A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Drug: Sevasemten Dose 2Drug: Sevasemten Dose 3Drug: Sevasemten Dose 4Drug: Sevasemten Dose 5Drug: Sevasemten Dose 1Drug: Placebo
- Registration Number
- NCT05540860
- Lead Sponsor
- Edgewise Therapeutics, Inc.
- Brief Summary
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
The EDG-5506-210 protocol was amended to include an additional dose cohort and a cohort to include participants not currently treated with corticosteroids.
This is a 2-part, multi-center, Phase 2 study to evaluate the effect of EDG-5506 on safety, pharmacokinetics and biomarkers of muscle damage in approximately 54 children with DMD treated with oral, once-daily EDG-5506 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 54 participants aged 4 to 9 years inclusive will be randomized to EDG-5506 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.
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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- Male
- Target Recruitment
- 76
- A documented mutation on the DMD gene and phenotype consistent with Duchenne muscular dystrophy.
- 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.
- Body weight greater than or equal to 15 kg and less than 35 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
- 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.
- A forced vital capacity < 60% predicted at the Screening visit for those participants who are > 8 years old at Screening.
- A cardiac echocardiography showing left ventricular ejection < 45% at the Screening visit.
- Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the Screening visit in the present study.
- 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
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Cohort 1 Placebo Drug: Sevasemten Drug: Placebo Cohort 2 Sevasemten Dose 2 Drug: Sevasemten Drug: Placebo Cohort 2 Placebo Drug: Sevasemten Drug: Placebo Cohort 3 Sevasemten Dose 3 Drug: Sevasemten Drug: Placebo Cohort 3 Placebo Drug: Sevasemten Drug: Placebo Cohort 4 Sevasemten Dose 4 Drug: Sevasemten Drug: Placebo Cohort 4 Placebo Drug: Sevasemten Drug: Placebo Cohort 5 Sevasemten Dose 5 Drug: Sevasemten Drug: Placebo Cohort 5 Placebo Drug: Sevasemten Drug: Placebo Cohort 2NS Sevasemten Dose 2 Drug: Sevasemten Drug: Placebo Cohort 2NS Placebo Drug: Sevasemten Drug: Placebo Cohort 1 Sevasemten Dose 1 Drug: Sevasemten Drug: Placebo
- Primary Outcome Measures
Name Time Method Number of adverse events during treatment with sevasemten or placebo 24 months All participants
Severity of adverse events during treatment with sevasemten or placebo 24 months All participants
- Secondary Outcome Measures
Name Time Method Incidence of abnormal coagulation test results 24 months All participants
Incidence of abnormal urinalysis test results 24 months All participants
Incidence of abnormal clinical chemistry test results 24 months All participants
Incidence of abnormal hematology test results 24 months All participants
Pharmacokinetics as measured by steady state plasma concentration 24 months All participants
Change from Baseline in serum creatinine kinase 12 weeks All participants
Change from Baseline in fast skeletal muscle troponin I 12 weeks All participants
Trial Locations
- Locations (14)
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
UCLA Medical Center
🇺🇸Los Angeles, California, United States
UC Davis Medical Center
🇺🇸Sacramento, California, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
University of Florida
🇺🇸Gainesville, Florida, United States
Rare Disease Research
🇺🇸Atlanta, Georgia, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
Kennedy Krieger Institute
🇺🇸Baltimore, Maryland, United States
University of Massachusetts Memorial Medical Center
🇺🇸Worcester, Massachusetts, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
Cincinnati Children's Hospital
🇺🇸Cincinnati, Ohio, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Cook Children's Medical Center
🇺🇸Fort Worth, Texas, United States