Sarepta 51 (Young)

Study of Eteplirsen in Young Patients with DMD Amenable to Exon 51 Skipping

Hub Summary

This phase 2 open-label study is designed to determine the safety and efficacy of eteplirsen in young patients with DMD. Etepliresen (EXONDYS 51®) is an exon-skipping drug designed to treat patients with DMD amenable to exon 51 skipping. This study will enroll young males between the ages of 6 months and 4 years.

Study Number: NCT03218995

Description by Sarepta Therapeutics, Inc.

This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, PK, and efficacy of once-weekly IV infusions of eteplirsen in approximately 12 male patients, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD with a deletion mutation amenable to exon 51 skipping.

 
Primary Outcome Measures

  • Incidence of adverse events [ Time Frame: Up to 96 Weeks ]
  • Abnormal changes from baseline or clinically significant worsening of clinical safety laboratory abnormalities (hematology, chemistry, coagulation, and urinalysis) [ Time Frame: Change from Baseline ]
  • Abnormal changes from baseline or worsening of vital signs [ Time Frame: Change from Baseline ]
  • Abnormal changes from baseline or worsening of physical examination findings [ Time Frame: Change from Baseline ]
  • Abnormal changes from baseline or clinically significant worsening of electrocardiogram (ECG) and echocardiogram (ECHO) [ Time Frame: Change from Baseline ]

 
Secondary Outcome Measures

  • Maximum plasma concentration [ Time Frame: 24 Weeks ]
  • Time of Cmax (Tmax) [ Time Frame: 24 Weeks ]
  • Area under the concentration-time curve (AUC) [ Time Frame: 24 Weeks ]
  • Apparent volume of distribution at steady state (Vss) [ Time Frame: 24 Weeks ]
  • Clearance (CL) [ Time Frame: 24 Weeks ]
  • Elimination half-life (t½) [ Time Frame: 24 Weeks ]
  • Amount of drug eliminated in urine (Ae%) [ Time Frame: 24 Weeks ]

 
Can I take part?

 
Inclusion Criteria

  • Male between 6 months to 48 months of age (inclusive): 
    • Cohort 1: Age 24-48 months (enrolment closed)
    • Cohort 2: Age 6-24 months (currently enrolling)
  • Diagnosis of DMD with a deletion mutation amenable to exon 51 skipping
  • Parent(s) or legal guardian(s) who is willing to provide written informed consent

 
Exclusion Criteria

  • Received treatment that might have an effect on muscle strength or function within 12 weeks prior to dosing
  • Received previous or current treatment with any experimental treatment
  • Clinically significant illness other than DMD
  • Clinically significant laboratory abnormality
  • Any other condition that could interfere with the patient's participation
 
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Trial Status
Recruiting
UK Locations
Trial Sponsor
Sarepta Therapeutics, Inc.
Age
6 months to 4 years
Mutation Specific
Mutation specific therapies, Must be amenable to exon 51 skipping
Muscle Biopsy
No Muscle Biopsy Required
MRI
No
Phase
2
Length Of Participation
12 weeks
Therapeutic Category
Exon-skipping