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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

A 2-part 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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05540860
• Other study ID #: EDG-5506-210
• Status: Recruiting
• Phase: Phase 2
• Start date: October 24, 2022
• Est. completion date: February 2026

Study information

• Verified date: April 2024
• Source: Edgewise Therapeutics, Inc.
• Contact: Edgewise Therapeutics
• Phone: 720-262-7002
• Email: studies[@]edgewisetx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Recruiting
• Enrollment: 54
• Est. completion date: February 2026
• Est. primary completion date: February 2026
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years to 9 Years

Eligibility

Key Common 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 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 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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• EDG-5506 Dose 1
EDG-5506 is administered orally once per day
• EDG-5506 Dose 2
EDG-5506 is administered orally once per day
• EDG-5506 Dose 3
EDG-5506 is administered orally once per day
• EDG-5506 Dose 4
EDG-5506 is administered orally once per day
• EDG-5506 Dose 5
EDG-5506 is administered orally once per day
• Placebo
Placebo is administered orally once per day

Locations

Country	Name	City	State
United States	Rare Disease Research	Atlanta	Georgia
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Kennedy Krieger Institute	Baltimore	Maryland
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	University of Florida	Gainesville	Florida
United States	University of Iowa	Iowa City	Iowa
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	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	Washington University School of Medicine	Saint Louis	Missouri
United States	University of Massachusetts Memorial Medical Center	Worcester	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Edgewise Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of adverse events during treatment with EDG-5506 or placebo	All participants	24 months
Primary	Severity of adverse events during treatment with EDG-5506 or placebo	All participants	24 months
Secondary	Incidence of abnormal clinical chemistry test results	All participants	24 months
Secondary	Incidence of abnormal hematology test results	All participants	24 months
Secondary	Incidence of abnormal coagulation test results	All participants	24 months
Secondary	Incidence of abnormal urinalysis test results	All participants	24 months
Secondary	Pharmacokinetics as measured by steady state plasma concentration	All participants	24 months
Secondary	Change from Baseline in serum creatinine kinase	All participants	12 weeks
Secondary	Change from Baseline in fast skeletal muscle troponin I	All participants	12 weeks

External Links

•   Sponsor Website