University of Kansas Medical Center

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

Purpose

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.

Condition

Duchenne Muscular Dystrophy

Eligibility

Eligible Ages: Between 4 Years and 9 Years
Eligible Genders: Male
Accepts Healthy Volunteers: No

Inclusion Criteria

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. Key Common

Exclusion Criteria

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.

Study Design

Phase: Phase 2
Study Type: Interventional
Allocation: Randomized
Intervention Model: Sequential Assignment
Primary Purpose: Treatment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

Arm	Description	Assigned Intervention
Experimental Cohort 1	Drug: Sevasemten Drug: Placebo	Drug: Sevasemten Dose 1 Sevasemten is administered orally once per day Drug: Placebo Placebo is administered orally once per day
Experimental Cohort 2	Drug: Sevasemten Drug: Placebo	Drug: Sevasemten Dose 2 Sevasemten is administered orally once per day Drug: Placebo Placebo is administered orally once per day
Experimental Cohort 3	Drug: Sevasemten Drug: Placebo	Drug: Sevasemten Dose 3 Sevasemten is administered orally once per day Drug: Placebo Placebo is administered orally once per day
Experimental Cohort 4	Drug: Sevasemten Drug: Placebo	Drug: Sevasemten Dose 4 Sevasemten is administered orally once per day Drug: Placebo Placebo is administered orally once per day
Experimental Cohort 5	Drug: Sevasemten Drug: Placebo	Drug: Sevasemten Dose 5 Sevasemten is administered orally once per day Drug: Placebo Placebo is administered orally once per day
Experimental Cohort 2NS	Drug: Sevasemten Drug: Placebo	Drug: Sevasemten Dose 2 Sevasemten is administered orally once per day Drug: Placebo Placebo is administered orally once per day

Recruiting Locations

University of Kansas Medical Center
Kansas City, Kansas 66160

More Details

Status: Recruiting
Sponsor: Edgewise Therapeutics, Inc.

Study Contact

Edgewise Therapeutics
720-262-7002
studies@edgewisetx.com

Detailed Description

The EDG-5506-210 protocol was amended to enroll additional participants across select cohorts. 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 24 months. This study will have up to a 4-week Screening period, a 12-week randomized, double-blind, placebo controlled treatment period (Part A), a 92-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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.