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

About

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.

Primary Purpose

Treatment

Study Type

Interventional

Phase

Phase II

Eligibility

Gender

Male

Healthy Volunteers

Minimum Age

4 Years

Maximum Age

9 Years

Inclusion Criteria:

Aged 4 to 9 years with a documented mutation on the DMD gene and phenotype consistent with Duchenne muscular dystrophy.
Able to complete stand from supine in ≤ 10 seconds at the Screening and Baseline visits and able to perform the 4-stair climb in < 10 seconds at the Screening and Baseline visits.
Body weight greater than or equal to 15 kg and less than 35 kg at the Screening visit and Body Mass index (BMI) between 5th and 95th percentile (CDC 2000) for age.
Treatment with a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit.

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 sampling.
A forced vital capacity < 60% predicted at the Screening visit
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. Any previous dystrophin targeted therapies require a 6 month washout prior to the Screening visit.
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.

Join this Trial

Contact our clinical trial navigators for opportunities that may be suitable for you