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

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ClinicalTrials.gov Identifier: NCT06100887
Recruitment Status : Not yet recruiting
First Posted : October 25, 2023
Last Update Posted : October 27, 2023
Sponsor:
Information provided by (Responsible Party):
Edgewise Therapeutics, Inc.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: EDG-5506 Dose 1 Drug: EDG-5506 Dose 2 Drug: Placebo Phase 2

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Estimated Study Start Date : December 2023
Estimated Primary Completion Date : June 2025
Estimated Study Completion Date : June 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Cohort 1
Drug: EDG-5506 Drug: Placebo
 Drug: EDG-5506 Dose 1
EDG-5506 is administered orally once per day

Drug: Placebo
Placebo is administered orally once per day
Experimental: Cohort 2
Drug: EDG-5506 Drug: Placebo
 Drug: EDG-5506 Dose 2
EDG-5506 is administered orally once per day

Drug: Placebo
Placebo is administered orally once per day


Outcome Measures
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Primary Outcome Measures :
  1. Number of adverse events during treatment with EDG-5506 or placebo [ Time Frame: 12 months ]
    All participants

  2. Severity of adverse events during treatment with EDG-5506 or placebo [ Time Frame: 12 months ]
    All participants


Secondary Outcome Measures :
  1. Incidence of abnormal clinical chemistry test results [ Time Frame: 12 months ]
    All participants

  2. Incidence of abnormal hematology test results [ Time Frame: 12 months ]
    All participants

  3. Incidence of abnormal coagulation test results [ Time Frame: 12 months ]
    All participants

  4. Incidence of abnormal urinalysis test results [ Time Frame: 12 months ]
    All participants

  5. Pharmacokinetics as measured by steady state plasma concentration [ Time Frame: 12 months ]
    All participants

  6. Change from Baseline in serum creatine kinase [ Time Frame: 12 weeks ]
    All participants

  7. Change from Baseline in fast skeletal muscle troponin I [ Time Frame: 12 weeks ]
    All participants


Eligibility Criteria
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Ages Eligible for Study:   6 Years to 14 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06100887


Contacts
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Contact: Edgewise Therapeutics 720-262-7002 studies@edgewisetx.com

Locations
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United States, California
UCLA Medical Center
Los Angeles, California, United States, 90095
UC Davis Medical Center
Sacramento, California, United States, 95817
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
United States, Massachusetts
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, United States, 01605
United States, Missouri
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
United States, North Carolina
Rare Disease Research
Hillsborough, North Carolina, United States, 27278
United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Sponsors and Collaborators
Edgewise Therapeutics, Inc.
Investigators
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Study Chair: Sam Collins, MBBS, PhD Edgewise Therapeutics, Inc.
More Information
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Additional Information:

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Responsible Party: Edgewise Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT06100887    
Other Study ID Numbers: EDG-5506-215
First Posted: October 25, 2023    Key Record Dates
Last Update Posted: October 27, 2023
Last Verified: October 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Edgewise Therapeutics, Inc.:
Duchenne Muscular Dystrophy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked