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EDG-5506 for Duchenne Muscular Dystrophy

Phase 2

Recruiting

Research Sponsored by Edgewise Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Body weight ≥ 20 kg at the Screening visit

Aged 6 to 14 with a documented mutation on the DMD gene and phenotype consistent with DMD

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Awards & highlights

Study Summary

This trial tests a gene therapy in kids with Duchenne muscular dystrophy to see if it is safe and works. Part A is a blind placebo-controlled study, followed by an open-label study in Part B.

Who is the study for?

This trial is for children and adolescents aged 6 to 14 with Duchenne muscular dystrophy who have had gene therapy. They must be able to stand from lying down in under 8 seconds, climb stairs in less than 10 seconds, weigh at least 20 kg, and have been on a stable dose of corticosteroids for over six months.Check my eligibility

What is being tested?

The FOX study tests EDG-5506's safety and effects compared to a placebo in kids previously treated with gene therapy for Duchenne muscular dystrophy. Part A is blinded and randomized; participants don't know if they're getting the drug or placebo. Part B will be open-label.See study design

What are the potential side effects?

Specific side effects of EDG-5506 are not listed but may include reactions typical of clinical trials such as nausea, headache, fatigue or allergic reactions. The trial aims to assess these potential side effects thoroughly.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I weigh at least 20 kg.

Select...

I am between 6 and 14 years old with a confirmed DMD gene mutation.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of adverse events during treatment with EDG-5506 or placebo

Severity of adverse events during treatment with EDG-5506 or placebo

Secondary outcome measures

Change from Baseline in fast skeletal muscle troponin I

Change from Baseline in serum creatine kinase

Incidence of abnormal clinical chemistry test results

+4 more

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 2Experimental Treatment2 Interventions

Drug: EDG-5506 Drug: Placebo

Group II: Cohort 1Experimental Treatment2 Interventions

Drug: EDG-5506 Drug: Placebo

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Placebo

1995

Completed Phase 3

~2670

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Edgewise Therapeutics, Inc.Lead Sponsor

11 Previous Clinical Trials

842 Total Patients Enrolled

Sam Collins, MBBS, PhDStudy ChairEdgewise Therapeutics, Inc.

6 Previous Clinical Trials

461 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the age limit of this research restricted to those under thirty?

"As outlined in the prerequisites, participants of this research must be between 6 to 14 years old."

Answered by AI

How many medical centers have this research project underway?

"Seven research facilities are participating in this trial, such as UC Davis Medical Center (Sacramento), University of Florida (Gainesville) and University of Massachusetts Memorial Medical Centre (Worcester)."

Answered by AI

How precarious is Cohort 1 for individuals?

"After assessing the safety of Cohort 1 using a scale from 1 to 3, we at Power assigned it an intermediate rating of 2. This is due to its Phase 2 status, which implies that there are data points confirming safety but none indicating efficacy."

Answered by AI

Could I enroll in this clinical investigation?

"To qualify for this clinical experiment, individuals with Duchenne muscular dystrophy must be between 6 and 14 years of age. The research is searching for 24 participants in total."

Answered by AI

Is there still the opportunity to be part of this trial?

"Clinicaltrials.gov's directory indicates that this particular trial, which was initially posted on December 1st 2023 and edited lastly on October 10th 2023, is no longer seeking participants. Nevertheless, there are 90 additional clinical trials currently open for enrollment."

Answered by AI

What aims does this research endeavor seek to fulfill?

"This clinical trial will span approximately 12 months and its primary outcome is the severity of adverse events while participants are receiving EDG-5506 or a placebo. Secondary outcomes include abnormal hematology, coagulation, and urinalysis results among all participants."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2024. "Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy (FOX)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06100887.

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