A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

• ClinicalTrials.gov Identifier: NCT05540860
• Recruitment Status: Recruiting
• First Posted: September 15, 2022
• Last Update Posted: April 2, 2024
• Sponsor: Edgewise Therapeutics, Inc.
• Information provided by (Responsible Party): Edgewise Therapeutics, Inc.

Tracking Information

• First Submitted Date: September 6, 2022
• First Posted Date: September 15, 2022
• Last Update Posted Date: April 2, 2024
• Actual Study Start Date: October 24, 2022
• Estimated Primary Completion Date: February 2026 (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: 24 months ]
  All participants
• Severity of adverse events during treatment with EDG-5506 or placebo [ Time Frame: 24 months ]
  All participants

Original Primary Outcome Measures (submitted: September 12, 2022)

• 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

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

• Incidence of abnormal clinical chemistry test results [ Time Frame: 24 months ]
  All participants
• Incidence of abnormal hematology test results [ Time Frame: 24 months ]
  All participants
• Incidence of abnormal coagulation test results [ Time Frame: 24 months ]
  All participants
• Incidence of abnormal urinalysis test results [ Time Frame: 24 months ]
  All participants
• Pharmacokinetics as measured by steady state plasma concentration [ Time Frame: 24 months ]
  All participants
• Change from Baseline in serum creatinine 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 (submitted: September 12, 2022)

• 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
• Number of participants with changes in clinical chemistry [ Time Frame: 12 months ]
  All participants
• Number of participants with changes in hematology [ Time Frame: 12 months ]
  All participants
• Number of participants with changes in coagulation [ Time Frame: 12 months ]
  All participants
• Number of participants with changes in urinalysis [ Time Frame: 12 months ]
  All participants
• Number of participants with changes in vital signs [ Time Frame: 12 months ]
  All participants
• Number of participants with changes in physical examination [ Time Frame: 12 months ]
  All participants
• Number of participants with changes in neurological examination [ Time Frame: 12 months ]
  All participants
• Number of participants with changes in ECG PR interval [ Time Frame: 12 months ]
  All participants
• Number of participants with changes in ECG QRS interval [ Time Frame: 12 months ]
  All participants
• Number of participants with changes in ECG QT interval [ Time Frame: 12 months ]
  All participants
• Number of participants with changes in ECG QTc interval [ Time Frame: 12 months ]
  All participants
• Number of participants with changes in FVC [ 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 creatinine kinase [ Time Frame: 12 weeks ]
  All participants
• Change from Baseline in serum myoglobin [ Time Frame: 12 weeks ]
  All participants
• Change from Baseline in fast skeletal muscle troponin I [ Time Frame: 12 weeks ]
  All participants

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
• Official Title: A 2-part Phase 2 Study of Safety, Pharmacokinetics and Biomarkers in Children With Duchenne Muscular Dystrophy Including a Randomized, Double-Blind, Placebo-Controlled Part A, Followed by an Open-Label Part B
• Brief Summary: The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an open-label part B.

Detailed Description

The EDG-5506-210 protocol was amended to include an additional dose cohort and a cohort to include participants not currently treated with corticosteroids.

This 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 54 children with DMD treated with oral, once-daily EDG-5506 for 24 months. This study will have up to a 4-week Screening period, a 12-week randomized, double-blind, placebo controlled treatment period (Part A), a 92-week open-label extension period (Part B), and a 2-week follow up period.

Approximately 54 participants aged 4 to 9 years inclusive will be randomized to EDG-5506 or placebo in a 2:1 ratio. Five dose cohorts (C1, C2, C3, C4 and C5) of approximately 9 participants each will be enrolled sequentially.

An additional cohort, Cohort 2NS, to include participants (aged 4 to 7 years inclusive) not currently treated with corticosteroids, will enroll approximately 9 participants after Cohort 2 safety review and in parallel with the additional cohorts.

• 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: EDG-5506 Dose 3
  EDG-5506 is administered orally once per day
• Drug: EDG-5506 Dose 4
  EDG-5506 is administered orally once per day
• Drug: EDG-5506 Dose 5
  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
• Experimental: Cohort 3
  Drug: EDG-5506 Drug: Placebo
  Interventions:

  • Drug: EDG-5506 Dose 3
  • Drug: Placebo
• Experimental: Cohort 4
  Drug: EDG-5506 Drug: Placebo
  Interventions:

  • Drug: EDG-5506 Dose 4
  • Drug: Placebo
• Experimental: Cohort 5
  Drug: EDG-5506 Drug: Placebo
  Interventions:

  • Drug: EDG-5506 Dose 5
  • Drug: Placebo
• Experimental: Cohort 2NS
  Drug: EDG-5506 Drug: Placebo
  Interventions:

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

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: January 15, 2024): 54
• Original Estimated Enrollment (submitted: September 12, 2022): 27
• Estimated Study Completion Date: February 2026
• Estimated Primary Completion Date: February 2026 (Final data collection date for primary outcome measure)

Eligibility Criteria

Key Common Inclusion Criteria:

1. A documented mutation on the DMD gene and phenotype consistent with Duchenne muscular dystrophy.
2. Able to complete the stand from supine in ≤ 10 seconds and able to perform the 4-stair climb in < 10 seconds at the Screening visit.
3. Body weight greater than or equal to 15 kg and less than 35 kg at the Screening visit.

For Cohorts 1, 2, 3, 4 and 5:

Aged 4-9 years on a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit.

For Cohort 2 Non-Steroid (Cohort 2NS):

Aged 4-7 years not on corticosteroids within 6 months prior to the Baseline visit.

Key Common Exclusion Criteria:

1. 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 testing.
2. A forced vital capacity < 60% predicted at the Screening visit for those participants who are > 8 years old at Screening.
3. A cardiac echocardiography showing left ventricular ejection < 45% at the Screening visit.
4. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the Screening visit in the present study.
5. Receipt of a stable dose of an approved exon-skipping therapy with a treatment duration of less than 1 year prior to the Screening visit.

For Cohort 2 Non-Steroid (Cohort 2NS):

Receipt of oral corticosteroids for the treatment of Duchenne muscular dystrophy in the previous 6 months. Participants will not be tapered off steroids for the purpose of this study and oral corticosteroids for the treatment of Duchenne muscular dystrophy may be initiated after the Week 16 visit.

Sex/Gender

• Sexes Eligible for Study: Male

• Ages: 4 Years to 9 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: NCT05540860
• Other Study ID Numbers: EDG-5506-210
• 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: April 2024