Back to resultsOpen-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy (MESA)
Primary Purpose
Becker Muscular Dystrophy
Status
Enrolling by invitation
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
EDG-5506
Sponsored by
About this trial
This is an interventional treatment trial for Becker Muscular Dystrophy focused on measuring Becker Muscular Dystrophy
Eligibility Criteria
Inclusion Criteria: 1. Participation in EDG-5506-002 ARCH, EDG-5506-201 CANYON and GRAND CANYON, or EDG-5506-202 DUNE. Participants are eligible if they complete the respective prior study visits as follows: EDG-5506-002 ARCH: Complete the final study Visit 27 [Month 24]; or, completion of the ET visit prior to Visit 27 [Month 24] EDG-5506-201 CANYON and GRAND CANYON: Complete the final study visit (Cohorts 1, 2, 4, and 5: Visit 12 [Month 12]; Cohort 6: Visit 11 [Month 18]) EDG-5506-202 DUNE: Complete at least 36 weeks of open-label treatment Exclusion Criteria: Any clinically significant changes during or following participation in EDG-5506-002, EDG-5506-201, or EDG-5506-202 that would affect the potential safety of the participant to receive EDG-5506. Receipt of an investigational drug other than EDG-5506 within 30 days or 5 half-lives (whichever is longer) of dosing in the present study. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
Sites / Locations
- Rare Disease Research
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Treatment
Arm Description
Drug: EDG-5506
Outcomes
Primary Outcome Measures
Number of adverse events in those treated with EDG-5506
Severity of adverse events in those treated with EDG-5506
Secondary Outcome Measures
Incidence of treatment-emergent abnormal clinical chemistry laboratory test results
Incidence of treatment-emergent abnormal hematology laboratory test results
Full Information
NCT ID
NCT06066580
First Posted
September 27, 2023
Last Updated
September 27, 2023
Sponsor
Edgewise Therapeutics, Inc.
Collaborators
Medpace, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT06066580
Brief Title
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
Acronym
MESA
Official Title
An Open-Label Extension Study to Assess the Long-term Effect of EDG-5506 on Safety, Biomarkers, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
October 2023 (Anticipated)
Primary Completion Date
March 2028 (Anticipated)
Study Completion Date
March 2028 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Edgewise Therapeutics, Inc.
Collaborators
Medpace, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
EDG-5506-203 MESA is an open-label extension study to assess the long-term effect of EDG-5506 on safety, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy
Detailed Description
This is an open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of EDG-5506. EDG-5506-203 MESA will provide continued access to EDG-5506 treatment to participants with Becker muscular dystrophy who were previously enrolled in EDG-5506-002 ARCH, completed EDG-5506-201 CANYON and GRAND CANYON, or completed EDG-5506-202 DUNE.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Becker Muscular Dystrophy
Keywords
Becker Muscular Dystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
200 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Treatment
Arm Type
Experimental
Arm Description
Drug: EDG-5506
Intervention Type
Drug
Intervention Name(s)
EDG-5506
Intervention Description
EDG-5506 is administered orally once per day
Primary Outcome Measure Information:
Title
Number of adverse events in those treated with EDG-5506
Time Frame
19 Months
Title
Severity of adverse events in those treated with EDG-5506
Time Frame
19 Months
Secondary Outcome Measure Information:
Title
Incidence of treatment-emergent abnormal clinical chemistry laboratory test results
Time Frame
18 Months
Title
Incidence of treatment-emergent abnormal hematology laboratory test results
Time Frame
18 Months
10. Eligibility
Sex
Male
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
1. Participation in EDG-5506-002 ARCH, EDG-5506-201 CANYON and GRAND CANYON, or EDG-5506-202 DUNE. Participants are eligible if they complete the respective prior study visits as follows:
EDG-5506-002 ARCH: Complete the final study Visit 27 [Month 24]; or, completion of the ET visit prior to Visit 27 [Month 24]
EDG-5506-201 CANYON and GRAND CANYON: Complete the final study visit (Cohorts 1, 2, 4, and 5: Visit 12 [Month 12]; Cohort 6: Visit 11 [Month 18])
EDG-5506-202 DUNE: Complete at least 36 weeks of open-label treatment
Exclusion Criteria:
Any clinically significant changes during or following participation in EDG-5506-002, EDG-5506-201, or EDG-5506-202 that would affect the potential safety of the participant to receive EDG-5506.
Receipt of an investigational drug other than EDG-5506 within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.
Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sam Collins, MBBS, PhD
Organizational Affiliation
Edgewise Therapeutics, Inc.
Official's Role
Study Chair
Facility Information:
Facility Name
Rare Disease Research
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Links:
URL
http://www.edgewisetx.com
Description
Sponsor Website
Learn more about this trial
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
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