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

Sponsor

Edgewise Therapeutics, Inc. (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT06066580

Collaborator

Medpace, Inc. (Industry)

200

Enrollment

Location

Arm

Anticipated Duration (Months)

3.8

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Becker Muscular Dystrophy	Drug: EDG-5506	Phase 2

Detailed Description

This is an open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of EDG-5506. EDG-5506-203 MESA will provide continued access to EDG-5506 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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

200 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Anticipated Study Start Date :

Oct 1, 2023

Anticipated Primary Completion Date :

Mar 1, 2028

Anticipated Study Completion Date :

Mar 1, 2028

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Treatment Drug: EDG-5506	Drug: EDG-5506 EDG-5506 is administered orally once per day

Arm

Intervention/Treatment

Experimental: Treatment

Drug: EDG-5506

Drug: EDG-5506

EDG-5506 is administered orally once per day

Outcome Measures

Primary Outcome Measures

Number of adverse events in those treated with EDG-5506 [19 Months]
Severity of adverse events in those treated with EDG-5506 [19 Months]

Secondary Outcome Measures

Incidence of treatment-emergent abnormal clinical chemistry laboratory test results [18 Months]
Incidence of treatment-emergent abnormal hematology laboratory test results [18 Months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

Inclusion Criteria:

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:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Rare Disease Research	Atlanta	Georgia	United States	30329

Sponsors and Collaborators

Edgewise Therapeutics, Inc.
Medpace, Inc.

Investigators

Study Chair: Sam Collins, MBBS, PhD, Edgewise Therapeutics, Inc.

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

Sponsor Website

Publications

None provided.

Responsible Party:

Edgewise Therapeutics, Inc.

ClinicalTrials.gov Identifier:

NCT06066580

Other Study ID Numbers:

EDG-5506-203

First Posted:

Oct 4, 2023

Last Update Posted:

Oct 4, 2023

Last Verified:

Sep 1, 2023

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Edgewise Therapeutics, Inc.

Becker Muscular Dystrophy

Additional relevant MeSH terms:

Muscular Dystrophies

Muscular Dystrophy, Duchenne

Study Results

No Results Posted as of Oct 4, 2023