Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy (FOX)

Duchenne Muscular Dystrophy Clinical Trial

Official title:

A Phase 2 Study to Evaluate the Effect of EDG-5506 on Safety, Pharmacokinetics, and Biomarkers in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06100887
• Other study ID #: EDG-5506-215
• Status: Recruiting
• Phase: Phase 2
• Start date: March 22, 2024
• Est. completion date: June 2025

Study information

• Verified date: March 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 FOX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy including a randomized, double-blind, placebo-controlled Part A, followed by an open-label part B.

Description:

FOX 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 24 children and adolescents with Duchenne muscular dystrophy treated with oral, once-daily EDG-5506.This study will have up to a 4-week Screening period, a 12-week randomized double-blind, placebo-controlled treatment period (Part A), followed by a 40-week open-label extension period (Part B) . Approximately twenty-four (24) participants aged 6 to 14, inclusive, will be randomized to EDG-5506 or placebo in a 2:1 ratio. Two dose cohorts (Cohort 1 and Cohort 2) of approximately 12 participants each will be enrolled.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 24
• Est. completion date: June 2025
• Est. primary completion date: June 2025
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Years to 14 Years

Eligibility

Key Inclusion Criteria:

• Aged 6 to 14 with a documented mutation on the DMD gene and phenotype consistent with DMD.
• Prior receipt of an AAV-based gene therapy (= 2 years after study drug administration in an open-label study or = 3 years after randomization in a randomized study).
• Able to complete stand from supine in = 8 seconds at the Screening visit and able to perform the 4-stair climb in < 10 seconds at the Screening visit.
• Body weight = 20 kg at the Screening visit.
• Treatment with a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit.

Key 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.
• Screening visit cardiac echocardiography showing left ventricular ejection fraction (LVEF) < 40%.
• Receipt of an investigational drug (other than the AAV-based gene therapy per Inclusion criteria) within 30 days or 5 half-lives (whichever is longer) of the Screening visit in the present study.
• Receipt of an exon-skipping therapy within 6 months prior to the Screening 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
• Placebo
Placebo is administered orally once per day

Locations

Country	Name	City	State
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of Florida	Gainesville	Florida
United States	Rare Disease Research	Hillsborough	North Carolina
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	12 months
Primary	Severity of adverse events during treatment with EDG-5506 or placebo	All participants	12 months
Secondary	Incidence of abnormal clinical chemistry test results	All participants	12 months
Secondary	Incidence of abnormal hematology test results	All participants	12 months
Secondary	Incidence of abnormal coagulation test results	All participants	12 months
Secondary	Incidence of abnormal urinalysis test results	All participants	12 months
Secondary	Pharmacokinetics as measured by steady state plasma concentration	All participants	12 months
Secondary	Change from Baseline in serum creatine kinase	All participants	12 weeks
Secondary	Change from Baseline in fast skeletal muscle troponin I	All participants	12 weeks

External Links

•   Sponsor Website