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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.
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 ]
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 ]
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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Recruiting
Sarepta Therapeutics, Inc.