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A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

NCT ID: NCT05540860

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

76 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-10-24

Study Completion Date

2027-01-31

Brief Summary

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The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an open-label part B.

Detailed Description

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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 in approximately 72 children with DMD treated with oral, once-daily sevasemten for up to 48 months. This study will have up to a 4-week Screening period, a 12-week randomized, double-blind, placebo controlled treatment period (Part A), up to a 196-week open-label extension period (Part B), and a 2-week follow up period.

Approximately 72 participants aged 4 to 9 years inclusive will be randomized to sevasemten or placebo in a 2:1 ratio. Five dose cohorts (C1, C2, C3, C4 and C5) of approximately 9 participants each will be enrolled sequentially. Approximately 18 total additional participants may be added across Cohorts 2, 3, or 4.

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.

After review of emerging data, the protocol was amended so all dose cohorts receive the same dose in Part B.

Conditions

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Duchenne Muscular Dystrophy

Keywords

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Duchenne Muscular Dystrophy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Cohort 1

Drug: Sevasemten Drug: Placebo

Group Type EXPERIMENTAL

Sevasemten Dose 1

Intervention Type DRUG

Sevasemten is administered orally once per day

Placebo

Intervention Type DRUG

Placebo is administered orally once per day

Cohort 2

Drug: Sevasemten Drug: Placebo

Group Type EXPERIMENTAL

Sevasemten Dose 2

Intervention Type DRUG

Sevasemten is administered orally once per day

Placebo

Intervention Type DRUG

Placebo is administered orally once per day

Cohort 3

Drug: Sevasemten Drug: Placebo

Group Type EXPERIMENTAL

Sevasemten Dose 3

Intervention Type DRUG

Sevasemten is administered orally once per day

Placebo

Intervention Type DRUG

Placebo is administered orally once per day

Cohort 4

Drug: Sevasemten Drug: Placebo

Group Type EXPERIMENTAL

Sevasemten Dose 4

Intervention Type DRUG

Sevasemten is administered orally once per day

Placebo

Intervention Type DRUG

Placebo is administered orally once per day

Cohort 5

Drug: Sevasemten Drug: Placebo

Group Type EXPERIMENTAL

Sevasemten Dose 5

Intervention Type DRUG

Sevasemten is administered orally once per day

Placebo

Intervention Type DRUG

Placebo is administered orally once per day

Cohort 2NS

Drug: Sevasemten Drug: Placebo

Group Type EXPERIMENTAL

Sevasemten Dose 2

Intervention Type DRUG

Sevasemten is administered orally once per day

Placebo

Intervention Type DRUG

Placebo is administered orally once per day

Interventions

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Sevasemten Dose 1

Sevasemten is administered orally once per day

Intervention Type DRUG

Sevasemten Dose 2

Sevasemten is administered orally once per day

Intervention Type DRUG

Sevasemten Dose 3

Sevasemten is administered orally once per day

Intervention Type DRUG

Sevasemten Dose 4

Sevasemten is administered orally once per day

Intervention Type DRUG

Sevasemten Dose 5

Sevasemten is administered orally once per day

Intervention Type DRUG

Placebo

Placebo is administered orally once per day

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. A documented mutation on the DMD gene and phenotype consistent with Duchenne muscular dystrophy.
2. Able to complete the stand from supine in ≤ 10 seconds and able to perform the 4-stair climb in \< 10 seconds at the Screening visit.
3. Body weight greater than or equal to 15 kg at the Screening visit.

For Cohorts 1, 2, 3, 4 and 5:

Aged 4-9 years on a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit.

For Cohort 2 Non-Steroid (Cohort 2NS):

Aged 4-7 years not on corticosteroids within 6 months prior to the Baseline visit.

Exclusion Criteria

1. Medical history or clinically significant physical exam/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. This includes venous access that would be too difficult to facilitate repeated blood testing.
2. A forced vital capacity \< 60% predicted at the Screening visit for those participants who are \> 8 years old at Screening.
3. A cardiac echocardiography showing left ventricular ejection \< 45% at the Screening visit.
4. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the Screening visit in the present study.
5. Receipt of a stable dose of an approved exon-skipping therapy with a treatment duration of less than 1 year prior to the Screening visit.

For Cohort 2 Non-Steroid (Cohort 2NS):

Receipt of oral corticosteroids for the treatment of Duchenne muscular dystrophy in the previous 6 months. Participants will not be tapered off steroids for the purpose of this study and oral corticosteroids for the treatment of Duchenne muscular dystrophy may be initiated after the Week 16 visit.
Minimum Eligible Age

4 Years

Maximum Eligible Age

9 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Edgewise Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Arkansas Children's Hospital

Little Rock, Arkansas, United States

Site Status

UCLA Medical Center

Los Angeles, California, United States

Site Status

UC Davis Medical Center

Sacramento, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

Rare Disease Research

Atlanta, Georgia, United States

Site Status

University of Iowa

Iowa City, Iowa, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Kennedy Krieger Institute

Baltimore, Maryland, United States

Site Status

University of Massachusetts Memorial Medical Center

Worcester, Massachusetts, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Cook Children's Medical Center

Fort Worth, Texas, United States

Site Status

Countries

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United States

Related Links

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http://edgewisetx.com

Sponsor Website

Other Identifiers

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EDG-5506-210

Identifier Type: -

Identifier Source: org_study_id