A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults

NCT04585464 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: EDG-5506-001
• Study Type: interventional
• Target: 127
• Locations: 1 country
• Sites: 1

Brief Summary

EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers. This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult healthy volunteers and in adults with Becker muscular dystrophy (BMD).

Conditions

Healthy Volunteer; Becker Muscular Dystrophy

Keywords

Healthy Volunteers; Becker Muscular Dystrophy

Outcome Measures

Primary Outcomes (5)

• Incidence, frequency, severity and dose-relationship of adverse events

  Up to 42 days of monitoring

• Incidence of abnormal laboratory test results (clinical chemistry, hematology, urinalysis, coagulation)

  Up to 42 days of monitoring

• Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG)

  Up to 42 days of monitoring

• Incidence of abnormal vital signs

  Up to 42 days of monitoring

• Incidence of abnormal physical exam findings

  Up to 42 days of monitoring

Secondary Outcomes (7)

• Plasma maximum measured drug concentration (Cmax)

  Up to 42 days of testing

• Time of maximum concentration (Tmax)

  Up to 42 days of testing

• Area under the concentration-time curve (AUC)

  Up to 42 days of testing

• Plasma half-life (T½)

  Up to 42 days of testing

• Renal clearance (CLR)

  Up to 42 days of testing

Study Arms (3)

Healthy Volunteer: Single Ascending Dose

EXPERIMENTAL

Single oral ascending dose in healthy volunteers Interventions: Drug: EDG-5506 Drug: Placebo

Drug: EDG-5506; Drug: Placebo

Healthy Volunteer: Multiple Ascending Dose

EXPERIMENTAL

Multiple oral ascending doses in healthy volunteers Interventions: Drug: EDG-5506 Drug: Placebo

Drug: EDG-5506; Drug: Placebo

Becker Muscular Dystrophy: Multiple Ascending Dose

EXPERIMENTAL

Multiple oral ascending doses in adults with Becker muscular dystrophy Interventions: Drug: EDG-5506 Drug: Placebo

Drug: EDG-5506; Drug: Placebo

Interventions

EDG-5506 DRUG

EDG-5506 is administered orally as a single dose or once daily

Becker Muscular Dystrophy: Multiple Ascending Dose; Healthy Volunteer: Multiple Ascending Dose; Healthy Volunteer: Single Ascending Dose

Placebo DRUG

Placebo is administered orally as a single dose or once daily

Becker Muscular Dystrophy: Multiple Ascending Dose; Healthy Volunteer: Multiple Ascending Dose; Healthy Volunteer: Single Ascending Dose

Eligibility Criteria

• Age: 18 Years - 55 Years
• Gender Eligibility Details: Healthy volunteers: Male and non-childbearing potential females. Male Becker muscular dystrophy (BMD) participants
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• For all potential participants (Healthy volunteers and BMD): Male or for HV: female. For all: adults aged 18 to 55 years at time of consent.
• For HVs: Good general health, with no significant medical history, no clinically significant abnormalities on physical exam
• For BMD: Diagnosis of BMD based on documentation of mutation(s) in the dystrophin gene and BMD phenotype
• For BMD: Ability to ambulate
• For all: Weight greater than or equal to 50 kg and BMI less than 33 kg/m2
• For HV: Females must be of non-childbearing potential.
• For all: Males with female partners must use a medically accepted contraceptive regimen from first dose through 90 days after the last dose
• For all: Non-smoker and must not have used any tobacco products within 3 months prior to the Screening visit.
• For all: Able and willing to attend the necessary visits at the study center.

You may not qualify if:

• For all: History of, or physical exam findings indicating clinically significant endocrine, neurological, gastrointestinal, cardiovascular, hematological, hepatic, immunological, renal, respiratory, or genitourinary abnormalities or diseases that, in the opinion of the Investigator, would render the subject being unsuitable for the study.
• For all: Unable to refrain from strenuous exercise for 3 days prior to check-in and during study.
• For all: Participation in any other investigational drug study within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.

Sponsors & Collaborators

• Edgewise Therapeutics, Inc.: lead
• Worldwide Clinical Trials: collaborator

Study Sites (1)

Worldwide Clinical Trials

San Antonio, Texas, 78217, United States

Location

Related Publications (1)

• Donovan J, Silverman JA, Barthel B, DuVall M, Madden M, MacDougall J, Kilburn NR, Bronson A, Evanchik M, Gordon G, Koch K, Russell AJ. A Phase 1, Double-Blind, Placebo-Controlled Trial of Sevasemten (EDG-5506), a Selective Modulator of Fast Skeletal Muscle Contraction, in Healthy Volunteers and Adults With Becker Muscular Dystrophy. Muscle Nerve. 2025 Sep;72(3):399-407. doi: 10.1002/mus.28444. Epub 2025 Jun 2.

  PMID: 40452637 DERIVED

Related Links

• Sponsor Website

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Medical Director

  Edgewise Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 2, 2020
• First Posted: October 14, 2020
• Study Start: October 12, 2020
• Primary Completion: November 15, 2021
• Study Completion: December 27, 2021
• Last Updated: June 28, 2022

Record last verified: 2022-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)