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

key information

study id #: NCT03218995

condition: Duchenne Muscular Dystrophy

status: recruiting

purpose:

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.

intervention: Eteplirsen

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT03218995

last updated: August 01, 2019

study details

start date: August 16, 2017

estimated completion: August 31, 2020

phase of development: Phase 2

size / enrollment: 12

primary outcomes:

  • 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 outcomes:

  • 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]

inclusion criteria:
• 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

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

study contacts

sponsor: Sarepta Therapeutics

contacts: Medical Information, +1-888-727-3782, clinicaltrials@sarepta.com

investigators: Medical Director; Sarepta Therapeutics, Inc.

locations: Belgium, France, Italy, United Kingdom