Trial reportMuscle & nerve2025
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.
Trial report in Muscle & nerve, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04585464 (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), which is not on this map. Cited by 7 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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
Who cites it
7 citing papers in PubMed.
- Trial
- Givinostat enhances antisense oligonucleotide efficacy in theMolecular therapy. Nucleic acids · 2026Article
- Downstream Pathways of Dystrophin Deficiency in Duchenne Muscular Dystrophy: Implications for Muscle Degeneration and Regeneration.Journal of cachexia, sarcopenia and muscle · 2026Review
- Systemic regeneration medicines for muscular dystrophy: progress and challenges in pro-regenerative drug development.Cell regeneration (London, England) · 2026Review
- Effects of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults with Becker muscular dystrophy: results of a phase 1b, open-label study.EBioMedicine · 2026Article
- Human Disposition, Metabolism, and Excretion of Sevasemten (EDG-5506), a Selective Modulator of Fast Myosin in Healthy Volunteers.Journal of clinical pharmacology · 2026Article
- Duchenne Muscular Dystrophy: Contemporary Therapeutic Options and Real-World Challenges in Treatment Selection.Muscles (Basel, Switzerland) · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors.
Funding
Abstract
INTRODUCTION/
aimsSevasemten (EDG-5506) is an orally administered, investigational small molecule that selectively modulates fast muscle fiber contraction by inhibiting fast myosin ATPase. This study assessed the safety, tolerability, and pharmacokinetics (PK)/pharmacodynamics (PD) of sevasemten in healthy adult volunteers (HVs) and adults with Becker muscular dystrophy (BMD).
methodsThis randomized, double-blind, placebo-controlled phase 1 study was conducted at a single site. Eligible participants were 18-55 years of age with a body mass index < 30 kg/m
resultsThe study enrolled 97 HVs in 7 single-dose cohorts (N = 57) and 5 multiple-dose cohorts (N = 40). Seven adults with BMD were enrolled in a multiple-dose cohort. There were no serious AEs or AEs leading to trial discontinuation; the most common AEs were dizziness and somnolence. For adults with BMD, serum biomarkers of muscle injury trended down progressively with sevasemten treatment (average maximal reductions of 70% for creatine kinase, 98% for fast skeletal muscle troponin I, and 45% for myoglobin). Plasma proteomic analysis identified a signature of 125 elevated proteins characteristic of BMD that was reversibly lowered with sevasemten treatment. DISCUSSION: Sevasemten was generally well tolerated. Preliminary observations of decreases in biomarkers of muscle damage in adults with BMD support further clinical development.
trial registrationNCT04585464.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.