A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy

Becker Muscular Dystrophy Clinical Trial

— ARCH  

Official title:

A Phase 1b, Open-label Study of the Safety and Pharmacokinetics of EDG-5506 in Adults With Becker Muscular Dystrophy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05160415
• Other study ID #: EDG-5506-002
• Status: Completed
• Phase: Phase 1
• Start date: December 28, 2021
• Est. completion date: March 1, 2024

Study information

• Verified date: June 2024
• Source: Edgewise Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The ARCH study is an open-label, single-center, Phase 1b study of sevasemtem (EDG-5506) to assess the safety and pharmacokinetics (PK) of sevasemten in adults with Becker muscular dystrophy (BMD).

Sevasemten is an investigational product intended to protect and improve function of dystrophic muscle fibers.

Description:

This open-label study will evaluate the safety, tolerability, and pharmacokinetics (PK) of sevasemten in participants with BMD who completed the first-in-human study, EDG-5506-001, as well as additional (treatment-naïve) participants from outside the EDG-5506-001 study to meet the target sample size.

All participants will receive sevasemten. On-site visits will occur approximately monthly for the first 12 months, followed by every 3 months to assess safety and measures of function. This study will have a 24 month treatment period, followed by a 4 week follow-up period.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: March 1, 2024
• Est. primary completion date: March 1, 2024
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 55 Years

Eligibility

Inclusion Criteria:

1. Participants who have completed Study EDG-5506-001.

2. Participants who were not from Study EDG-5506-001 must meet the following:

1. Male sex at birth and aged 18 to 55 years inclusive at time of consent.

2. Documented dystrophin mutation with phenotype consistent with BMD.

3. Ambulatory at Screening (defined as ability to complete 100 meter [m] timed test, with or without assistance).

4. Body weight = 50 kg at the Screening visit.

5. Body mass index (BMI) between 20 and 34 kg/m2 inclusive.

Exclusion Criteria:

1. Receipt of oral corticosteroids for >5 days in the previous 6 months at a dose of >5 mg equivalent per day. Lower oral doses or inhaled/intranasal steroids are permitted.

2. Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers.

3. Participation in any other investigational drug study or use of use of an investigational drug within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.

4. Medical history or other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory result or abnormality that may increase the risk of study participation or, in the Investigator's judgment, make the participant inappropriate for the study. Includes venous access that would be too difficult to facilitate repeated blood sampling.

Related Conditions & MeSH terms

• Becker Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• Sevasemten
Daily oral dose

Locations

Country	Name	City	State
United States	Rare Disease Research	Atlanta	Georgia

Sponsors (2)

Lead Sponsor	Collaborator
Edgewise Therapeutics, Inc.	Medpace, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Number of participants with changes in biomarkers of muscle fiber damage		24 Months
Primary	Incidence of AEs in those treated with sevasemten		25 Months
Primary	Frequency of AEs in those treated with sevasemten		25 Months
Primary	Severity of AEs in those treated with sevasemten		25 Months
Secondary	Incidence of treatment-emergent abnormal clinical chemistry test results		24 Months
Secondary	Incidence of treatment-emergent abnormal hematology test results		24 Months
Secondary	Incidence of treatment-emergent abnormal coagulation test results		24 Months
Secondary	Incidence of treatment-emergent abnormal urinalysis test results		24 Months
Secondary	Number of participants with changes in clinical chemistry		24 Months
Secondary	Number of participants with changes in hematology		24 Months
Secondary	Number of participants with changes in coagulation		24 Months
Secondary	Number of participants with changes in urinalysis		24 Months
Secondary	Number of participants with changes in vital signs		24 Months
Secondary	Number of participants with changes in physical examination		24 Months
Secondary	Number of participants with changes in ECG PR Interval		24 Months
Secondary	Number of participants with changes in ECG QRS Interval		24 Months
Secondary	Number of participants with changes in ECG QT Interval		24 Months
Secondary	Number of participants with changes in ECG QTc Interval		24 Months
Secondary	Number of participants with changes in FVC	Assessed by spirometry	24 Months
Secondary	Number of participants with changes in FEV1	As assessed by spirometry	24 Months

External Links

•   Sponsor Website