NCT05540860

A 2-part Phase 2 Study of Safety, Pharmacokinetics and Biomarkers in Children With Duchenne Muscular Dystrophy Including a Randomized, Double-Blind, Placebo-Controlled Part A, Followed by an Open-Label Part B

Active, enrolled Phase 2 Last updated 10 November 2025

What this trial tests

Phase 2 trial testing Sevasemten Dose 1 in Duchenne Muscular Dystrophy in 76 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

24 October 2022

Primary endpoint
1 January 2027

1 January 2027

Quick facts

Lead sponsor	Edgewise Therapeutics, Inc.
Phase	Phase 2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	randomized
Design	sequential
Masking	quadruple
Primary purpose	treatment
Enrollment	76
Start date	24 October 2022
Primary completion	1 January 2027
Estimated completion	1 January 2027
Sites	14 locations across United States

Drugs / interventions tested

• Sevasemten Dose 1 — full drug profile →
• Sevasemten Dose 2
• Sevasemten Dose 3
• Sevasemten Dose 4
• Sevasemten Dose 5
• Placebo

Conditions studied

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

Sponsor

Edgewise Therapeutics, Inc. — full company profile →

Who can join

Adults 4 to 9, male only, with Duchenne Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Number of adverse events during treatment with sevasemten or placebo
  Time frame: 48 months
  All participants
• Severity of adverse events during treatment with sevasemten or placebo
  Time frame: 48 months
  All participants

Sponsor's own description

The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an open-label part B.

Publications & conference data

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

1. Modulating fast skeletal muscle contraction protects skeletal muscle in animal models of Duchenne muscular dystrophy.
   Russell AJ, DuVall M, Barthel B, Qian Y, et al · · 2023 · cited 23× · PMID 36995778 · DOI 10.1172/jci153837
2. Cell type mapping of inflammatory muscle diseases highlights selective myofiber vulnerability in inclusion body myositis.
   Wischnewski S, Thäwel T, Ikenaga C, Kocharyan A, et al · · 2024 · cited 19× · PMID 38834884 · DOI 10.1038/s43587-024-00645-9
3. 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
4. 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.
   Donovan J, Silverman JA, Barthel B, DuVall M, et al · · 2025 · cited 2× · PMID 40452637 · DOI 10.1002/mus.28444
5. A computational tool to optimize clinical trial parameter selection in Duchenne muscular dystrophy: A practical guide and case studies.
   Wilk J, Aggarwal V, Pauley M, Corey D, et al · · 2025 · cited 2× · PMID 39600251 · DOI 10.1002/psp4.13281
6. Duchenne Muscular Dystrophy: Contemporary Therapeutic Options and Real-World Challenges in Treatment Selection.
   Pesco MT, Öztürk GZ, Bhadola SC, Chrzanowski SM, et al · · 2026 · PMID 41892993 · DOI 10.3390/muscles5010021