Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Sevasemten.
- Who it may be relevant to
- Registry conditions: Becker Muscular Dystrophy. Basic parameters: No limits · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Belgium, Denmark, France, Germany +5
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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
Overview
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.
Interventions
- Drug Sevasemten
Sevasemten is administered orally once per day
Primary outcome measures
- Number of adverse events in those treated with sevasemten [Time frame: 55 Months]
- Severity of adverse events in those treated with sevasemten [Time frame: 55 Months]
Secondary outcome measures (2)
- Incidence of treatment-emergent abnormal clinical chemistry laboratory test results [Time frame: 54 Months]
- Incidence of treatment-emergent abnormal hematology laboratory test results [Time frame: 54 Months]
Eligibility criteria
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 \[Week 52\])
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 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.
3\. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 24 centers
- Arkansas Children's Hospital — Little Rock
- UC San Diego — La Jolla
- UCLA Medical Center — Los Angeles
- UC Irvine Medical Center — Orange
- Stanford Neuroscience Health Center — Palo Alto
- UC Davis Medical Center — Sacramento
- UC Denver — Aurora
- University of Florida — Gainesville
- … and 16 more centers
France · 4 centers
- Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timon — Marseille
- CHU de Nantes — Nantes
- CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires — Nice
- AP-HP Hopital Pitie-Salpetriere — Paris
Spain · 4 centers
- Hospital Universitario Vall d'Hebron — Barcelona
- Hospital Universitario de Bellvitge — Barcelona
- Hospital Universitario Donostia — Donostia / San Sebastian
- Hospital Universitari i Politecnic La Fe — Valencia
United Kingdom · 4 centers
- University College London Hospital — London
- St. George's University Hospitals NHS Foundation Trust — London
- Newcastle Freeman Hospital — Newcastle
- Salford Royal Hospital — Salford
Belgium · 3 centers
- University Hospital Gent — Ghent
- Universitaire Ziekenhuizen Leuven — Leuven
- Centre Hospitalier Régional de la Citadelle — Liége
Italy · 3 centers
- Fondazione IRCCS Ca'Granda Ospedale Maggiore — Milan
- Azienda Ospedale - Università Padova — Padova
- Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuo — Rome
Denmark · 1 center
- Rigshospitalet — Copenhagen
Germany · 1 center
- Klinikum der Ludwig-Maximilians-Universitaet Muenchen — Munich
Israel · 1 center
- Hadassah University Hospital — Jerusalem
Netherlands · 1 center
- Leids Universitair Medisch Centrum — Leiden
Identifiers
NCT: NCT06066580 · EDG-5506-203