Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)



Sponsor:   Sarepta Therapeutics, Inc.


Sommario: The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and
SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame
deletion mutations amenable to skipping exon 45 and exon 53, respectively.




TIPOLOGIA STUDIO

 
Interventistico



FASE

 
Fase 3




TRATTAMENTO

  
    
  
  • SRP-4045
  • SRP-4053
  • Placebo
    • 






OBIETTIVO PRIMARIO


  
  • Misura: Change From Baseline in the 4-Step Ascend Velocity at Week 96
  • Tempo: Baseline, Week 96
  • Descrizione: 




OBIETTIVO SECONDARIO


  
  • Misura: Change from Baseline in the Total Distance Walked During 6MWT at Week 96
  • Tempo: Baseline, Week 96
  • Descrizione: 
  • Misura: Change from Baseline in Rise from Floor Velocity at Week 96
  • Tempo: Baseline, Week 96
  • Descrizione: 
  • Misura: Change From Baseline in the 4-Step Ascend Velocity at Week 144
  • Tempo: Baseline, Week 144
  • Descrizione: 
  • Misura: Change From Baseline in Total Distance Walked During the 10-meter walk/run (10-MWR) Velocity
  • Tempo: Baseline, Week 96
  • Descrizione: 
  • Misura: Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 96
  • Tempo: Baseline, Week 96
  • Descrizione: The NSAA is a clinician administered scale that rates the participant's performance on
various functional activities. During this assessment, participants will be asked to
perform 17 different functional activities, including a 10 meter walk/run, rising from a
sit to standing, standing on 1 leg, climbing a box step, descending a box step, rising
from lying to sitting, rising from the floor, lifting the head, standing on heels, and
jumping. Participants will be graded as follows: 2 = achieves goal without any
assistance; 1 = modified method but achieves goal independent of physical assistance from
another person; and 0 = unable to achieve goal independently. NSAA Total Score ranges
from 0 to 34, with a score of 34 implying normal function.
  • Misura: Change from Baseline in Dystrophin Protein Levels Determined by Western Blot at Weeks 48 or 96
  • Tempo: Baseline, Week 48 or Week 96
  • Descrizione: 
  • Misura: Change from Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Weeks 48 or 96
  • Tempo: Baseline, Week 48 or Week 96
  • Descrizione: 




CRITERI DI ELIGIBILITA'


    
             
  • 
        Criteri di inclusione e esclusione
        
    Inclusion Criteria:

  -  Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53
     skipping

  -  Stable dose of oral corticosteroids for at least 24 weeks prior to Week 1, and the
     dose is expected to remain constant throughout the study (except for modifications
     to accommodate changes in weight).

  -  Intact right and left biceps brachii muscles or 2 alternative upper arm muscle
     groups

  -  Mean 6MWT ≥300 meters and ≤450 meters

  -  Stable pulmonary function: forced vital capacity (FVC) ≥50% predicted

Exclusion Criteria:

  -  Treatment with gene therapy at any time

  -  Previous treatment with SMT C1100 within 1 week prior to Week 1 and previous
     treatment with PRO045 (BMN 045), PRO053 (BMN 053), or PRO051 (BMN 051) within 24
     weeks prior to Week 1

  -  Current or previous treatment with any other experimental treatment within 12 weeks
     prior to Week 1

  -  Major surgery within 3 months prior to Week 1

  -  Presence of other clinically significant illness

  -  Other inclusion/exclusion criteria may apply.
    
        
    • 
    




SESSO

  
Maschi




  


ETA' MINIMA



Età Minima: 6 Years

Età Massima: 13 Years






LUOGO


  
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
Ferrara, 44124
Istituto Giannina Gaslini
Genoa, 16147
Az Ospedaliera Universitaria Policlinico G Martino
Messina, 98125
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milano, 20133
Policlinico Universitario A Gemelli
Rome, 00168