scholarly journals Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy

2020 ◽  
Vol 30 (6) ◽  
pp. 492-502 ◽  
Author(s):  
Kathryn R. Wagner ◽  
Hoda Z. Abdel-Hamid ◽  
Jean K. Mah ◽  
Craig Campbell ◽  
Michela Guglieri ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Phase 2 ◽  
Open Label ◽  
Phase 2 Trial ◽  
Open Label Extension ◽  
Randomized Phase 2

scholarly journals Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial

2021 ◽  
pp. 1-13
Author(s):  
Craig M. McDonald ◽  
Perry B. Shieh ◽  
Hoda Z. Abdel-Hamid ◽  
Anne M. Connolly ◽  
Emma Ciafaloni ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
Adverse Events ◽  
Exon Skipping ◽  
Control Group ◽  
Phase 2 ◽  
Open Label ◽  
M Phase ◽  
Positive Treatment

Background Eteplirsen received accelerated FDA approval for treatment of Duchenne muscular dystrophy (DMD) with mutations amenable to exon 51 skipping, based on demonstrated dystrophin production. Objective To report results from PROMOVI, a phase 3, multicenter, open-label study evaluating efficacy and safety of eteplirsen in a larger cohort. Methods Ambulatory patients aged 7–16 years, with confirmed mutations amenable to exon 51 skipping, received eteplirsen 30 mg/kg/week intravenously for 96 weeks. An untreated cohort with DMD not amenable to exon 51 skipping was also enrolled. Results 78/79 eteplirsen-treated patients completed 96 weeks of treatment. 15/30 untreated patients completed the study; this cohort was considered an inappropriate control group because of genotype-driven differences in clinical trajectory. At Week 96, eteplirsen-treated patients showed increased exon skipping (18.7-fold) and dystrophin protein (7-fold) versus baseline. Post-hoc comparisons with patients from eteplirsen phase 2 studies (4658-201/202) and mutation-matched external natural history controls confirmed previous results, suggesting clinically notable attenuation of decline on the 6-minute walk test over 96 weeks (PROMOVI: –68.9 m; phase 2 studies: –67.3 m; external controls: –133.8 m) and significant attenuation of percent predicted forced vital capacity annual decline (PROMOVI: –3.3%, phase 2 studies: –2.2%, external controls: –6.0%; p <  0.001). Adverse events were generally mild to moderate and unrelated to eteplirsen. Most frequent treatment-related adverse events were headache and vomiting; none led to treatment discontinuation. Conclusions This large, multicenter study contributes to the growing body of evidence for eteplirsen, confirming a positive treatment effect, favorable safety profile, and slowing of disease progression versus natural history.

Abstract OT2-04-04: Neoadjuvant HER2-targeted therapy +/- immunotherapy with pembrolizumab (neoHIP): An open label randomized phase 2 trial

2020 ◽  
Author(s):  
Heather McArthur ◽  
Jorge Leal ◽  
David Page ◽  
Christina DiLauro Abaya ◽  
Reva Basho ◽  
...  
Keyword(s):  
Targeted Therapy ◽  
Phase 2 ◽  
Open Label ◽  
Phase 2 Trial ◽  
Randomized Phase 2

scholarly journals Early phase 2 trial of TAS‐205 in patients with Duchenne muscular dystrophy

2020 ◽  
Vol 7 (2) ◽  
pp. 181-190 ◽  
Author(s):  
Hirofumi Komaki ◽  
Yoshihiro Maegaki ◽  
Tsuyoshi Matsumura ◽  
Kazuhiro Shiraishi ◽  
Hiroyuki Awano ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Early Phase ◽  
Phase 2 ◽  
Phase 2 Trial

scholarly journals Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study

PLoS Medicine ◽  
2020 ◽  
Vol 17 (9) ◽  
pp. e1003222 ◽  
Author(s):  
Edward C. Smith ◽  
Laurie S. Conklin ◽  
Eric P. Hoffman ◽  
Paula R. Clemens ◽  
Jean K. Mah ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Interim Analysis ◽  
Extension Study ◽  
Efficacy And Safety ◽  
Open Label ◽  
Open Label Extension

scholarly journals Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

The Lancet ◽  
2011 ◽  
Vol 378 (9791) ◽  
pp. 595-605 ◽  
Author(s):  
Sebahattin Cirak ◽  
Virginia Arechavala-Gomeza ◽  
Michela Guglieri ◽  
Lucy Feng ◽  
Silvia Torelli ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Dose Escalation ◽  
Exon Skipping ◽  
Phase 2 ◽  
Open Label ◽  
Dose Escalation Study ◽  
Open Label Phase
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