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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05540860
Recruitment Status : Recruiting
First Posted : September 15, 2022
Last Update Posted : January 31, 2023
Sponsor:
Information provided by (Responsible Party):
Edgewise Therapeutics, Inc.

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: Placebo Phase 2

Detailed Description:

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 27 children with DMD treated with oral, once-daily EDG-5506 for 12 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 40-week open-label extension period (Part B), and a 2-week follow up period.

Twenty-seven (27) participants aged 4 to 9 years inclusive will be randomized to EDG-5506 or placebo in a 2:1 ratio. Three dose cohorts will be enrolled sequentially. Within each cohort, randomization will be stratified by age at screening (< 7.0 or ≥ 7.0 years old).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 27 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 : June 2024
Estimated Study Completion Date : June 2024


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




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. Number of participants with changes in clinical chemistry [ Time Frame: 12 months ]
    All participants

  6. Number of participants with changes in hematology [ Time Frame: 12 months ]
    All participants

  7. Number of participants with changes in coagulation [ Time Frame: 12 months ]
    All participants

  8. Number of participants with changes in urinalysis [ Time Frame: 12 months ]
    All participants

  9. Number of participants with changes in vital signs [ Time Frame: 12 months ]
    All participants

  10. Number of participants with changes in physical examination [ Time Frame: 12 months ]
    All participants

  11. Number of participants with changes in neurological examination [ Time Frame: 12 months ]
    All participants

  12. Number of participants with changes in ECG PR interval [ Time Frame: 12 months ]
    All participants

  13. Number of participants with changes in ECG QRS interval [ Time Frame: 12 months ]
    All participants

  14. Number of participants with changes in ECG QT interval [ Time Frame: 12 months ]
    All participants

  15. Number of participants with changes in ECG QTc interval [ Time Frame: 12 months ]
    All participants

  16. Number of participants with changes in FVC [ Time Frame: 12 months ]
    All participants

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

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

  19. Change from Baseline in serum myoglobin [ Time Frame: 12 weeks ]
    All participants

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



Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 9 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Aged 4 to 9 years with a documented mutation on the DMD gene and phenotype consistent with Duchenne muscular dystrophy.
  2. Able to complete stand from supine in ≤ 10 seconds at the Screening and Baseline visits and able to perform the 4-stair climb in < 10 seconds at the Screening and Baseline visits.
  3. Body weight greater than or equal to 15 kg and less than 35 kg at the Screening visit and Body Mass index (BMI) between 5th and 95th percentile (CDC 2000) for age.
  4. Treatment with a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit.

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 sampling.
  2. A forced vital capacity < 60% predicted at the Screening visit
  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. Any previous dystrophin targeted therapies require a 6 month washout prior to the Screening visit.
  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.

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, Georgia
Rare Disease Research Recruiting
Atlanta, Georgia, United States, 30329
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
United States, Missouri
Washington University School of Medicine Recruiting
Saint Louis, Missouri, United States, 63110
Sponsors and Collaborators
Edgewise Therapeutics, Inc.
Investigators
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Study Chair: Sam Collins, MBBS, PhD Edgewise Therapeutics, Inc.
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: January 31, 2023
Last Verified: January 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