Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy

NCT06066580
clinicaltrials.gov
Phase
Phase 2
clinicaltrials.gov
Recruitment status
Enrolling by invitation
clinicaltrials.gov
Participants
260
clinicaltrials.gov
Lead sponsor
Edgewise Therapeutics, Inc.
clinicaltrials.gov
Start date
2023-11-02
clinicaltrials.gov
Number of sites
48
clinicaltrials.gov

Summary
EDG-5506-203 MESA is an open-label extension study to assess the long-term effect of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy
clinicaltrials.gov
Acronym
MESA
clinicaltrials.gov
Short title
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
clinicaltrials.gov
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
clinicaltrials.gov

Control arm
Open label
clinicaltrials.gov
Registry reports results
No
clinicaltrials.gov
Listing at source
Listed
clinicaltrials.gov
Recruitment status
Enrolling by invitation
clinicaltrials.gov
Phase
Phase 2
clinicaltrials.gov
Study type
Interventional
clinicaltrials.gov

Completion date
2031-02-01
clinicaltrials.gov
First posted
2023-10-04
clinicaltrials.gov
Registry last updated
2026-09-17
clinicaltrials.gov
Primary completion date
2031-02-01
clinicaltrials.gov
Start date
2023-11-02
clinicaltrials.gov

Participants
260
clinicaltrials.gov
Participant count basis
Estimated
clinicaltrials.gov
Planned participants
260
clinicaltrials.gov
Protocol amendments
6
clinicaltrials.gov
Number of sites
48
clinicaltrials.gov

Data monitoring committee
Yes
clinicaltrials.gov
Registry sponsor category
Industry
clinicaltrials.gov
Lead sponsor
Edgewise Therapeutics, Inc.
clinicaltrials.gov
Source registry
ClinicalTrials.gov
clinicaltrials.gov

Registry ID
NCT06066580
clinicaltrials.gov
ClinicalTrials.gov ID
NCT06066580
clinicaltrials.gov
Registry record
Open registry record
clinicaltrials.gov

Allocation
Not applicable
Model
Single group
Purpose
Treatment
Masking
No masking
Sources for study design
clinicaltrials.gov

  1. Number of adverse events in those treated with sevasemten

    Timeframe: 55 Months

  2. Severity of adverse events in those treated with sevasemten

    Timeframe: 55 Months

  1. Incidence of treatment-emergent abnormal clinical chemistry laboratory test results

    Timeframe: 54 Months

  2. Incidence of treatment-emergent abnormal hematology laboratory test results

    Timeframe: 54 Months

Sources for outcomes
clinicaltrials.gov

Sex
Male
Age groups
Children, Adults, Older adults
Healthy volunteers
No

Key Inclusion Criteria:

1. Males with a diagnosis of BMD and 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 (Visit 14 [Week 52])

Key Exclusion Criteria:

  1. 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 sevasemten.
  2. Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers.
  3. Receipt of an investigational drug other than sevasemten within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.
  4. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
Sources for eligibility
clinicaltrials.gov

  • Arkansas Children's HospitalLittle Rock, United States
  • UC San DiegoLa Jolla, United States
  • UCLA Medical CenterLos Angeles, United States
  • UC Irvine Medical CenterOrange, United States
  • Stanford Neuroscience Health CenterPalo Alto, United States
  • UC Davis Medical CenterSacramento, United States
  • UC DenverAurora, United States
  • University of FloridaGainesville, United States
  • Rare Disease ResearchAtlanta, United States
  • Northwestern UniversityChicago, United States
  • Indiana University School of MedicineIndianapolis, United States
  • University of IowaIowa City, United States
  • University of Kansas Medical CenterKansas City, United States
  • Kennedy Krieger InstituteBaltimore, United States
  • University of Massachusetts Memorial Medical CenterWorcester, United States
  • Washington University School of MedicineSt Louis, United States
  • University of Rochester Medical CenterRochester, United States
  • Rare Disease Research, LLC NCHillsborough, United States
  • University of Cincinnati Gardner Neuroscience InstituteCincinnati, United States
  • Nationwide Children's HospitalColumbus, United States
  • University of PennsylvaniaPhiladelphia, United States
  • National Neuromuscular Research InstituteAustin, United States
  • Neurology Rare Disease CenterDenton, United States
  • Virginia Commonwealth University HealthRichmond, United States
  • St Vincent's HospitalMelbourne, Australia

And 23 more, listed on the interactive page.

Sources for sites
clinicaltrials.gov

Sources for references
clinicaltrials.gov

Related records

Drugs (1)

Collaborators (1)

Lead sponsors (1)

Conditions (2)