Becker Muscular Dystrophy Clinical Trial
— MESAOfficial 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
| Verified date | March 2024 |
| Source | Edgewise Therapeutics, Inc. |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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
| Status | Enrolling by invitation |
| Enrollment | 200 |
| Est. completion date | March 2028 |
| Est. primary completion date | March 2028 |
| Accepts healthy volunteers | No |
| Gender | Male |
| Age group | N/A and older |
| Eligibility | 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: 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 EDG-5506. 2. 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. 3. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months. |
| Country | Name | City | State |
|---|---|---|---|
| Netherlands | Leids Universitair Medisch Centrum | Leiden | |
| United States | Rare Disease Research | Atlanta | Georgia |
| United States | UC Denver | Aurora | Colorado |
| United States | Kennedy Krieger Institute | Baltimore | Maryland |
| United States | University of Cincinnati Gardner Neuroscience Institute | Cincinnati | Ohio |
| United States | Nationwide Children's Hospital | Columbus | Ohio |
| United States | Neurology Rare Disease Center | Denton | Texas |
| United States | University of Iowa | Iowa City | Iowa |
| United States | University of Kansas Medical Center | Kansas City | Kansas |
| United States | Arkansas Children's Hospital | Little Rock | Arkansas |
| United States | Virginia Commonwealth University Health | Richmond | Virginia |
| United States | UC Davis Medical Center | Sacramento | California |
| United States | Washington University School of Medicine | Saint Louis | Missouri |
| United States | University of Massachusetts Memorial Medical Center | Worcester | Massachusetts |
| Lead Sponsor | Collaborator |
|---|---|
| Edgewise Therapeutics, Inc. | Medpace, Inc. |
United States, Netherlands,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Number of adverse events in those treated with EDG-5506 | 19 Months | ||
| Primary | Severity of adverse events in those treated with EDG-5506 | 19 Months | ||
| Secondary | Incidence of treatment-emergent abnormal clinical chemistry laboratory test results | 18 Months | ||
| Secondary | Incidence of treatment-emergent abnormal hematology laboratory test results | 18 Months |
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