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

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ClinicalTrials.gov Identifier: NCT03218995
Recruitment Status : Recruiting
First Posted : July 17, 2017
Last Update Posted : July 25, 2019
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE July 9, 2017
First Posted Date  ICMJE July 17, 2017
Last Update Posted Date July 25, 2019
Actual Study Start Date  ICMJE August 16, 2017
Estimated Primary Completion Date March 15, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 12, 2017)
  • 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 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03218995 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: July 12, 2017)
  • 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 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping
Official Title  ICMJE An Open-label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE Drug: Eteplirsen

Eteplirsen will be administered once a week by IV infusion for up to 96 weeks.

The starting dose is 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg over the course of the dose-titration period.

Other Names:
  • AVI-4658
  • EXONDYS 51®
Study Arms  ICMJE Experimental: Experimental: Treated Group
Intervention: Drug: Eteplirsen
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 12, 2017)
12
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 15, 2021
Estimated Primary Completion Date March 15, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 6 Months to 48 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Medical Information +1-888-727-3782 clinicaltrials@sarepta.com
Listed Location Countries  ICMJE Belgium,   France,   Italy,   United Kingdom
Removed Location Countries Germany
 
Administrative Information
NCT Number  ICMJE NCT03218995
Other Study ID Numbers  ICMJE 4658-102
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Sarepta Therapeutics, Inc.
Study Sponsor  ICMJE Sarepta Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: Medical Director Sarepta Therapeutics, Inc.
PRS Account Sarepta Therapeutics, Inc.
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP