NCT04906460

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

Recruiting now Phase 1, PHASE2 Last updated 15 December 2025

What this trial tests

Phase 1, PHASE2 trial testing WVE-N531 in Duchenne Muscular Dystrophy in 26 participants. Currently enrolling.

Timeline

28 September 2021

Primary endpoint
27 June 2026

24 April 2027

Quick facts

Lead sponsor	Wave Life Sciences Ltd.
Phase	Phase 1, PHASE2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	26
Start date	28 September 2021
Primary completion	27 June 2026
Estimated completion	24 April 2027
Sites	5 locations across Jordan, United Kingdom, United States

Drugs / interventions tested

• WVE-N531 — full drug profile →

Conditions studied

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

Sponsor

Wave Life Sciences Ltd. — full company profile →

Who can join

Adults 4 to 18, male only, with Duchenne Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD). To participate in the study, patients must have a documented mutation of the DMD gene that is amenable to exon 53 skipping intervention. This study has 3 parts, Part A, Part B, including Part B Extension Arm, and Part C. Part A is completed. Part B is completed. Following completion of Part B, all patients elected to continue to receive study drug in the optional Part B open-label Extension Arm. Part C has been added to the study and will enroll new patients.

Publications & conference data

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

1. Amplifying gene expression with RNA-targeted therapeutics.
   Khorkova O, Stahl J, Joji A, Volmar CH, et al · · 2023 · cited 78× · PMID 37253858 · DOI 10.1038/s41573-023-00704-7
2. RNA therapeutics in the clinic.
   Curreri A, Sankholkar D, Mitragotri S, Zhao Z. · · 2023 · cited 62× · PMID 36684099 · DOI 10.1002/btm2.10374
3. Antisense Oligonucleotide Therapy for the Nervous System: From Bench to Bedside with Emphasis on Pediatric Neurology.
   Amanat M, Nemeth CL, Fine AS, Leung DG, et al · · 2022 · cited 47× · PMID 36365206 · DOI 10.3390/pharmaceutics14112389
4. Clinical applications of exon-skipping antisense oligonucleotides in neuromuscular diseases.
   Torres-Masjoan L, Aguti S, Zhou H, Muntoni F. · · 2025 · cited 21× · PMID 40308063 · DOI 10.1016/j.ymthe.2025.04.038
5. Nucleic acid therapeutics: Past, present, and future.
   Naeem S, Zhang J, Zhang Y, Wang Y. · · 2025 · cited 21× · PMID 39897578 · DOI 10.1016/j.omtn.2024.102440
6. Comprehensive review of adverse reactions and toxicology in ASO-based therapies for Duchenne Muscular Dystrophy: From FDA-approved drugs to peptide-conjugated ASO.
   Sabrina Haque U, Kohut M, Yokota T. · · 2024 · cited 20× · PMID 38983605 · DOI 10.1016/j.crtox.2024.100182
7. Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpoint.
   Servais L, Strijbos P, Poleur M, Mirea A, et al · · 2024 · cited 12× · PMID 39613806 · DOI 10.1038/s41598-024-80177-9
8. Advanced Gene-Targeting Therapies for Motor Neuron Diseases and Muscular Dystrophies.
   Chamakioti M, Karantzelis N, Taraviras S. · · 2022 · cited 11× · PMID 35563214 · DOI 10.3390/ijms23094824