Traitements6programmes
Essais3liés
Publications0liées
SourceDBlocale

Traitements

6
MoléculeIndication / populationPhaseObjectifPaysRésultat
DYNE-251The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension (LTE) period (288 weeks). Myopathies Phase 1/2 À vérifier United States, Australia, Belgium, Canada, Ireland, Italy, South Korea, Spain, … À vérifier
zeleciment basivarsen (DYNE-101)The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1). Myopathies Phase 3 À vérifier United States, Australia, Belgium, Denmark, France, Germany, Italy, Japan, … À vérifier
Zeleciment Rostudirsen (DYNE-251)The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping. Myopathies Phase 3 À vérifier United States À vérifier
DYNE-251The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension (LTE) period (288 weeks). Myopathies Phase 1/2 À vérifier United States, Australia, Belgium, Canada, Ireland, Italy, South Korea, Spain, United Kingdom À vérifier
zeleciment basivarsen (DYNE-101)The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1). Myopathies Phase 3 À vérifier United States, Australia, Belgium, Denmark, France, Germany, Italy, Japan, Netherlands, Spain, United Kingdom À vérifier
Zeleciment Rostudirsen (DYNE-251)The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping. Myopathies Phase 3 À vérifier United States À vérifier

Essais cliniques

3
MoléculeIndication / populationPhaseNCTTitreStatut
Zeleciment Rostudirsen (DYNE-251) Myopathies Phase 3 NCT07608432 Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) RECRUITING
zeleciment basivarsen (DYNE-101) Myopathies Phase 3 NCT07486934 Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 RECRUITING
DYNE-251 Myopathies Phase 1/2 NCT05524883 DELIVER — Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping ACTIVE_NOT_RECRUITING

Publications

0
MoléculeIndication / populationTitreJournalDate
Aucune publication.