Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics
ClinicalTrials.gov Identifier:
NCT01540409
First received: February 23, 2012
Last updated: October 17, 2013
Last verified: July 2012

February 23, 2012
October 17, 2013
February 2012
April 2015   (final data collection date for primary outcome measure)
The primary efficacy endpoint will be the change from baseline in the percent of dystrophin positive fibers in muscle biopsy tissue as measured by immunohistochemistry (IHC). [ Time Frame: 20 Weeks ] [ Designated as safety issue: Yes ]
The primary efficacy endpoint will be based on the pre-treatment and post-treament percent of dystrophin positive fibers as measured in the muscle biopsy tissue on(IHC).
Same as current
Complete list of historical versions of study NCT01540409 on ClinicalTrials.gov Archive Site
The secondary efficacy endpoints will be the change from baseline in: CD3, CD4, and CD8 lymphocyte counts in muscle biopsy tissue; 6-Minute Walk Test (6-MWT) distance. [ Time Frame: 20,80 Weeks ] [ Designated as safety issue: Yes ]
A key secondary efficacy endpoint will be based on the pre-treatment and post-treatment CD3, CD4, and CD8 lymphocyte counts as measured in the muscle biopsy tissue as well as a 6-Minute Walk Test(6-MWT)distance.
Same as current
Not Provided
Not Provided
 
Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201

The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 80 weeks of treatment with eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) subjects who have successfully completed the 28-week eteplirsen study: Study 4658-US-201. This study will also evaluate the correlation between biomarkers for DMD and the clinical status of participating DMD subjects.

Not Provided
Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Duchenne Muscular Dystrophy
Drug: AVI-4658 (Eteplirsen)
Eteplirsen will be administered once weekly via an IV infusion over a period of at least 60 minutes. Their are two treatment groups, 30 mg/kg and 50 mg/kg.
Experimental: AVI-4658 (Eteplirsen)
Multiple-Dose Extension Study
Intervention: Drug: AVI-4658 (Eteplirsen)
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
12
July 2015
April 2015   (final data collection date for primary outcome measure)

Inclusion Criteria:

A subject must meet all of the following criteria to be eligible for this study.

  1. The subject and/or their parent/legal guardian are willing and able to provide signed informed consent.
  2. The subject has successfully completed 28 weeks of treatment in Study 4658-US-201.
  3. The subject has a parent(s) or legal guardian(s) who is able to understand and comply with all of the study procedure requirements.

Exclusion Criteria:

A subject who meets any of the following criteria will be excluded from this study.

1. The subject has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the subject or make it unlikely that the course of treatment or follow-up would be completed or impair the assessment of study results.

Male
7 Years to 13 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01540409
4658-us-202
No
Sarepta Therapeutics
Sarepta Therapeutics
Not Provided
Principal Investigator: Jerry R Mendell, MD Nationwide Childtren's Hospital
Sarepta Therapeutics
July 2012

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