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

• ClinicalTrials.gov Identifier: NCT06100887
• Recruitment Status: Recruiting
• First Posted: October 25, 2023
• Last Update Posted: March 27, 2024
• Sponsor: Edgewise Therapeutics, Inc.
• Information provided by (Responsible Party): Edgewise Therapeutics, Inc.

Tracking Information

• First Submitted Date: October 20, 2023
• First Posted Date: October 25, 2023
• Last Update Posted Date: March 27, 2024
• Actual Study Start Date: March 22, 2024
• Estimated Primary Completion Date: June 2025 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 20, 2023)

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

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: October 20, 2023)

• Incidence of abnormal clinical chemistry test results [ Time Frame: 12 months ]
  All participants
• Incidence of abnormal hematology test results [ Time Frame: 12 months ]
  All participants
• Incidence of abnormal coagulation test results [ Time Frame: 12 months ]
  All participants
• Incidence of abnormal urinalysis test results [ Time Frame: 12 months ]
  All participants
• Pharmacokinetics as measured by steady state plasma concentration [ Time Frame: 12 months ]
  All participants
• Change from Baseline in serum creatine kinase [ Time Frame: 12 weeks ]
  All participants
• Change from Baseline in fast skeletal muscle troponin I [ Time Frame: 12 weeks ]
  All participants

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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
• 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.

• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Sequential Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Duchenne Muscular Dystrophy

Intervention

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

Study Arms

• Experimental: Cohort 1
  Drug: EDG-5506 Drug: Placebo
  Interventions:

  • Drug: EDG-5506 Dose 1
  • Drug: Placebo
• Experimental: Cohort 2
  Drug: EDG-5506 Drug: Placebo
  Interventions:

  • Drug: EDG-5506 Dose 2
  • Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: October 20, 2023): 24
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: June 2025
• Estimated Primary Completion Date: June 2025 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: Male

• Ages: 6 Years to 14 Years (Child)
• Accepts Healthy Volunteers: No

Contacts

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

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT06100887
• Other Study ID Numbers: EDG-5506-215
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Edgewise Therapeutics, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Edgewise Therapeutics, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Edgewise Therapeutics, Inc.
• Verification Date: March 2024