EDG-5506 for Becker Muscular Dystrophy

Not currently recruiting at 61 trial locations

Overseen By Edgewise Therapeutics

Age: < 65

Sex: Male

Trial Phase: Phase 2

Sponsor: Edgewise Therapeutics, Inc.

Must not be taking: Oral corticosteroids

Disqualifiers: Cardiac issues, Respiratory issues, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of a new drug, EDG-5506 (also known as sevasemten), for treating Becker muscular dystrophy, a genetic disorder that causes muscle weakness. Participants will receive either the drug or a placebo (a substance with no active drug) to compare results. Ideal candidates are adults with a documented genetic mutation linked to Becker muscular dystrophy who can walk a specific distance, even with mobility aids. As a Phase 2 trial, this research measures how well the treatment works in an initial, smaller group, allowing participants to contribute to important early-stage findings.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that sevasemten (also known as EDG-5506) has been tested for safety in people with Becker muscular dystrophy. This treatment reduces the use of certain muscle fibers, helping to protect muscles from damage.

Previous tests examined how well people tolerate sevasemten, including any side effects or bodily changes after taking the drug. Although specific side effects aren't listed, the treatment's progression to a Phase 2 trial suggests some initial safety information is available. This phase typically involves testing in larger groups to confirm safety and effectiveness after initial smaller group trials.

Sevasemten remains investigational, meaning it is not yet approved for general use. The testing aims to ensure it is safe and effective for those with Becker muscular dystrophy. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Becker muscular dystrophy?

Researchers are excited about EDG-5506 for Becker Muscular Dystrophy because it offers a new approach by targeting muscle health directly. Unlike existing treatments that mainly focus on managing symptoms or slowing disease progression, EDG-5506 aims to enhance muscle function and strength, potentially improving quality of life for patients. This treatment is particularly promising because it works by stabilizing muscle fibers, which could reduce muscle damage and lead to better overall outcomes.

What evidence suggests that EDG-5506 might be an effective treatment for Becker muscular dystrophy?

Research has shown that EDG-5506, also known as sevasemten, may help treat Becker muscular dystrophy. In earlier studies, patients who took EDG-5506 experienced a 28% drop in CK enzymes, which indicate muscle damage. This suggests there could be less muscle damage over time. Additionally, patients maintained stable physical abilities, with little change in performing everyday activities. These results suggest that EDG-5506 might help slow muscle weakening in people with this condition. Participants in this trial will receive either EDG-5506 or a placebo to further evaluate its effectiveness. ^{1 2 6 7 8}

Who Is on the Research Team?

Roxana D. Dreghici

Principal Investigator

Edgewise Therapeutics, Inc.

Joanne Donovan, MD, PhD

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.

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.

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?

Group I: Adult Cohort 6 Experimental Treatment 2 Interventions

Drug: Sevasemten Drug: Placebo

Group II: Adult Cohort 2 Experimental Treatment 2 Interventions

Drug: Sevasemten Drug: Placebo

Group III: Adult Cohort 1 Experimental Treatment 2 Interventions

Drug: Sevasemten Drug: Placebo

Group IV: Adolescent Cohort 5 Experimental Treatment 2 Interventions

Drug: Sevasemten Drug: Placebo

Group V: Adolescent Cohort 4 Experimental Treatment 2 Interventions

Drug: Sevasemten Drug: Placebo

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

A study involving 91 non-ambulatory boys and men with Duchenne muscular dystrophy (DMD) demonstrated that reliable assessments of their physical abilities can be achieved, with high reliability scores (ICCs) for various tests, including forced vital capacity (FVC) and upper extremity function.

The research indicated that different forms of corticosteroids had beneficial effects on upper extremity function, lung capacity, and hand strength, suggesting that corticosteroid use should be considered in clinical trials for DMD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Outcome reliability in non-ambulatory boys/men with Duchenne muscular dystrophy. Connolly, AM., Malkus, EC., Mendell, JR., et al.[2021]

In a study of 33 patients with Becker muscular dystrophy (BMD), no clear relationship was found between dystrophin levels (3%-78%) and overall disease severity, suggesting that dystrophin levels above 10% do not significantly impact disease progression.

However, in patients with a specific mutation (exon 45-47 deletion), muscle strength and fatty infiltration were correlated with age, indicating that the mutation site may play a more critical role in disease course than the amount of dystrophin produced.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dystrophin levels and clinical severity in Becker muscular dystrophy patients. van den Bergen, JC., Wokke, BH., Janson, AA., et al.[2017]

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An update on Becker muscular dystrophy. Straub, V., Guglieri, M.[2023]

A new human induced pluripotent stem cell (hiPSC) line was successfully created from fibroblasts of a Becker muscular dystrophy (BMD) patient, which maintains normal genetic characteristics and pluripotency.

This hiPSC line can be used to better understand the disease mechanisms of BMD and for testing potential drug treatments, providing a valuable resource for future research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Generation of a human induced pluripotent stem cell line (INNDSUi003-A) derived from patient with Becker muscular dystrophy (BMD). Ji, X., Wang, Y., Sun, P., et al.[2022]

The novel DT-DEC01 therapy, which uses Dystrophin Expressing Chimeric (DEC) cells, was found to be safe in a small study of 3 boys aged 6-15 years, with no therapy-related adverse events reported up to 22 months after treatment.

Preliminary efficacy was observed through electromyography (EMG) assessments, showing increased motor unit potentials in treated muscles at 12 months, indicating potential benefits for muscle function in boys with Duchenne muscular dystrophy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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. Niezgoda, A., Biegański, G., Wachowiak, J., et al.[2023]

In a phase 3 study involving 131 children aged 4 to under 8 years with Duchenne muscular dystrophy, edasalonexent was generally well-tolerated and had a manageable safety profile, with most side effects being mild gastrointestinal issues like diarrhea.

While edasalonexent did not show statistically significant improvements in overall muscle function compared to placebo, younger patients (6 years or younger) demonstrated more promising results, suggesting that early treatment may help slow disease progression.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial. Finkel, RS., McDonald, CM., Lee Sweeney, H., et al.[2021]

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Neurodevelopmental, behavioral, and emotional symptoms in Becker muscular dystrophy. Lambert, JT., Darmahkasih, AJ., Horn, PS., et al.[2020]

Two boys with Becker's muscular dystrophy showed significant and lasting strength improvements after treatment with prednisone, suggesting that corticosteroids may be beneficial for some patients with this condition.

Both patients experienced a decrease in serum creatine kinase levels, and muscle biopsies indicated a normalization of muscle tissue in one patient during treatment, highlighting the potential efficacy of prednisone in managing Becker's muscular dystrophy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prednisone therapy in Becker's muscular dystrophy. Johnsen, SD.[2017]

In a Phase 2 study involving 51 ambulant Duchenne muscular dystrophy (DMD) patients, drisapersen at a dose of 6 mg/kg/week showed a potential benefit in improving the 6-minute walking distance (6MWD) compared to placebo, with a mean difference of 27.1 meters at week 24.

Drisapersen was generally well-tolerated, with the most common side effects being injection site reactions and subclinical proteinuria, indicating a manageable safety profile for this treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy. McDonald, CM., Wong, B., Flanigan, KM., et al.[2022]

Vamorolone, a novel corticosteroid, shows promise in treating Becker muscular dystrophy (BMD) by improving muscle strength and reducing inflammation in a mouse model, indicating its potential efficacy for this genetic condition.

Compared to prednisolone, vamorolone has a better safety profile, causing fewer side effects related to behavior and growth, while also increasing dystrophin protein levels in both heart and skeletal muscle.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice. McCormack, NM., Nguyen, NY., Tully, CB., et al.[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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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]

In a phase 2 study involving boys aged 4 to 8 with Duchenne muscular dystrophy, edasalonexent was found to be well-tolerated, with mild gastrointestinal side effects and no serious adverse events reported.

The treatment with edasalonexent at 100 mg/kg/day showed promising results in slowing disease progression and preserving muscle function, alongside reductions in NF-κB-regulated gene levels and improvements in muscle health biomarkers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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. Finkel, RS., Finanger, E., Vandenborne, K., et al.[2021]

Citations

1. institut-myologie.org institut-myologie.org/en/2025/07/17/edg-5506-sevasemten-stabilises-functional-scores-in-beckers-myopathy/

EDG-5506 (sevasemten) stabilises functional scores in ... In the adult group, 12 months' treatment with EDG-5506 reduced the level of CK enzymes in the blood by 28%, and resulted in relatively stable ...

2. edgewisetx.com edgewisetx.com/wp-content/uploads/2024/08/WMS_Symposium_Donovan_ARCH_6_mos_FINAL_SLIDES_for_pdf.pdf

EDG-5506 pharmacokinetics (PK) of EDG-5506 in adults with Becker muscular dystrophy ... Source: Data on file for month 4 (n=10) or 6 (n=2) as available. Baseline.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05291091

NCT05291091 | Phase 2 Study of EDG-5506 in Becker ... A double-blind, randomized, placebo-controlled design to investigate the safety and efficacy of sevasemten in adults with Becker muscular dystrophy after 18 ...

4. neurologylive.com neurologylive.com/view/therapeutic-potential-edg-5506-treating-becker-muscular-dystrophy-joanne-donovan

Therapeutic Potential of EDG-5506 in Treating Becker ... The results so far have been highly encouraging, with reductions in muscle damage biomarkers and physical improvements seen in patients." Becker ...

5. emjreviews.com emjreviews.com/neurology/symposium/spotlight-on-becker-muscular-dystrophy-understanding-the-lived-experience-of-becker-and-clinical-advancements-with-a-novel-agent-s110225/

Spotlight on Becker Muscular Dystrophy Notably, patients also showed stabilisation of function, with a mean change of -0.2 points in NSAA score at 24 months,15 diverging from the ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05160415?cond=Becker%20muscular%20dystrophy&rank=3

A Study of EDG-5506 in Adult Males With Becker Muscular ... This open-label study evaluated the safety, tolerability, and pharmacokinetics (PK) of sevasemten in participants with BMD who completed the ...

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40452637/

A Phase 1, Double-Blind, Placebo-Controlled Trial of ... Sevasemten (EDG-5506) is an orally administered, investigational small molecule that selectively modulates fast muscle fiber contraction by inhibiting fast ...

8. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0960896622004424

P.124 EDG-5506 targets fast skeletal myosin and reduces ... To assess safety, pharmacokinetics (PK), pharmacodynamics and biomarkers of muscle damage in healthy adults and adults with BMD.