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Trial record 38 of 87 for:    Recruiting, Not yet recruiting, Available Studies | "Muscular Dystrophies"

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 : November 1, 2017
Sponsor:
Information provided by (Responsible Party):

July 9, 2017
July 17, 2017
November 1, 2017
August 16, 2017
April 2020   (Final data collection date for primary outcome measure)
  • 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 ]
Same as current
Complete list of historical versions of study NCT03218995 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
 
Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping
An Open-label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
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.
Not Provided
Interventional
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Duchenne Muscular Dystrophy
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®
Experimental: Experimental: Treated Group
Intervention: Drug: Eteplirsen
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
12
April 2020
April 2020   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: Male
6 Months to 48 Months   (Child)
No
Contact: Medical Information 617.274.4000 medinfo@sarepta.com
Belgium,   France,   Germany,   Italy,   United Kingdom
 
 
NCT03218995
4658-102
Yes
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
Plan to Share IPD: No
Sarepta Therapeutics
Sarepta Therapeutics
Not Provided
Study Chair: Catherine Stehman-Breen Sarepta Therapeutics
Sarepta Therapeutics
October 2017

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