tested for Becker muscular dystrophy, a condition with no approved therapies, and it may offer a novel approach compared to existing treatments like vamorolone, which focuses on increasing dystrophin protein and reducing inflammation.46789

What data supports the idea that EDG-5506 for Becker Muscular Dystrophy is an effective treatment?

The available research does not provide specific data on the effectiveness of EDG-5506 for Becker Muscular Dystrophy. However, it mentions that there are no drugs and few clinical trials for this condition, indicating a lack of direct evidence for EDG-5506. In contrast, another treatment, prednisone, showed dramatic and sustained improvement in strength for some patients with Becker Muscular Dystrophy, suggesting it might be a more effective option based on current data.78101112

Who Is on the Research Team?

JD

Joanne Donovan, MD, PhD

Principal Investigator

Edgewise Therapeutics, Inc.

RD

Roxana D. Dreghici

Principal Investigator

Edgewise Therapeutics, Inc.

Are You a Good Fit for This Trial?

This trial is for adolescents (12-17) and adults (18-50) with Becker muscular dystrophy confirmed by genetic testing. Participants must be able to complete a 100-meter walk within certain times, perform specific physical assessments, and have been ambulatory beyond certain ages with or without steroids.

Inclusion Criteria

I can complete a mobility test with a score between 10 and 32.
I can walk 100 meters in less than 200 seconds, with or without help from a device.
I can do a specific physical test and score between 5 and 32.
See 7 more

Exclusion Criteria

Medical history or clinically significant physical examination/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. This includes contraindications to magnetic resonance imaging such as non-compatible implanted medical devices or severe claustrophobia
Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study
My lung function is severely reduced or I need help breathing during the day.
See 2 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Treatment

Participants receive sevasemten or placebo to evaluate safety, pharmacokinetics, biomarkers, and functional measures

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Extension

Participants in the GRAND CANYON cohort receive extended treatment to evaluate long-term safety and efficacy

18 months

What Are the Treatments Tested in This Trial?

Interventions

  • EDG-5506
  • Placebo
Trial Overview The GRAND CANYON study tests the safety and effectiveness of EDG-5506 at different doses (5 mg, 10 mg, 12.5 mg) compared to a placebo in treating Becker muscular dystrophy. It's randomized and double-blind, meaning neither participants nor researchers know who gets the drug or placebo.
How Is the Trial Designed?
5Treatment groups
Experimental Treatment
Group I: Adult Cohort 6Experimental Treatment2 Interventions
Drug: Sevasemten Drug: Placebo
Group II: Adult Cohort 2Experimental Treatment2 Interventions
Drug: Sevasemten Drug: Placebo
Group III: Adult Cohort 1Experimental Treatment2 Interventions
Drug: Sevasemten Drug: Placebo
Group IV: Adolescent Cohort 5Experimental Treatment2 Interventions
Drug: Sevasemten Drug: Placebo
Group V: Adolescent Cohort 4Experimental Treatment2 Interventions
Drug: Sevasemten Drug: Placebo

Find a Clinic Near You

Who Is Running the Clinical Trial?

Edgewise Therapeutics, Inc.

Lead Sponsor

Trials
13
Recruited
1,000+

Sysnav

Industry Sponsor

Trials
9
Recruited
760+

Medpace, Inc.

Industry Sponsor

Trials
98
Recruited
30,400+

Dr. August J. Troendle

Medpace, Inc.

Chief Executive Officer since 1992

MD from the University of Maryland, School of Medicine; MBA from Boston University

Dr. Reinilde Heyrman

Medpace, Inc.

Chief Medical Officer since 2017

MD

ImagingNMD

Collaborator

Trials
1
Recruited
180+

SYSNAV

Industry Sponsor

Trials
13
Recruited
1,200+

Published Research Related to This Trial

In a study of 70 patients with Becker muscular dystrophy (BMD), 77.1% exhibited neurodevelopmental, behavioral, or emotional symptoms, with the most common being inattention/hyperactivity (35.7%) and emotional dysregulation (38.6%).
Despite the presence of various symptoms, the type of dystrophin gene mutation did not significantly affect the number or type of symptoms, indicating that these issues are common across different mutation sites.
Neurodevelopmental, behavioral, and emotional symptoms in Becker muscular dystrophy.Lambert, JT., Darmahkasih, AJ., Horn, PS., et al.[2020]
Recent research has focused on Becker muscular dystrophy (BMD) as a target for clinical trials, especially as treatment strategies for Duchenne muscular dystrophy (DMD) aim to convert DMD patients to a BMD phenotype.
While there have been advancements in understanding BMD through natural history studies and improved diagnostics, the lack of long-term data and the variability in disease severity pose challenges for designing effective clinical trials.
An update on Becker muscular dystrophy.Straub, V., Guglieri, M.[2023]
The bmx mouse model, created using CRISPR/Cas9 technology, successfully mimics Becker muscular dystrophy (BMD) by expressing a common mutation in the dystrophin gene, allowing for the study of muscle and heart dysfunction associated with the disease.
This model shows significant muscle weakness and heart dysfunction compared to wild-type mice, with increased muscle damage and inflammation, making it a valuable tool for understanding BMD and testing potential therapies.
The X-linked Becker muscular dystrophy (bmx) mouse models Becker muscular dystrophy via deletion of murine dystrophin exons 45-47.Heier, CR., McCormack, NM., Tully, CB., et al.[2023]

Citations

Neurodevelopmental, behavioral, and emotional symptoms in Becker muscular dystrophy. [2020]
An update on Becker muscular dystrophy. [2023]
The X-linked Becker muscular dystrophy (bmx) mouse models Becker muscular dystrophy via deletion of murine dystrophin exons 45-47. [2023]
Prednisone therapy in Becker's muscular dystrophy. [2017]
Dystrophin levels and clinical severity in Becker muscular dystrophy patients. [2017]
Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy. [2022]
Assessment of Motor Unit Potentials Duration as the Biomarker of DT-DEC01 Cell Therapy Efficacy in Duchenne Muscular Dystrophy Patients up to 12 Months After Systemic-Intraosseous Administration. [2023]
Disease-modifying effects of edasalonexent, an NF-κB inhibitor, in young boys with Duchenne muscular dystrophy: Results of the MoveDMD phase 2 and open label extension trial. [2021]
Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice. [2023]
A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial. [2021]
11.United Statespubmed.ncbi.nlm.nih.gov
Outcome reliability in non-ambulatory boys/men with Duchenne muscular dystrophy. [2021]
Generation of a human induced pluripotent stem cell line (INNDSUi003-A) derived from patient with Becker muscular dystrophy (BMD). [2022]
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