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Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02420379
First Posted: April 17, 2015
Last Update Posted: October 3, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Sarepta Therapeutics
April 10, 2015
April 17, 2015
October 3, 2017
May 2015
December 2017   (Final data collection date for primary outcome measure)
Number of patients with treatment emergent adverse events [ Time Frame: 96 weeks ]
Same as current
Complete list of historical versions of study NCT02420379 on ClinicalTrials.gov Archive Site
Change from baseline in percent of dystrophin-positive skeletal muscle fibers [ Time Frame: 96 weeks ]
Change from baseline in percent of dystrophin-positive skeletal muscle fibers [ Time Frame: Baseline to Week 96 ]
Not Provided
Drug concentration in plasma [ Time Frame: 96 weeks ]
 
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy
This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

Safety, including adverse event monitoring and routine laboratory assessments, will be followed on an ongoing basis for all patients.

Clinical efficacy, including functional tests and MRI, will be assessed at regularly scheduled study visits. Patients will undergo one baseline and one follow-up muscle biopsy.

Population and serial PK will be collected.

Interventional
Phase 2
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Duchenne Muscular Dystrophy (DMD)
Drug: eteplirsen
Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks.
Other Names:
  • AVI-4658
  • EXONDYS 51®
  • Experimental: Open-Label
    Approximately 20 patients will receive weekly infusions of eteplirsen 30 mg/kg .
    Intervention: Drug: eteplirsen
  • No Intervention: Control Group
    Approximately 20 patients with DMD not amenable to exon 51 skipping will be observed for 96 weeks.
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
40
February 2018
December 2017   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Male 4-6 years of age.
  • Diagnosis of DMD, genotypically confirmed.
  • Stable dose of oral corticosteroids for at least 12 weeks or has not received corticosteroids for at least 12 weeks.
  • Intact right and left biceps muscles or two alternative upper arm muscle groups.
  • Parent that is willing to provide consent and comply with study procedures.

Exclusion Criteria:

  • Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids).
  • Previous or current treatment with any other experimental treatments within 12 weeks or participation in any other clinical trial within 6 months.
  • Major surgery within 3 months prior to the first dose of study drug, or planned surgery during this study which would interfere with the ability to perform study activities.
  • Presence of other clinically significant illness.
Sexes Eligible for Study: Male
4 Years to 6 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT02420379
4658-203
No
Not Provided
Not Provided
Sarepta Therapeutics
Sarepta Therapeutics
Not Provided
Study Chair: Chris Mix, MD Sarepta Therapeutics, Inc.
Study Chair: Catherine Stehman-Breen, MD, MS Sarepta Therapeutics
Sarepta Therapeutics
October 2017

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