Healthy VolunteerPhase 1NCT04585464
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
A Phase 1 study testing EDG-5506 in people with Healthy Volunteer and Becker Muscular Dystrophy, run by Edgewise Therapeutics, Inc. at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy Volunteer, Becker Muscular Dystrophy
- What is being tested?
- EDG-5506
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 127 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Oct 2020to 27 Dec 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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.
Show all 9Show fewer
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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).
Read the 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.
- Conditions
- Healthy Volunteer, Becker Muscular Dystrophy
- Being tested
- EDG-5506, Placebo
- Keywords
- Healthy Volunteers, Becker Muscular Dystrophy
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EDG-5506Drug
EDG-5506 is administered orally as a single dose or once daily
PlaceboDrug
Placebo is administered orally as a single dose or once daily
| Study group | Role | What they receive |
|---|---|---|
| Healthy Volunteer: Single Ascending Dose | Experimental | Single oral ascending dose in healthy volunteers
Interventions:
Drug: EDG-5506 Drug: Placebo Receives: Drug: EDG-5506; Drug: Placebo |
| Healthy Volunteer: Multiple Ascending Dose | Experimental | Multiple oral ascending doses in healthy volunteers
Interventions:
Drug: EDG-5506 Drug: Placebo Receives: 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 Receives: Drug: EDG-5506; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence, frequency, severity and dose-relationship of adverse events
Measured: Up to 42 days of monitoring
Incidence of abnormal laboratory test results (clinical chemistry, hematology, urinalysis, coagulation)
Measured: Up to 42 days of monitoring
Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG)
Measured: Up to 42 days of monitoring
Incidence of abnormal vital signs
Measured: Up to 42 days of monitoring
Incidence of abnormal physical exam findings
Measured: Up to 42 days of monitoring
Secondary outcomes (7)
Plasma maximum measured drug concentration (Cmax)
Measured: Up to 42 days of testing
Time of maximum concentration (Tmax)
Measured: Up to 42 days of testing
Area under the concentration-time curve (AUC)
Measured: Up to 42 days of testing
Plasma half-life (T½)
Measured: Up to 42 days of testing
Renal clearance (CLR)
Measured: Up to 42 days of testing
Drug excreted unchanged in urine (Amt0-24)
Measured: Up to 42 days of testing
Fraction excreted in urine (Fe)
Measured: Up to 42 days of testing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2020
- Study start12 Oct 2020
- Primary completion15 Nov 2021
- Study completion27 Dec 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Healthy VolunteerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 127 participants (actual)
Sponsor and investigators
- Lead sponsor
- Edgewise Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Worldwide Clinical Trials
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Edgewise Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04585464
- Sponsor's protocol ID
- EDG-5506-001
- Status last verified
- Jun 2022
- First submitted
- 2 Oct 2020
- Last updated
- 28 Jun 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 40452637
Study at a glance
From the registry record, last updated by the sponsor on 28 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04585464
- Acronym / sponsor ID
- EDG-5506-001
- Conditions
- Healthy Volunteer, Becker Muscular Dystrophy
- Keywords
- Healthy Volunteers, Becker Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 127 participants (actual)
- Lead sponsor
- Edgewise Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Worldwide Clinical Trials
- Study start
- 12 Oct 2020 (actual)
- Primary completion
- 15 Nov 2021 (actual)
- Study completion
- 27 Dec 2021 (actual)
- First posted
- 14 Oct 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Healthy Volunteer: Single Ascending Dose | Experimental | Drug: EDG-5506, Drug: PlaceboSingle oral ascending dose in healthy volunteers Interventions: Drug: EDG-5506 Drug: Placebo |
| Healthy Volunteer: Multiple Ascending Dose | Experimental | Drug: EDG-5506, Drug: PlaceboMultiple oral ascending doses in healthy volunteers Interventions: Drug: EDG-5506 Drug: Placebo |
| Becker Muscular Dystrophy: Multiple Ascending Dose | Experimental | Drug: EDG-5506, Drug: PlaceboMultiple oral ascending doses in adults with Becker muscular dystrophy Interventions: Drug: EDG-5506 Drug: Placebo |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EDG-5506EDG-5506 is administered orally as a single dose or once daily | Drug | Becker Muscular Dystrophy: Multiple Ascending Dose, Healthy Volunteer: Multiple Ascending Dose, Healthy Volunteer: Single Ascending Dose |
| PlaceboPlacebo is administered orally as a single dose or once daily | Drug | Becker Muscular Dystrophy: Multiple Ascending Dose, Healthy Volunteer: Multiple Ascending Dose, Healthy Volunteer: Single Ascending Dose |
Primary outcome
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
7 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jun 2022.
- Derived from this study: PubMed 40452637Donovan 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.
- Sponsor WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Edgewise Therapeutics, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing EDG-5506 in people with Healthy Volunteer and Becker Muscular Dystrophy, run by Edgewise Therapeutics, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence, frequency, severity and dose-relationship of adverse events (time frame: Up to 42 days of monitoring). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04585464, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jun 2022.