Wave Life Sciences USA, Inc. — Biomedical Watch V2

Traitements4programmes

Essais2liés

Publications1liées

SourceDBlocale

Traitements

4

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
WVE-004 10 mg Q12WDescription non affichée : incohérence de maladie détectée. Consultez l’essai clinique officiel associé.	Sclérose latérale amyotrophique	Phase 1/2	À vérifier	Netherlands, United Kingdom	À vérifier
WVE-N531This 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.	Myopathies	Phase 1/2	À vérifier	United States, Jordan, United Kingdom	À vérifier
WVE-004 10 mg Q12WDescription non affichée : incohérence de maladie détectée. Consultez l’essai clinique officiel associé.	Sclérose latérale amyotrophique	Phase 1/2	À vérifier	Netherlands, United Kingdom	À vérifier
WVE-N531This 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.	Myopathies	Phase 1/2	À vérifier	United States, Jordan, United Kingdom	À vérifier

Essais cliniques

2

Molécule	Indication / population	Phase	NCT	Titre	Statut
WVE-N531	Myopathies	Phase 1/2	NCT04906460	Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)	ACTIVE_NOT_RECRUITING
WVE-004 10 mg Q12W	Sclérose latérale amyotrophique	Phase 1/2	NCT05683860	Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)	TERMINATED

Publications

1

Molécule	Indication / population	Titre	Journal	Date
WVE-N531		Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy.	Pharmaceutics