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Trial record 1 of 1 for:    NCT05540860
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A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

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

Study Description
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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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: EDG-5506 Dose 1 Drug: EDG-5506 Dose 2 Drug: EDG-5506 Dose 3 Drug: EDG-5506 Dose 4 Drug: EDG-5506 Dose 5 Drug: Placebo Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 54 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : October 24, 2022
Estimated Primary Completion Date : February 2026
Estimated Study Completion Date : February 2026

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

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

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

Drug: Placebo
Placebo is administered orally once per day
Experimental: Cohort 2NS
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: 24 months ]
    All participants

  2. Severity of adverse events during treatment with EDG-5506 or placebo [ Time Frame: 24 months ]
    All participants


Secondary Outcome Measures :
  1. Incidence of abnormal clinical chemistry test results [ Time Frame: 24 months ]
    All participants

  2. Incidence of abnormal hematology test results [ Time Frame: 24 months ]
    All participants

  3. Incidence of abnormal coagulation test results [ Time Frame: 24 months ]
    All participants

  4. Incidence of abnormal urinalysis test results [ Time Frame: 24 months ]
    All participants

  5. Pharmacokinetics as measured by steady state plasma concentration [ Time Frame: 24 months ]
    All participants

  6. Change from Baseline in serum creatinine 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:   4 Years to 9 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.

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): NCT05540860


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

Locations
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United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
United States, California
UCLA Medical Center Recruiting
Los Angeles, California, United States, 90095
UC Davis Medical Center Recruiting
Sacramento, California, United States, 95817
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
United States, Georgia
Rare Disease Research Recruiting
Atlanta, Georgia, United States, 30329
United States, Iowa
University of Iowa Recruiting
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
United States, Maryland
Kennedy Krieger Institute Recruiting
Baltimore, Maryland, United States, 21205
United States, Massachusetts
University of Massachusetts Memorial Medical Center Recruiting
Worcester, Massachusetts, United States, 01605
United States, Missouri
Washington University School of Medicine Recruiting
Saint Louis, Missouri, United States, 63110
United States, Ohio
Cincinnati Children's Hospital Recruiting
Cincinnati, Ohio, United States, 45229
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
United States, Texas
Cook Children's Medical Center Recruiting
Fort Worth, Texas, United States, 76104
Sponsors and Collaborators
Edgewise Therapeutics, Inc.
More Information
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Additional Information:

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Responsible Party: Edgewise Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05540860    
Other Study ID Numbers: EDG-5506-210
First Posted: September 15, 2022    Key Record Dates
Last Update Posted: April 2, 2024
Last Verified: April 2024
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