Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
- Registration Number
- NCT06066580
- Lead Sponsor
- Edgewise Therapeutics, Inc.
- Brief 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
- Detailed Description
This is an open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of sevasemten. EDG-5506-203 MESA will provide continued access to sevasemten 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.
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- Male
- Target Recruitment
- 260
- 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
-
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.
-
Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers.
-
Receipt of an investigational drug other than sevasemten 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Treatment Sevasemten Drug: Sevasemten
- Primary Outcome Measures
Name Time Method Number of adverse events in those treated with sevasemten 37 Months Severity of adverse events in those treated with sevasemten 37 Months
- Secondary Outcome Measures
Name Time Method Incidence of treatment-emergent abnormal clinical chemistry laboratory test results 36 Months Incidence of treatment-emergent abnormal hematology laboratory test results 36 Months
Trial Locations
- Locations (41)
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
UC San Diego
🇺🇸La Jolla, California, United States
UCLA Medical Center
🇺🇸Los Angeles, California, United States
UC Irvine Medical Center
🇺🇸Orange, California, United States
Stanford Neuroscience Health Center
🇺🇸Palo Alto, California, United States
UC Davis Medical Center
🇺🇸Sacramento, California, United States
UC Denver
🇺🇸Aurora, Colorado, United States
University of Florida
🇺🇸Gainesville, Florida, United States
Rare Disease Research
🇺🇸Atlanta, Georgia, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
Kennedy Krieger Institute
🇺🇸Baltimore, Maryland, United States
University of Massachusetts Memorial Medical Center
🇺🇸Worcester, Massachusetts, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
University of Rochester Medical Center
🇺🇸Rochester, New York, United States
Rare Disease Research, LLC NC
🇺🇸Hillsborough, North Carolina, United States
University of Cincinnati Gardner Neuroscience Institute
🇺🇸Cincinnati, Ohio, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
🇮🇹Rome, Italy
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
National Neuromuscular Research Institute
🇺🇸Austin, Texas, United States
Neurology Rare Disease Center
🇺🇸Denton, Texas, United States
Virginia Commonwealth University Health
🇺🇸Richmond, Virginia, United States
University Hospital Gent
🇧🇪Gent, Belgium
Universitaire Ziekenhuizen Leuven
🇧🇪Leuven, Belgium
Centre Hospitalier Régional de la Citadelle
🇧🇪Liége, Belgium
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
🇫🇷Marseille, France
CHU de Nantes
🇫🇷Nantes Cedex, France
CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
🇫🇷Nice Cedex 1, France
AP-HP Hopital Pitie-Salpetriere
🇫🇷Paris, France
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
🇩🇪Munich, Germany
Fondazione IRCCS Ca'Granda Ospedale Maggiore
🇮🇹Milan, Italy
Azienda Ospedale - Università Padova
🇮🇹Padova, Italy
Leids Universitair Medisch Centrum
🇳🇱Leiden, Netherlands
Hospital Universitario Vall d'Hebron
🇪🇸Barcelona, Spain
Hospital Universitario de Bellvitge
🇪🇸Barcelona, Spain
Hospital Universitario Donostia
🇪🇸San Sebastian, Spain
Hospital Universitari i Politecnic La Fe
🇪🇸Valencia, Spain
University College London Hospital
🇬🇧London, United Kingdom
Newcastle Freeman Hospital
🇬🇧Newcastle, United Kingdom
Salford Royal Hospital
🇬🇧Salford, United Kingdom