Study of SRP-4045 and SRP-4053 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 and SRP-4053 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 Total Distance Walked During 6MWT at Week 96
  • Tempo: Baseline, Week 96
  • Descrizione:

OBIETTIVO SECONDARIO

  • Misura: Change from Baseline in the Total Distance Walked During 6MWT at Week 144 (Week 48 of the Open-Label Extension Period)
  • Tempo: Baseline, Week 144
  • Descrizione:
  • 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:
  • Misura: Participant's Ability to Rise Independently From the Floor, as indicated by a North Star Ambulatory Assessment (NSAA) Subscore
  • Tempo: Week 96, Week 144
  • Descrizione: The NSAA is a clinician administered scale that rates the participant's performance on various functional activities. During this assessment, the participant's ability to rise independently from the floor (without external support) will be reported as an NSAA subscore of "2" (without modification) or "1" (Gower's maneuver).
  • Misura: Time to Loss of Ambulation (LOA)
  • Tempo: Baseline, Week 96, and Week 144
  • Descrizione:
  • Misura: Change From Baseline in the NSAA Total Score at Week 96 and Week 144
  • Tempo: Baseline, Week 96 and Week 144
  • 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 Forced Vital Capacity Percent (FVC%) Predicted at Week 96 and Week 144
  • Tempo: Baseline, Week 96 and Week 144
  • 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 or 2 alternative upper 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

REFERENTE PER INFORMAZIONI

Medical Information
+1 800-690-2003
clinicaltrials@sarepta.com