An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy



Sponsor:   Sarepta Therapeutics, Inc.


Sommario: The main objective of this study is to evaluate the safety and tolerability of long-term
treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy
(DMD).




TIPOLOGIA STUDIO

 
Interventistico



FASE

 
Fase 3




TRATTAMENTO

  
    
  
  • Casimersen
  • Golodirsen
    • 






OBIETTIVO PRIMARIO


  
  • Misura: Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
  • Tempo: Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
  • Descrizione: A treatment-emergent adverse event (TEAE) was any untoward medical occurrence in a
clinical study participant that did not necessarily have a causal relationship with the
study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign,
disease, condition, or test abnormality that occurred during or after administration of
the study drug, whether or not considered related to the study drug. A TESAE was any TEAE
that resulted in any of the following outcomes: death, a life-threatening event, required
or prolonged inpatient hospitalization, persistent or significant disability/incapacity,
or an important medical event (that is, may have jeopardized the participant and may have
required medical or surgical intervention to prevent one of the previously mentioned
outcomes). A summary of serious and all other non-serious TEAEs regardless of causality
is located in the Reported Adverse Events module.






CRITERI DI ELIGIBILITA'


    
             
  • 
        Criteri di inclusione e esclusione
        
    Inclusion Criteria:

  -  Completed a clinical trial evaluating casimersen or golodirsen, per protocol.

  -  Is between 7 and 23 years of age, inclusive, at enrollment.

Other inclusion/exclusion criteria apply.
    
        
    • 
    




SESSO

  
Maschi




  


ETA' MINIMA



Età Minima: 7 Years

Età Massima: 23 Years






LUOGO


  
UOSD Centro Traslazionale di Miologia e Patologie Neurogenerative
Genova, 16147
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
Cona, 44124
Fondazione Policlinico Universitario A Gemelli
Milano, 20123