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Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping
study id #: NCT03218995
condition: Duchenne Muscular Dystrophy
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.
mechanism of action: Exon-skipping to promote dystrophin production
last updated: December 28, 2018
start date: August 16, 2017
estimated completion: August 31, 2020
phase of development: Phase 2
size / enrollment: 12
- 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]
- 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]
• Male between 6 months to 48 months of age (inclusive)
• 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
• 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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