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

Primary Purpose

Duchenne Muscular Dystrophy

Status
Not yet recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
EDG-5506 Dose 1
EDG-5506 Dose 2
Placebo
Sponsored by
Edgewise Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Duchenne Muscular Dystrophy

Eligibility Criteria

6 Years - 14 Years (Child)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • UCLA Medical Center
  • UC Davis Medical Center
  • University of Florida
  • University of Massachusetts Memorial Medical Center
  • Washington University School of Medicine
  • Rare Disease Research
  • Nationwide Children's Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Cohort 1

Cohort 2

Arm Description

Drug: EDG-5506 Drug: Placebo

Drug: EDG-5506 Drug: Placebo

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Incidence of abnormal clinical chemistry test results
All participants
Incidence of abnormal hematology test results
All participants
Incidence of abnormal coagulation test results
All participants
Incidence of abnormal urinalysis test results
All participants
Pharmacokinetics as measured by steady state plasma concentration
All participants
Change from Baseline in serum creatine kinase
All participants
Change from Baseline in fast skeletal muscle troponin I
All participants

Full Information

First Posted
October 20, 2023
Last Updated
October 20, 2023
Sponsor
Edgewise Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT06100887
Brief Title
Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy (FOX)
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
December 2023 (Anticipated)
Primary Completion Date
June 2025 (Anticipated)
Study Completion Date
June 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Edgewise Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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.
Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Duchenne Muscular Dystrophy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
24 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Cohort 1
Arm Type
Experimental
Arm Description
Drug: EDG-5506 Drug: Placebo
Arm Title
Cohort 2
Arm Type
Experimental
Arm Description
Drug: EDG-5506 Drug: Placebo
Intervention Type
Drug
Intervention Name(s)
EDG-5506 Dose 1
Intervention Description
EDG-5506 is administered orally once per day
Intervention Type
Drug
Intervention Name(s)
EDG-5506 Dose 2
Intervention Description
EDG-5506 is administered orally once per day
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo is administered orally once per day
Primary Outcome Measure Information:
Title
Number of adverse events during treatment with EDG-5506 or placebo
Description
All participants
Time Frame
12 months
Title
Severity of adverse events during treatment with EDG-5506 or placebo
Description
All participants
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Incidence of abnormal clinical chemistry test results
Description
All participants
Time Frame
12 months
Title
Incidence of abnormal hematology test results
Description
All participants
Time Frame
12 months
Title
Incidence of abnormal coagulation test results
Description
All participants
Time Frame
12 months
Title
Incidence of abnormal urinalysis test results
Description
All participants
Time Frame
12 months
Title
Pharmacokinetics as measured by steady state plasma concentration
Description
All participants
Time Frame
12 months
Title
Change from Baseline in serum creatine kinase
Description
All participants
Time Frame
12 weeks
Title
Change from Baseline in fast skeletal muscle troponin I
Description
All participants
Time Frame
12 weeks

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
14 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Edgewise Therapeutics
Phone
720-262-7002
Email
studies@edgewisetx.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sam Collins, MBBS, PhD
Organizational Affiliation
Edgewise Therapeutics, Inc.
Official's Role
Study Chair
Facility Information:
Facility Name
UCLA Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
UC Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
University of Massachusetts Memorial Medical Center
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01605
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Rare Disease Research
City
Hillsborough
State/Province
North Carolina
ZIP/Postal Code
27278
Country
United States
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Links:
URL
http://edgewisetx.com
Description
Sponsor Website

Learn more about this trial

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

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