Back to resultsA Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
Primary Purpose
Healthy Volunteer, Becker Muscular Dystrophy
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
EDG-5506
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Healthy Volunteer focused on measuring Healthy Volunteers, Becker Muscular Dystrophy
Eligibility Criteria
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.
Sites / Locations
- Worldwide Clinical Trials
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
Healthy Volunteer: Single Ascending Dose
Healthy Volunteer: Multiple Ascending Dose
Becker Muscular Dystrophy: Multiple Ascending Dose
Arm Description
Single oral ascending dose in healthy volunteers Interventions: Drug: EDG-5506 Drug: Placebo
Multiple oral ascending doses in healthy volunteers Interventions: Drug: EDG-5506 Drug: Placebo
Multiple oral ascending doses in adults with Becker muscular dystrophy Interventions: Drug: EDG-5506 Drug: Placebo
Outcomes
Primary Outcome Measures
Incidence, frequency, severity and dose-relationship of adverse events
Incidence of abnormal laboratory test results (clinical chemistry, hematology, urinalysis, coagulation)
Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG)
Incidence of abnormal vital signs
Incidence of abnormal physical exam findings
Secondary Outcome Measures
Plasma maximum measured drug concentration (Cmax)
Time of maximum concentration (Tmax)
Area under the concentration-time curve (AUC)
Plasma half-life (T½)
Renal clearance (CLR)
Drug excreted unchanged in urine (Amt0-24)
Fraction excreted in urine (Fe)
Full Information
NCT ID
NCT04585464
First Posted
October 2, 2020
Last Updated
June 27, 2022
Sponsor
Edgewise Therapeutics, Inc.
Collaborators
Worldwide Clinical Trials
1. Study Identification
Unique Protocol Identification Number
NCT04585464
Brief Title
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
Official 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
Study Type
Interventional
2. Study Status
Record Verification Date
June 2022
Overall Recruitment Status
Completed
Study Start Date
October 12, 2020 (Actual)
Primary Completion Date
November 15, 2021 (Actual)
Study Completion Date
December 27, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Edgewise Therapeutics, Inc.
Collaborators
Worldwide Clinical Trials
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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).
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Healthy Volunteer, Becker Muscular Dystrophy
Keywords
Healthy Volunteers, Becker Muscular Dystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
127 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Healthy Volunteer: Single Ascending Dose
Arm Type
Experimental
Arm Description
Single oral ascending dose in healthy volunteers
Interventions:
Drug: EDG-5506 Drug: Placebo
Arm Title
Healthy Volunteer: Multiple Ascending Dose
Arm Type
Experimental
Arm Description
Multiple oral ascending doses in healthy volunteers
Interventions:
Drug: EDG-5506 Drug: Placebo
Arm Title
Becker Muscular Dystrophy: Multiple Ascending Dose
Arm Type
Experimental
Arm Description
Multiple oral ascending doses in adults with Becker muscular dystrophy
Interventions:
Drug: EDG-5506 Drug: Placebo
Intervention Type
Drug
Intervention Name(s)
EDG-5506
Intervention Description
EDG-5506 is administered orally as a single dose or once daily
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo is administered orally as a single dose or once daily
Primary Outcome Measure Information:
Title
Incidence, frequency, severity and dose-relationship of adverse events
Time Frame
Up to 42 days of monitoring
Title
Incidence of abnormal laboratory test results (clinical chemistry, hematology, urinalysis, coagulation)
Time Frame
Up to 42 days of monitoring
Title
Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG)
Time Frame
Up to 42 days of monitoring
Title
Incidence of abnormal vital signs
Time Frame
Up to 42 days of monitoring
Title
Incidence of abnormal physical exam findings
Time Frame
Up to 42 days of monitoring
Secondary Outcome Measure Information:
Title
Plasma maximum measured drug concentration (Cmax)
Time Frame
Up to 42 days of testing
Title
Time of maximum concentration (Tmax)
Time Frame
Up to 42 days of testing
Title
Area under the concentration-time curve (AUC)
Time Frame
Up to 42 days of testing
Title
Plasma half-life (T½)
Time Frame
Up to 42 days of testing
Title
Renal clearance (CLR)
Time Frame
Up to 42 days of testing
Title
Drug excreted unchanged in urine (Amt0-24)
Time Frame
Up to 42 days of testing
Title
Fraction excreted in urine (Fe)
Time Frame
Up to 42 days of testing
10. Eligibility
Sex
All
Gender Based
Yes
Gender Eligibility Description
Healthy volunteers: Male and non-childbearing potential females. Male Becker muscular dystrophy (BMD) participants
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Edgewise Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Worldwide Clinical Trials
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78217
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Links:
URL
http://www.edgewisetx.com
Description
Sponsor Website
Learn more about this trial
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
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