EDG-5506 for Duchenne Muscular Dystrophy

(FOX Trial)

Not currently recruiting at 7 trial locations

Overseen By Edgewise Therapeutics

Age: < 18

Sex: Male

Trial Phase: Phase 2

Sponsor: Edgewise Therapeutics, Inc.

Must be taking: Corticosteroids

Must not be taking: Exon-skipping therapy

Disqualifiers: Low LVEF, 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called EDG-5506 for children and teens with Duchenne muscular dystrophy (DMD), a genetic condition that causes muscle weakness. The researchers aim to assess the safety and effectiveness of the treatment, particularly for those who have already undergone gene therapy. Suitable candidates for this trial are individuals aged 6 to 17 with a confirmed DMD gene mutation who can perform specific physical tasks, such as standing up quickly. The trial includes different groups, with some receiving the treatment and others a placebo (a substance with no active drug), to ensure accurate results. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

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

The trial requires that participants have been on a stable dose of corticosteroids for at least 6 months before starting. It doesn't specify if you need to stop other medications, so it's best to discuss with the trial team.

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

Research has shown that EDG-5506 is generally safe and well-tolerated. In one study, participants experienced only mild side effects, such as diarrhea and headaches. Another study with healthy adult volunteers also examined the safety and tolerability of EDG-5506, supporting its potential as a treatment. Although EDG-5506 remains under study and lacks approval for general use, these early findings suggest it is relatively safe for trial participants. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for Duchenne Muscular Dystrophy, which often focus on managing symptoms and slowing progression with corticosteroids or exon-skipping therapies, EDG-5506 offers a novel approach. Researchers are excited about EDG-5506 because it targets muscle repair and regeneration directly. This drug works by modulating the muscle contraction process, potentially reducing muscle damage and improving muscle function. If successful, EDG-5506 could provide a more direct therapeutic benefit by enhancing the muscle's resilience and repair capability, offering hope for improved quality of life for patients.

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

Research has shown that EDG-5506, also known as Sevasemten, may help treat Duchenne Muscular Dystrophy (DMD). This experimental drug targets fast muscle fibers, potentially reducing muscle damage. Early trials demonstrated that EDG-5506 positively affects biomarkers, indicators of muscle health. Studies on Becker Muscular Dystrophy, a condition similar to DMD, showed improvements in these biomarkers, suggesting possible benefits for DMD patients as well. Although more research is needed, these early results offer hope for improved muscle function in people with DMD. ^{1 2 3 4 6}

Who Is on the Research Team?

Sam Collins, MBBS, PhD

Principal Investigator

Edgewise Therapeutics, Inc.

Are You a Good Fit for This Trial?

This trial is for children and adolescents aged 6 to 14 with Duchenne muscular dystrophy who have had gene therapy. They must be able to stand from lying down in under 8 seconds, climb stairs in less than 10 seconds, weigh at least 20 kg, and have been on a stable dose of corticosteroids for over six months.

Inclusion Criteria

I received AAV-based gene therapy over 2 or 3 years ago, depending on the study type.

I can stand up from lying down in less than 8 seconds and climb 4 stairs in less than 10 seconds.

I weigh at least 20 kg.

Exclusion Criteria

Your heart's pumping ability is less than 40% as shown in a heart ultrasound during the screening visit.

I haven't taken any experimental drugs recently, except for the AAV-based gene therapy.

I have not taken exon-skipping therapy in the last 6 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Up to 4 weeks

Treatment (Part A)

Participants receive a 12-week randomized, double-blind, placebo-controlled treatment with sevasemten or placebo

12 weeks

Open-label Extension (Part B)

Participants continue treatment in a 40-week open-label extension period with sevasemten

40 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• EDG-5506

Trial Overview The FOX study tests EDG-5506's safety and effects compared to a placebo in kids previously treated with gene therapy for Duchenne muscular dystrophy. Part A is blinded and randomized; participants don't know if they're getting the drug or placebo. Part B will be open-label.

How Is the Trial Designed?

Group I: Cohort 3 Experimental Treatment 2 Interventions

Drug: Sevasemten Drug: Placebo

Group II: Cohort 2 Experimental Treatment 2 Interventions

Drug: Sevasemten Drug: Placebo

Group III: Cohort 1 Experimental Treatment 2 Interventions

Drug: Sevasemten Drug: Placebo

Who Is Running the Clinical Trial?

Edgewise Therapeutics, Inc.

Lead Sponsor

Trials

13

Recruited

1,000+

Citations

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

EDG-5506 BMD and Duchenne Muscular Dystrophy (DMD) Represent Severe. Dystrophinopathies. BMD and DMD represent a continuum of the same disease; Edgewise's approach aims ...

2. 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 ...

3. parentprojectmd.org parentprojectmd.org/clinical-trial/a-study-of-edg-5506-in-children-with-duchenne-muscular-dystrophy-lynx/

A Study of EDG-5506 in Children With Duchenne Muscular ... This is a 2-part, multi-center, Phase 2 study to evaluate the effect of sevasemten (EDG-5506) on safety, pharmacokinetics and biomarkers of muscle damage.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05540860

A Study of EDG-5506 in Children With Duchenne Muscular ... The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a ...

5. neurology.org neurology.org/doi/10.1212/WNL.0000000000206199

Effects of EDG-5506, a Fast Myosin Modulator, on Function ... Objective:Assess the effect of treatment with EDG-5506 on biomarkers of muscle damage and function in adults with Becker Muscular Dystrophy.

6. withpower.com withpower.com/trial/edg-5506-for-duchenne-muscular-dystrophy-a6447

EDG-5506 for Duchenne Muscular Dystrophy (LYNX Trial) The treatment was found to be safe and well tolerated, with mild side effects like diarrhea and headache, indicating its potential for therapeutic use in DMD ...