Becker Muscular DystrophyPhase 1ARCHNCT05160415
A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy
A Phase 1 study testing Sevasemten in people with Becker Muscular Dystrophy, run by Edgewise Therapeutics, Inc. at 1 site in United States.
Trial summary
The ARCH study was an open-label, single-center, Phase 1b study of sevasemtem (EDG-5506) to assess the safety and pharmacokinetics (PK) of sevasemten in adults with Becker muscular dystrophy (BMD). Sevasemten is an investigational product intended to protect and improve function of dystrophic muscle fibers. Read more about this study
- Who is it for?
- Ages 18 to 55 years, men only
- Conditions the study is for
- Becker Muscular Dystrophy
- What is being tested?
- Sevasemten
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Dec 2021to 1 Mar 2024
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- Participants who have completed Study EDG-5506-001.
- Participants who were not from Study EDG-5506-001 must meet the following:
- Male sex at birth and aged 18 to 55 years inclusive at time of consent.
- Documented dystrophin mutation with phenotype consistent with BMD.
- Ambulatory at Screening (defined as ability to complete 100 meter [m] timed test, with or without assistance).
- Body weight ≥ 50 kg at the Screening visit.
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- Body mass index (BMI) between 20 and 34 kg/m2 inclusive.
- Female sexual partners of male participants must use highly effective contraception (<1% failure rate per year) through 6 months after last dose.
- Capable of giving signed informed consent.
Who cannot join
- Any clinically significant changes during or following the completion of Study EDG 5506-001 that would affect the potential safety of the participant to receive EDG 5506.
- Cardiac echocardiogram ejection fraction <45% or New York Heart Association (NYHA) Class III or Class IV.
- Baseline 12-lead electrocardiogram (ECG) that demonstrates clinically relevant abnormalities that may affect participant safety or interpretation of study results.
- Forced vital capacity (FVC) predicted <65% or using daytime (mechanical or noninvasive) ventilatory support.
- Moderate or severe renal or hepatic impairment (eGFR <60 mL/min/1.73 m2).
- Positive test for hepatitis C antibody (unless negative HCV PCR), hepatitis B surface antigen, or human immunodeficiency virus (HIV) antibody at screening.
Show all 12Show fewer
- History of substance abuse or dependency.
- Receipt of oral corticosteroids for >5 days in the previous 6 months at a dose of >5 mg equivalent per day. Lower oral doses or inhaled/intranasal steroids are permitted.
- Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers.
- Participation in any other investigational drug study or use of use of an investigational drug within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.
- Participants who are unlikely to comply with the study protocol or, in the opinion of the Investigator, would not be a suitable candidate for participation in the study.
- Medical history or other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory result or abnormality that may increase the risk of study participation or, in the Investigator's judgment, make the participant inappropriate for the study. Includes venous access that would be too difficult to facilitate repeated blood sampling.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1b, Open-label Study of the Safety and Pharmacokinetics of EDG-5506 in Adults With Becker Muscular Dystrophy
The ARCH study was an open-label, single-center, Phase 1b study of sevasemtem (EDG-5506) to assess the safety and pharmacokinetics (PK) of sevasemten in adults with Becker muscular dystrophy (BMD).
Sevasemten is an investigational product intended to protect and improve function of dystrophic muscle fibers.
Read the detailed description
This open-label study evaluated the safety, tolerability, and pharmacokinetics (PK) of sevasemten in participants with BMD who completed the first-in-human study, EDG-5506-001, as well as additional (treatment-naïve) participants from outside the EDG-5506-001 study to meet the target sample size.
All participants received sevasemten. This study had a 24 month treatment period, followed by an optional 4 week follow-up period. On-site visits occurred approximately monthly for the first 12 months, followed by every 3 months to assess safety and measures of function.
- Conditions
- Becker Muscular Dystrophy
- Being tested
- Sevasemten
- Keywords
- 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.
SevasemtenDrug
Also called EDG-5506
Daily oral dose of 10 mg daily until Visit 8 (Day 57), followed by 15 mg daily until Visit 13 (Month 6), followed by 20 mg until Visit 21 (Month 15), followed by 10 mg daily to Visit 27 (Month 24).
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Drug: Sevasemten Receives: Drug: Sevasemten |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Treated With Sevasemten Experiencing AEs
An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.
The numerator of the percentage is the number of participants experiencing at least one AE after first dose of study drug up to 25 months.
Measured: From first dose of study drug to 25 months
Frequency of AEs in Those Treated With Sevasemten
An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.
The endpoint is the cumulative total number of AEs occurring after first dose of study drug up to 25 months among participants who received at least one dose of study drug.
Measured: From first dose of study drug to 25 months
Number of Participants Treated With Sevasemten With AEs by Maximum Severity
An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.
The severity of an AE is graded, according to the study protocol definitions of AE severity/intensity, as "mild", "moderate" or "severe". Participants who reported multiple AEs are counted only once at the highest severity reported.
Measured: From first dose of study drug to 25 months
Secondary outcomes (19)
Percentage of Participants Experiencing Treatment-Emergent Abnormal Clinical Chemistry Test Results
Treatment-emergent defined as any event that occurs after the start of study drug or was present at baseline and worsened after taking study drug.
The numerator of the percentage is the number of participants experiencing at least one treatment-emergent abnormal clinical chemistry test result after first dose of study drug up to 25 months.
Measured: From first dose of study drug to 25 months
Percentage of Participants Experiencing Treatment-Emergent Abnormal Hematology Test Results
Treatment-emergent defined as any event that occurs after the start of study drug or was present at baseline and worsened after taking study drug.
The numerator of the percentage is the number of participants experiencing at least one treatment-emergent abnormal hematology test result after first dose of study drug up to 25 months.
Measured: From first dose of study drug to 25 months
Percentage of Participants Experiencing Treatment-Emergent Abnormal Coagulation Test Results
Treatment-emergent defined as any event that occurs after the start of study drug or was present at baseline and worsened after taking study drug.
The numerator of the percentage is the number of participants experiencing at least one treatment-emergent abnormal coagulation test result after first dose of study drug up to 25 months.
Measured: From first dose of study drug to 25 months
Percentage of Participants Experiencing Treatment-Emergent Abnormal Urinalysis Test Results
Treatment-emergent defined as any event that occurs after the start of study drug or was present at baseline and worsened after taking study drug.
The numerator of the percentage is the number of participants experiencing at least one treatment-emergent abnormal urinalysis test result after first dose of study drug up to 25 months.
Measured: From first dose of study drug to 25 months
Number of Participants With Clinically Significant Changes in Clinical Chemistry
Refer to Protocol for list of clinical chemistry tests that were performed. Clinically significant changes in clinical chemistry are defined as adverse events related to clinical chemistry tests or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted.
Measured: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Hematology
Refer to Protocol for list of hematology tests that were performed. Clinically significant changes in hematology are defined as adverse events related to hematology tests or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted.
Measured: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Coagulation
Refer to Protocol for list of coagulation tests that were performed. Clinically significant changes in coagulation are defined as adverse events related to coagulation tests or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted.
Measured: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Urinalysis
Refer to Protocol for list of urinalysis tests that were performed. Clinically significant changes in urinalysis are defined as adverse events related to urinalysis tests or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted.
Measured: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Vital Signs
Supine systolic and diastolic blood pressure, pulse rate, respiratory rate, and temperature were measured. Clinically significant changes in vital signs are defined as adverse events related to vital signs or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted.
Measured: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Physical and Neurological Examinations
A physical examination included head, ears, eyes, nose, mouth, skin, heart and lung, lymph nodes, and gastrointestinal and musculoskeletal systems. A neurological examination was also conducted to include upper and lower limb tone, power, reflexes, and examination of cranial nerves II-XII (excluding ophthalmoscopy). Clinically significant changes in physical and neurological examinations are defined as adverse events related to physical and neurological examinations or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted.
Measured: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in ECG PR Interval
Triplicate 12-lead electrocardiogram (ECG) parameters were obtained using an ECG machine that automatically measured PR interval. Clinically significant changes in ECG PR interval are defined as adverse events related to ECG PR interval or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted.
Measured: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in ECG QRS Interval
Triplicate 12-lead electrocardiogram (ECG) parameters were obtained using an ECG machine that automatically measured QRS interval. Clinically significant changes in ECG QRS interval are defined as adverse events related to ECG QRS interval or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted.
Measured: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in ECG QT Interval
Triplicate 12-lead electrocardiogram (ECG) parameters were obtained using an ECG machine that automatically measured QT interval. Clinically significant changes in ECG QT interval are defined as adverse events related to ECG QT interval or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted.
Measured: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in ECG QTc Interval
Triplicate 12-lead electrocardiogram (ECG) parameters were obtained using an ECG machine that automatically measured QT corrected (QTc) Interval. QT corrected by Bazett's formula (QTcB) and QT corrected by Fridericia's formula (QTcF) were both recorded. Clinically significant changes in ECG QTc interval are defined as adverse events related to ECG QTc interval or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted.
Measured: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Forced Vital Capacity (FVC)
Assessed by spirometry. Clinically significant changes in FVC are defined as adverse events related to FVC or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted.
Measured: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Forced Expiratory Volume in 1 Second (FEV1)
Assessed by spirometry. Clinically significant changes in FEV1 are defined as adverse events related to FEV1 or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted.
Measured: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Cardiac Function as Assessed by Echocardiogram
Echocardiographic examinations were performed by a qualified individual (sonographer) at the site to evaluate left-ventricular systolic and diastolic function, geometry, and mass, as well as left-atrial and right-ventricular function and geometry via two-dimensional, doppler, and/or speckle-tracking imaging techniques. Valvular competence, including presence or absence of regurgitation, was evaluated and quantified, while overall cardiac health was qualitatively evaluated (e.g., presence/absence of pericardiac effusion).
Clinically significant changes in cardiac function are defined as adverse events related to cardiac function as assessed by echocardiogram or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted.
Measured: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Columbia Suicide Severity Rating Scale (C-SSRS)
The C-SSRS is a questionnaire used for suicide assessment. The assessment includes "yes" or "no" responses for 6 questions. Questions 1-5 are related to suicidal ideation, including: 1=Wish to be Dead, 2=Non-specific Active Suicidal Thoughts, 3=Active Suicidal Ideation with Any Methods (Not Plan) without Intent to Act, 4=Active Suicidal Ideation with Some Intent to Act, without Specific Plan, 5=Active Suicidal Ideation with Specific Plan and Intent. Question 6 is related to suicidal behavior and asks about actual attempts. Numeric ratings were provided for severity of ideation (if present), from 1 to 5, with 5 being the most severe. For this study, clinically significant changes in C-SSRS are defined as responses of "yes" to suicidal ideation or suicidal behavior item as measured by C-SSRS or investigator identified results reported from first dose of study drug to 24 months.
Measured: From first dose of study drug to 24 months
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
Values below the limit of quantitation (BLQ) of 0.500 ng/mL were treated as 0 before the first quantifiable concentration and as missing elsewhere.
Single PK concentration was collected at each visit, except for Day 1. For treatment-naive participants, Day 1 was a serial collection at pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, and 24 hours post-dose. The last timepoint was on Day 2. The dose on Day 2 was administered only after the 24 hour timepoint. For treatment experienced participants, Day 1 was pre-dose only.
Days 29 and 57 have window of +/- 3 days; Months 3-24 have window of +/- 5 days.
Measured: Pre-dose; 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, and 24 hours post-dose; 29 and 57 days post-dose; 3, 4, 6, 7, 8, 10, 12, 15, 18, 21, and 24 months post-dose.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2021
- Study start28 Dec 2021
- Primary completion1 Mar 2024
- Study completion1 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Becker Muscular DystrophyStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Edgewise Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Medpace, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05160415
- Sponsor's protocol ID
- EDG-5506-002
- Acronym
- ARCH
- Status last verified
- Jun 2025
- First submitted
- 2 Dec 2021
- Last updated
- 24 Jun 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Open-label Study of the Safety and Pharmacokinetics of EDG-5506 in Adults With Becker Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT05160415
- Acronym / sponsor ID
- ARCH / EDG-5506-002
- Conditions
- Becker Muscular Dystrophy
- Keywords
- Becker Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Edgewise Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Medpace, Inc.
- Study start
- 28 Dec 2021 (actual)
- Primary completion
- 1 Mar 2024 (actual)
- Study completion
- 1 Mar 2024 (actual)
- First posted
- 16 Dec 2021 (actual)
- Results
- Posted 24 Jun 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The ARCH study was an open-label, single-center, Phase 1b study of sevasemtem (EDG-5506) to assess the safety and pharmacokinetics (PK) of sevasemten in adults with Becker muscular dystrophy (BMD).
Sevasemten is an investigational product intended to protect and improve function of dystrophic muscle fibers.
Detailed description
This open-label study evaluated the safety, tolerability, and pharmacokinetics (PK) of sevasemten in participants with BMD who completed the first-in-human study, EDG-5506-001, as well as additional (treatment-naïve) participants from outside the EDG-5506-001 study to meet the target sample size.
All participants received sevasemten. This study had a 24 month treatment period, followed by an optional 4 week follow-up period. On-site visits occurred approximately monthly for the first 12 months, followed by every 3 months to assess safety and measures of function.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: SevasemtenDrug: Sevasemten |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- Participants who have completed Study EDG-5506-001.
- Participants who were not from Study EDG-5506-001 must meet the following:
- Female sexual partners of male participants must use highly effective contraception (<1% failure rate per year) through 6 months after last dose.
- Capable of giving signed informed consent.
Key exclusion criteria (12 listed)
- Any clinically significant changes during or following the completion of Study EDG 5506-001 that would affect the potential safety of the participant to receive EDG 5506.
- Cardiac echocardiogram ejection fraction <45% or New York Heart Association (NYHA) Class III or Class IV.
- Baseline 12-lead electrocardiogram (ECG) that demonstrates clinically relevant abnormalities that may affect participant safety or interpretation of study results.
- Forced vital capacity (FVC) predicted <65% or using daytime (mechanical or noninvasive) ventilatory support.
- Moderate or severe renal or hepatic impairment (eGFR <60 mL/min/1.73 m2).
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 |
|---|---|---|
| SevasemtenAlso: EDG-5506Daily oral dose of 10 mg daily until Visit 8 (Day 57), followed by 15 mg daily until Visit 13 (Month 6), followed by 20 mg until Visit 21 (Month 15), followed by 10 mg daily to Visit 27 (Month 24). | Drug | Treatment |
Primary outcome
Percentage of Participants Treated With Sevasemten Experiencing AEs
Time frame: From first dose of study drug to 25 months
An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The numerator of the percentage is the number of participants experiencing at least one AE after first dose of study drug up to 25 months.
Frequency of AEs in Those Treated With Sevasemten
Time frame: From first dose of study drug to 25 months
An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The endpoint is the cumulative total number of AEs occurring after first dose of study drug up to 25 months among participants who received at least one dose of study drug.
Number of Participants Treated With Sevasemten With AEs by Maximum Severity
Time frame: From first dose of study drug to 25 months
An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The severity of an AE is graded, according to the study protocol definitions of AE severity/intensity, as "mild", "moderate" or "severe". Participants who reported multiple AEs are counted only once at the highest severity reported.
19 secondary outcomes
Percentage of Participants Experiencing Treatment-Emergent Abnormal Clinical Chemistry Test Results
Time frame: From first dose of study drug to 25 months
Percentage of Participants Experiencing Treatment-Emergent Abnormal Hematology Test Results
Time frame: From first dose of study drug to 25 months
Percentage of Participants Experiencing Treatment-Emergent Abnormal Coagulation Test Results
Time frame: From first dose of study drug to 25 months
Percentage of Participants Experiencing Treatment-Emergent Abnormal Urinalysis Test Results
Time frame: From first dose of study drug to 25 months
Number of Participants With Clinically Significant Changes in Clinical Chemistry
Time frame: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Hematology
Time frame: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Coagulation
Time frame: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Urinalysis
Time frame: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Vital Signs
Time frame: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Physical and Neurological Examinations
Time frame: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in ECG PR Interval
Time frame: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in ECG QRS Interval
Time frame: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in ECG QT Interval
Time frame: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in ECG QTc Interval
Time frame: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Forced Vital Capacity (FVC)
Time frame: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Forced Expiratory Volume in 1 Second (FEV1)
Time frame: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Cardiac Function as Assessed by Echocardiogram
Time frame: From first dose of study drug to 24 months
Number of Participants With Clinically Significant Changes in Columbia Suicide Severity Rating Scale (C-SSRS)
Time frame: From first dose of study drug to 24 months
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
Time frame: Pre-dose; 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, and 24 hours post-dose; 29 and 57 days post-dose; 3, 4, 6, 7, 8, 10, 12, 15, 18, 21, and 24 months post-dose.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
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 Sevasemten in people with 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 12 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 24 Jun 2025. 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?
Yes. Results are posted in the registry (first posted 24 Jun 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Participants Treated With Sevasemten Experiencing AEs (time frame: From first dose of study drug to 25 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 24 Jun 2025.