Clinical trial NCT06066580: Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
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
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE2
Conditions: Becker Muscular Dystrophy
Keywords: Becker Muscular Dystrophy
Lead sponsor: Edgewise Therapeutics, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Medpace, Inc.
First posted: 2023-10-04
Enrollment: 260
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

3. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.

Primary outcomes:
measure: Number of adverse events in those treated with sevasemten
timeFrame: 37 Months
measure: Severity of adverse events in those treated with sevasemten
timeFrame: 37 Months
Secondary outcomes:
measure: Incidence of treatment-emergent abnormal clinical chemistry laboratory test results
timeFrame: 36 Months
measure: Incidence of treatment-emergent abnormal hematology laboratory test results
timeFrame: 36 Months
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: La Jolla
country: United States
facility: UC San Diego
state: California
city: Los Angeles
country: United States
facility: UCLA Medical Center
state: California
city: Orange
country: United States
facility: UC Irvine Medical Center
state: California
city: Palo Alto
country: United States
facility: Stanford Neuroscience Health Center
state: California
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
city: Aurora
country: United States
facility: UC Denver
state: Colorado
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Atlanta
country: United States
facility: Rare Disease Research
state: Georgia
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Worcester
country: United States
facility: University of Massachusetts Memorial Medical Center
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Hillsborough
country: United States
facility: Rare Disease Research, LLC NC
state: North Carolina
city: Cincinnati
country: United States
facility: University of Cincinnati Gardner Neuroscience Institute
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Austin
country: United States
facility: National Neuromuscular Research Institute
state: Texas
city: Denton
country: United States
facility: Neurology Rare Disease Center
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University Health
state: Virginia
city: Gent
country: Belgium
facility: University Hospital Gent
city: Leuven
country: Belgium
facility: Universitaire Ziekenhuizen Leuven
city: Liége
country: Belgium
facility: Centre Hospitalier Régional de la Citadelle
city: Marseille
country: France
facility: Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
city: Nantes Cedex
country: France
facility: CHU de Nantes
city: Nice Cedex 1
country: France
facility: CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
city: Paris
country: France
facility: AP-HP Hopital Pitie-Salpetriere
city: Munich
country: Germany
facility: Klinikum der Ludwig-Maximilians-Universitaet Muenchen
city: Milan
country: Italy
facility: Fondazione IRCCS Ca'Granda Ospedale Maggiore
city: Padova
country: Italy
facility: Azienda Ospedale - Università Padova
city: Rome
country: Italy
facility: Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
city: Leiden
country: Netherlands
facility: Leids Universitair Medisch Centrum
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron
city: Barcelona
country: Spain
facility: Hospital Universitario de Bellvitge
city: San Sebastian
country: Spain
facility: Hospital Universitario Donostia
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe
city: London
country: United Kingdom
facility: University College London Hospital
city: Newcastle
country: United Kingdom
facility: Newcastle Freeman Hospital
city: Salford
country: United Kingdom
facility: Salford Royal Hospital
Study officials:
affiliation: Edgewise Therapeutics, Inc.
name: Joanne M. Donovan, MD, PhD
role: STUDY_CHAIR
affiliation: Edgewise Therapeutics, Inc.
name: Roxana D. Dreghici, MD
role: STUDY_CHAIR
References: