A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
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ClinicalTrials.gov Identifier: NCT04585464 |
Recruitment Status :
Completed
First Posted : October 14, 2020
Last Update Posted : June 28, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Healthy Volunteer Becker Muscular Dystrophy | Drug: EDG-5506 Drug: Placebo | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 127 participants |
Allocation: | Randomized |
Intervention Model: | Sequential Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | October 12, 2020 |
Actual Primary Completion Date : | November 15, 2021 |
Actual Study Completion Date : | December 27, 2021 |

Arm | Intervention/treatment |
---|---|
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 |
- Incidence, frequency, severity and dose-relationship of adverse events [ Time Frame: 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 ]
- Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG) [ Time Frame: Up to 42 days of monitoring ]
- Incidence of abnormal vital signs [ Time Frame: Up to 42 days of monitoring ]
- Incidence of abnormal physical exam findings [ Time Frame: Up to 42 days of monitoring ]
- Plasma maximum measured drug concentration (Cmax) [ Time Frame: Up to 42 days of testing ]
- Time of maximum concentration (Tmax) [ Time Frame: Up to 42 days of testing ]
- Area under the concentration-time curve (AUC) [ Time Frame: Up to 42 days of testing ]
- Plasma half-life (T½) [ Time Frame: Up to 42 days of testing ]
- Renal clearance (CLR) [ Time Frame: Up to 42 days of testing ]
- Drug excreted unchanged in urine (Amt0-24) [ Time Frame: Up to 42 days of testing ]
- Fraction excreted in urine (Fe) [ Time Frame: Up to 42 days of testing ]

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Ages Eligible for Study: | 18 Years to 55 Years (Adult) |
Sexes Eligible for Study: | All |
Gender Based Eligibility: | Yes |
Gender Eligibility Description: | Healthy volunteers: Male and non-childbearing potential females. Male Becker muscular dystrophy (BMD) participants |
Accepts Healthy Volunteers: | Yes |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04585464
United States, Texas | |
Worldwide Clinical Trials | |
San Antonio, Texas, United States, 78217 |
Study Director: | Medical Director | Edgewise Therapeutics, Inc. |
Responsible Party: | Edgewise Therapeutics, Inc. |
ClinicalTrials.gov Identifier: | NCT04585464 |
Other Study ID Numbers: |
EDG-5506-001 |
First Posted: | October 14, 2020 Key Record Dates |
Last Update Posted: | June 28, 2022 |
Last Verified: | June 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Healthy Volunteers Becker Muscular Dystrophy |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |