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

June 27, 2022 updated by: Edgewise Therapeutics, Inc.

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

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
127

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Texas
      • San Antonio, Texas, United States, 78217
        • Worldwide Clinical Trials

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 55 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Healthy Volunteer: Single Ascending Dose

Single oral ascending dose in healthy volunteers

Interventions:

Drug: EDG-5506 Drug: Placebo
Drug: EDG-5506
EDG-5506 is administered orally as a single dose or once daily
Drug: Placebo
Placebo is administered orally as a single dose or once daily
Experimental: Healthy Volunteer: Multiple Ascending Dose

Multiple oral ascending doses in healthy volunteers

Interventions:

Drug: EDG-5506 Drug: Placebo
Drug: EDG-5506
EDG-5506 is administered orally as a single dose or once daily
Drug: Placebo
Placebo is administered orally as a single dose or once daily
Experimental: Becker Muscular Dystrophy: Multiple Ascending Dose

Multiple oral ascending doses in adults with Becker muscular dystrophy

Interventions:

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

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Incidence, frequency, severity and dose-relationship of adverse events
Time Frame: Up to 42 days of monitoring
Up to 42 days of monitoring
Incidence of abnormal laboratory test results (clinical chemistry, hematology, urinalysis, coagulation)
Time Frame: Up to 42 days of monitoring
Up to 42 days of monitoring
Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG)
Time Frame: Up to 42 days of monitoring
Up to 42 days of monitoring
Incidence of abnormal vital signs
Time Frame: Up to 42 days of monitoring
Up to 42 days of monitoring
Incidence of abnormal physical exam findings
Time Frame: Up to 42 days of monitoring
Up to 42 days of monitoring

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Plasma maximum measured drug concentration (Cmax)
Time Frame: Up to 42 days of testing
Up to 42 days of testing
Time of maximum concentration (Tmax)
Time Frame: Up to 42 days of testing
Up to 42 days of testing
Area under the concentration-time curve (AUC)
Time Frame: Up to 42 days of testing
Up to 42 days of testing
Plasma half-life (T½)
Time Frame: Up to 42 days of testing
Up to 42 days of testing
Renal clearance (CLR)
Time Frame: Up to 42 days of testing
Up to 42 days of testing
Drug excreted unchanged in urine (Amt0-24)
Time Frame: Up to 42 days of testing
Up to 42 days of testing
Fraction excreted in urine (Fe)
Time Frame: Up to 42 days of testing
Up to 42 days of testing

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Edgewise Therapeutics, Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Worldwide Clinical Trials

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, Edgewise Therapeutics, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 12, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 15, 2021

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 27, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 2, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 7, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 14, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 28, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 27, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • EDG-5506-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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