NCT05160415: ARCH

A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy

Completed Phase 1 Results posted Last updated 24 June 2025

What this trial tests

Phase 1 trial testing Sevasemten in Becker Muscular Dystrophy in 12 participants. Completed in 1 March 2024.

Timeline

28 December 2021

Primary endpoint
1 March 2024

1 March 2024

Quick facts

Lead sponsor	Edgewise Therapeutics, Inc.
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	12
Start date	28 December 2021
Primary completion	1 March 2024
Estimated completion	1 March 2024
Sites	1 location across United States

Drugs / interventions tested

• Sevasemten — full drug profile →

Conditions studied

• Becker Muscular Dystrophy — all drugs for Becker Muscular Dystrophy →

Sponsor

Edgewise Therapeutics, Inc. — full company profile →

Who can join

Adults 18 to 55, male only, with Becker Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Percentage of Participants Treated With Sevasemten Experiencing AEs Primary · 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.

Group	Value	95% CI
Treatment	100

Frequency of AEs in Those Treated With Sevasemten Primary · 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.

Group	Value	95% CI
Treatment	95

Number of Participants Treated With Sevasemten With AEs by Maximum Severity Primary · 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

Mild

Group	Value	95% CI
Treatment	10

Moderate

Group	Value	95% CI
Treatment	2

Severe

Group	Value	95% CI
Treatment	0

Percentage of Participants Experiencing Treatment-Emergent Abnormal Clinical Chemistry Test Results Secondary · From first dose of study drug to 25 months

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.

Group	Value	95% CI
Treatment	0

Percentage of Participants Experiencing Treatment-Emergent Abnormal Hematology Test Results Secondary · From first dose of study drug to 25 months

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.

Group	Value	95% CI
Treatment	0

Percentage of Participants Experiencing Treatment-Emergent Abnormal Coagulation Test Results Secondary · From first dose of study drug to 25 months

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.

Group	Value	95% CI
Treatment	0

Percentage of Participants Experiencing Treatment-Emergent Abnormal Urinalysis Test Results Secondary · From first dose of study drug to 25 months

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.

Group	Value	95% CI
Treatment	0

Number of Participants With Clinically Significant Changes in Clinical Chemistry Secondary · From first dose of study drug to 24 months

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.

Group	Value	95% CI
Treatment	0

Number of Participants With Clinically Significant Changes in Hematology Secondary · From first dose of study drug to 24 months

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.

Group	Value	95% CI
Treatment	0

Number of Participants With Clinically Significant Changes in Coagulation Secondary · From first dose of study drug to 24 months

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.

Group	Value	95% CI
Treatment	0

Number of Participants With Clinically Significant Changes in Urinalysis Secondary · From first dose of study drug to 24 months

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.

Group	Value	95% CI
Treatment	0

Number of Participants With Clinically Significant Changes in Vital Signs Secondary · From first dose of study drug to 24 months

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.

Group	Value	95% CI
Treatment	0

Adverse events — posted to ClinicalTrials.gov

Time frame: From first dose of study drug to 25 months.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Treatment

Serious: 0/12 (0%)

Deaths: 0/12

Other adverse events (43 terms — click to expand)

Reaction	System	Treatment
COVID-19	Infections and infestations	—
Fall	Injury, poisoning and procedural complications	—
Dizziness	Nervous system disorders	—
Arthralgia	Musculoskeletal and connective tissue disorders	—
Influenza	Infections and infestations	—
Nasopharyngitis	Infections and infestations	—
Viral upper respiratory tract infection	Infections and infestations	—
Procedural pain	Injury, poisoning and procedural complications	—
Headache	Nervous system disorders	—
Somnolence	Nervous system disorders	—
Gastrooesophageal reflux disease	Gastrointestinal disorders	—
Sinusitis	Infections and infestations	—
Gastroenteritis viral	Infections and infestations	—
Herpes zoster	Infections and infestations	—
Pharyngitis streptococcal	Infections and infestations	—
Arthropod sting	Injury, poisoning and procedural complications	—
Concussion	Injury, poisoning and procedural complications	—
Hand fracture	Injury, poisoning and procedural complications	—
Meniscus injury	Injury, poisoning and procedural complications	—
Post-traumatic pain	Injury, poisoning and procedural complications	—
Road traffic accident	Injury, poisoning and procedural complications	—
Back pain	Musculoskeletal and connective tissue disorders	—
Joint swelling	Musculoskeletal and connective tissue disorders	—
Musculoskeletal chest pain	Musculoskeletal and connective tissue disorders	—
Myalgia	Musculoskeletal and connective tissue disorders	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—
Temporomandibular joint syndrome	Musculoskeletal and connective tissue disorders	—
Diarrhoea	Gastrointestinal disorders	—
Dyspepsia	Gastrointestinal disorders	—
Epiploic appendagitis	Gastrointestinal disorders	—
Toothache	Gastrointestinal disorders	—
Gait inability	General disorders	—
Non-cardiac chest pain	General disorders	—
Euphoric mood	Psychiatric disorders	—
Panic attack	Psychiatric disorders	—
Hyperthyroidism	Endocrine disorders	—
Seasonal allergy	Immune system disorders	—
Dehydration	Metabolism and nutrition disorders	—
Testicle adenoma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—
Hiccups	Respiratory, thoracic and mediastinal disorders	—

Data from ClinicalTrials.gov NCT05160415 adverse events section.

Sponsor's own description

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.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Diffusion-tensor magnetic resonance imaging captures increased skeletal muscle fibre diameters in Becker muscular dystrophy.
   Cameron D, Abbassi-Daloii T, Heezen LGM, van de Velde NM, et al · · 2023 · cited 20× · PMID 37127427 · DOI 10.1002/jcsm.13242
2. An update on Becker muscular dystrophy.
   Straub V, Guglieri M. · · 2023 · cited 17× · PMID 37591308 · DOI 10.1097/wco.0000000000001191
3. Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in <i>bmx</i> model mice.
   McCormack NM, Nguyen NY, Tully CB, Oliver T, et al · · 2023 · cited 12× · PMID 37534133 · DOI 10.1016/j.isci.2023.107161
4. Molecular pathways involved in the control of contractile and metabolic properties of skeletal muscle fibers as potential therapeutic targets for Duchenne muscular dystrophy.
   Bonato A, Raparelli G, Caruso M. · · 2024 · cited 4× · PMID 39717824 · DOI 10.3389/fphys.2024.1496870
5. Lessons for future clinical trials in adults with Becker muscular dystrophy: Disease progression detected by muscle magnetic resonance imaging, clinical and patient-reported outcome measures.
   De Wel B, Iterbeke L, Huysmans L, Peeters R, et al · · 2024 · cited 4× · PMID 38504654 · DOI 10.1111/ene.16282
6. [Molecular therapies: present and future in neuromuscular diseases].
   Ziegler A, Walter MC, Schoser BE. · · 2023 · PMID 37221259 · DOI 10.1007/s00115-023-01495-3