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WVE-N531

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

Ensayo de Wave Life Sciences, Inc. en Distrofia muscular de Duchenne.

Más ensayos de: Distrofia muscular de Duchenne.

Fase

Fase 1/2

Estado

Reclutando pacientes

Participantes

26

previstos

Centros

5

Fin del objetivo primario

jun 2026

estimado por el promotor

Qué significa cada fase y cada estado.

Estudio de intervención, de un solo grupo, abierto. Comenzó en sep 2021.

Qué mide

Part A: Safety: Proportion of patients with adverse events (AEs) (Day 1 (initial dose) up to 24 weeks after the last dose of Part A) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThis 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.

Más ensayos de WVE

Todos los de WVE, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT04906460), actualizada en dic 2025.