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NCT Number: NCT04585464

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

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).

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Key information

Conditions

Healthy Volunteer Becker Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

18 year–55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Worldwide Clinical Trials

San Antonio, Texas, 78217, United States

About this study

Enrolled participants in this study will receive a single oral dose or multiple oral doses of EDG-5506 or a placebo. Blood and urine samples will be collected to measure how EDG-5506 is processed by the body and how the body responds when exposed to EDG-5506. Participants in the single ascending dose part of the study will remain in the clinic for 7 days with a 42-day follow-up period. Participants in the multiple ascending dose part of the study will remain in the clinic for 16 days with a 13-day follow-up period. Safety, tolerability, and pharmacokinetics of EDG-5506 will be assessed in healthy volunteers prior to enrolling participants with Becker muscular dystrophy.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

EDG-5506

Drug

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

Placebo

Drug

Placebo is administered orally as a single dose or once daily

Primary outcomes

  1. Incidence, frequency, severity and dose-relationship of adverse events

    Time frame: Up to 42 days of monitoring

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

    Time frame: Up to 42 days of monitoring

  3. Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG)

    Time frame: Up to 42 days of monitoring

  4. Incidence of abnormal vital signs

    Time frame: Up to 42 days of monitoring

  5. Incidence of abnormal physical exam findings

    Time frame: Up to 42 days of monitoring

Secondary outcomes

  1. Plasma maximum measured drug concentration (Cmax)

    Time frame: Up to 42 days of testing

  2. Time of maximum concentration (Tmax)

    Time frame: Up to 42 days of testing

  3. Area under the concentration-time curve (AUC)

    Time frame: Up to 42 days of testing

  4. Plasma half-life (T½)

    Time frame: Up to 42 days of testing

  5. Renal clearance (CLR)

    Time frame: Up to 42 days of testing

  6. Drug excreted unchanged in urine (Amt0-24)

    Time frame: Up to 42 days of testing

  7. Fraction excreted in urine (Fe)

    Time frame: Up to 42 days of testing

Sponsors and collaborators

Lead sponsor

Edgewise Therapeutics, Inc.

Industry

Collaborators

  • Worldwide Clinical Trials

Registry information

Official study title

A Phase 1, Randomized, Placebo-Controlled, Double-blind, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and PK of EDG-5506 in Adult Healthy Volunteers and Adults With Becker Muscular Dystrophy

Important dates

Study start
2020
Primary completion
2021
Study completion
2021
First posted
Oct 14, 2020
Registry last updated
Jun 28, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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