Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

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.
 
ClinicalTrials.gov Identifier: NCT02420379
Recruitment Status : Completed
First Posted : April 17, 2015
Last Update Posted : February 26, 2019
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE April 10, 2015
First Posted Date  ICMJE April 17, 2015
Last Update Posted Date February 26, 2019
Actual Study Start Date  ICMJE July 1, 2015
Actual Primary Completion Date December 17, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 14, 2015)
Number of patients with treatment emergent adverse events [ Time Frame: 96 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02420379 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 31, 2017)
Change from baseline in percent of dystrophin-positive skeletal muscle fibers [ Time Frame: 96 weeks ]
Original Secondary Outcome Measures  ICMJE
 (submitted: April 14, 2015)
Change from baseline in percent of dystrophin-positive skeletal muscle fibers [ Time Frame: Baseline to Week 96 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures
 (submitted: April 14, 2015)
Drug concentration in plasma [ Time Frame: 96 weeks ]
 
Descriptive Information
Brief Title  ICMJE Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
Official Title  ICMJE An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy
Brief Summary 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.
Detailed Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy (DMD)
Intervention  ICMJE 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®
Study Arms  ICMJE
  • 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.
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 Completed
Actual Enrollment  ICMJE
 (submitted: February 25, 2019)
33
Original Estimated Enrollment  ICMJE
 (submitted: April 14, 2015)
40
Actual Study Completion Date  ICMJE December 17, 2018
Actual Primary Completion Date December 17, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 4 Years to 6 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02420379
Other Study ID Numbers  ICMJE 4658-203
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Sarepta Therapeutics, Inc.
Study Sponsor  ICMJE Sarepta Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Sarepta Therapeutics, Inc.
PRS Account Sarepta Therapeutics, Inc.
Verification Date February 2019

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